A Prospective, Multicenter, Randomized, Study of the Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)
Phase: PHASE2
Conditions: Retinitis Pigmentosa
Sponsor: jCyte, Inc

 
 
23 Corporate Plaza, Suite 150, Newport Beach, CA 92660 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Amendment 6 to the JC-02 Clinical Research Protocol 
 
October 26, 2017 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
A Prospective, Multicenter, Randomized, Study of the Safety and Efficacy of 
Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult 
Subjects with Retinitis Pigmentosa (RP) 
 
NCT03073733 
 
 
 
 
 
 

jCyte, Inc 
Clinical Study Protocol
Protocol No. JC-02 Amendment 6  SOC 
26 October  2017
 
Page 1 of 6 
 
 
PROTOCOL TITLE: 
A Prospective, Multicenter, Randomized, Study 
of the Safety and Efficacy of Intravitreal 
Injection of Human Retinal Progenitor Cells 
(jCell) in Adult Subjects with Retinitis 
Pigmentosa (RP) 
PROTOCOL NUMBER: 
JC-02 
SPONSOR: 
jCyte, Inc 
IND NUMBER: 
IND 016299 
 
ORIGINAL PROTOCOL DATE: 
Amendment 1 Date: 
Amendment 2 Date: 
Amendment 2.1 Date: 
Amendment 3 Date: 
Amendment 4 Date: 
Amendment 5 Date: 
Amendment 6 Date: 
 
August 31, 2016 
January 6, 2017 
March 22, 2017 
April 13, 2017 
June 24, 2017 
August 14, 2017 
September 6, 2017 
October 26, 2017 
 
 
 
 
 

jCyte, Inc 
Clinical Study Protocol
Protocol No. JC-02 Amendment 6  SOC 
26 October  2017
 
Page 4 of 6 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Change: 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
charts used in a regular eye examination.  
These special low vision eye charts contain 
different-sized letters or numbers that can help 
determine the sharpness or clarity of the 
subject’s distance vision.  Performed properly, 
the examiner obtains not only the refractive 
error, but additional potentially essential 
information, such as the level and quality of 
visual acuity, sensitivity to blur, effects of 
glare and the quality of fixation. The optical 
theory involved is the same as when refracting 
the normal eye, but special adjustments in lens 
selection, presentation strategy, and “just 
noticeable difference”, are incorporated for low 
vision patients.  These adjustments usually 
include large lens increments and special 
techniques for exploring and refining cylinders. 
 
BCVA will be tested at scheduled time points 
in both the treated and the contralateral eye. 
Visual acuity will be measured with the 
electronic visual acuity testing algorithm (E-
ETDRS) for eyes with vision better than 
20/800, including all study eyes at baseline.  
The Freiburg Visual Acuity & Contrast Test 
(FrACT) using Landolt C’s has a broader range 
for testing and should be used for eyes worse 
than 20/800.  For any individual eye, the same 
method must be used throughout the study for 
consistency unless the eye has deteriorated to 
the point where E-ETDRS can no longer be 
administered.  
Trial frame refraction is the gold standard in 
obtaining the most accurate refractive error in 
low vision patients.  It involves the use of a 
trial frame, loose lenses and specialized low 
vision eye charts that are different from the eye 
charts used in a regular eye examination.  
These special low vision eye charts contain 
different-sized letters or numbers that can help 
determine the sharpness or clarity of the 
subject’s distance vision.  Performed properly, 
the examiner obtains not only the refractive 
error, but additional potentially essential 
information, such as the level and quality of 
visual acuity, sensitivity to blur, effects of 

jCyte, Inc 
Clinical Study Protocol
Protocol No. JC-02 Amendment 6  SOC 
26 October  2017
 
Page 6 of 6 
 
 
 
 
 
 
 
 
Reason for change: 
Following successful culture and testing, 5065 
µl of cell suspension containing 13 million or 6 
million human retinal progenitor cells 
suspended in clinical grade medium (BSS 
PLUS) will be provided by the dose 
preparation facility to the clinical site, on ice, 
with targeted administration to the patient 
within 4 hours of cell harvest. 
 
Should have been changed in a prior version; 
oversight. 
 
 

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Clinical Study Protocol
Protocol No. JC-02 
 
CONFIDENTIAL 
 
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CLINICAL RESEARCH PROTOCOL 
 
PROTOCOL TITLE: 
A Prospective, Multicenter, Randomized, Study of the 
Safety and Efficacy of Intravitreal Injection of Human 
Retinal Progenitor Cells (jCell) in Adult Subjects with 
Retinitis Pigmentosa (RP) 
PROTOCOL NUMBER: 
JC-02 
SPONSOR: 
jCyte, Inc. 
IND NUMBER: 
016299 
 
ORIGINAL PROTOCOL DATE: 
Amendment 1 Date:  
Amendment 2 Date: 
Amendment 2.1 Date: 
Amendment 3 Date: 
Amendment 4 Date: 
Amendment 5 Date: 
Amendment 6 Date: 
August 31, 2016 
January 6, 2017 
March 22, 2017 
April 13, 2017 
June 24, 2017 
August 14, 2017 
September 6, 2017 
October 26, 2017 
 
 
 

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Protocol No. JC-02 
 
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Investigator Protocol Agreement 
 
The signature below constitutes that I agree to the following: 
• I have reviewed the protocol and the attachments. 
• This trial will be conducted according to all stipulations of the protocol, including all 
statements regarding confidentiality, and according to local legal and regulatory 
requirements and applicable United States federal regulations and International 
Conference on Harmonization (ICH) guidelines. 
• I agree to periodic site monitoring of case report forms and source documents by the 
Sponsor or designee and by appropriate regulatory authorities. 
• I agree to supply the University of California, Irvine or designee with any information 
regarding ownership interest and financial ties with the Sponsor for the purpose of 
complying with regulatory requirements. 
 
 
 
Investigator Name (Print) 
 
Investigator Signature 
 
Date 
 
 

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Study Synopsis 
NAME OF COMPANY:jCyte, Inc 
NAME OF FINISHED PRODUCT: 
jCell 
NAME OF ACTIVE INGREDIENT: 
human retinal progenitor cells 
SUMMARY TABLE 
Volume:                       
Page:                            
Reference:                   
(For national Authority Use Only) 
Title of study: 
JC-02: A Prospective, Multicenter, Randomized, Study of the Safety and Efficacy of Intravitreal Injection of Human 
Retinal Progenitor Cells (jCell) in Adult Subjects with Retinitis Pigmentosa (RP) 
Investigators/Study Centers:  Dr. Mitul Mehta, University of California, Irvine/Gavin Herbert Eye Institute; Dr. 
David Liao, Retina-Vitreous Associates Medical Group, Los Angeles CA, others TBD 
Study Period: 2017-2020 
Phase of development: Phase 2b 
Objectives: 
Primary Objective 
To assess the changes in visual function as measured by best corrected visual acuity (BCVA) at 12 months following a 
single injection of jCell (Test) compared to sham-treated controls in a cohort of adult subjects with RP with baseline 
BCVA in tstudy eye equal to or worse than 20/80. 
Secondary Objectives 
• 
To assess the impact of jCell injection based on visual function (VF, CS) and functional vision (mobility, VA 
LV-VFQ-48), at specified time points over a 12 month observation period 
• 
To evaluate the safety and tolerability of jCell injection in subjects with RP 
Methodology: 
This is a prospective, multicenter, randomized, three-arm, single-masked, Phase 2b trial of human retinal progenitor 
cells (jCell) for the treatment of retinitis pigmentosa (RP). Study subjects will be screened for eligibility, informed 
consent obtained, and eligible subjects will be randomized 1:1:1 to either treatment with 3 x 106 jCell, 6 x 106 jCell or 
to sham treatment (mock injection). Randomization will be stratified by study site. Blood samples for HLA typing and 
antibody testing will be collected at baseline.  Blood samples for gene mutation typing may be collected anytime during 
the study. 
Test subjects will receive an intravitreal injection of either 3.0 or 6.0 x 106 hRPC (65 µL volume), depending upon 
randomization assignment, into the study eye; only one eye will be injected. Control subjects will receive a mock 
injection (sham treatment).  They will undergo the same screening and baseline testing. Control subjects will undergo 
essentially the same treatment procedure as Test subjects, except that instead of an intravitreal injection, they will 
receive a mock injection, that is, after topical anesthesia, the surface of the study eye will be pressed with the hub of an 
empty syringe to mimic the injection of cells, but no actual injection will be performed.   
Subjects will be monitored closely following injection or mock injection for 60 minutes prior to being released home 
on the day of treatment, based on intraocular pressure <30mm Hg and vital signs returned to pre-injection.  Following 
treatment, all subjects will be treated with ophthalmic corticosteroid eye drops to minimize any inflammation from 
injection for up to 7 days (including tapering schedule).  All subjects will receive treatment (jCell or mock injection) 
into the eye designated as the “study eye”.  Subjects will be followed for 12 months for safety and evidence of effect. 
Once a study subject has completed 12 months of follow up, that subject may request to have his/her treatment 
assignment revealed.  If the subjects was assigned to the Control group, the subject will be offered the opportunity to 
cross over to the Test group, assuming no safety or other issues that preclude this, and will be randomized to one of the 
two dose levels.  
Blood samples for antibody testing (PRA and DRA) will be collected at baseline and at months 1 and 12. 
Number of patients:  Subjects will be randomized 1:1:1 to the 3.0 x 106 dose level, the 6.0 x 106 dose level or the 
sham treatment control group.  A sample size of 25 subjects per treatment arm is needed to achieve the targeted level of 
statistical power; the trial could enroll up to 85 subjects if needed to address irregularities in trial conduct.  

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NAME OF COMPANY:jCyte, Inc 
NAME OF FINISHED PRODUCT: 
jCell 
NAME OF ACTIVE INGREDIENT: 
human retinal progenitor cells 
SUMMARY TABLE 
Volume:                       
Page:                            
Reference:                   
(For national Authority Use Only) 
Diagnosis and main criteria for inclusion: 
Inclusion Criteria  
The following conditions must be met before a subject may be enrolled in the study. 
 
1. Willing to give written informed consent, able to make the required study visits and follow study protocol 
instructions 
2. Clinical diagnosis of RP with visual acuity no better than 20/80 and no worse than 20/800 in the eye to be 
treated (study eye); if both eyes fall within this range, the non-dominant eye should be designated as the study 
eye. 
3. Absence of macular edema  
4. Subject must consent to testing for RP gene mutation typing 
5. Male or female, age 18 yr or older 
6. Adequate organ function: 
o 
blood counts (hematocrit, Hgb, WBC, platelets and differential) within normal range, or if outside of 
normal range, not clinically significant as judged by the investigator 
o 
liver function:  alanine transaminase [ALT] and aspartate transaminase [AST] ≤2 times the upper 
limit of the normal range 
o 
total bilirubin ≤1.5 times the upper limit of the normal range 
o 
renal function:  serum creatinine ≤1.25 times the upper limit of the normal range 
7. Negative infectious disease screen (hepatitis B, C or HIV).   
8. Willing to provide a blood sample for HLA typing, if not done previously with available results 
9. A female patient of childbearing potential must have a negative pregnancy test (urine human chorionic 
gonadotropin) at entry (prior to the first infusion).  Note:  Women of childbearing potential and men must be 
advised to use a medically accepted method of contraception for at least 4 months following the last injection.  
Exclusion Criteria 
Patients will be excluded from this study if they meet any of the following criteria: 
1. Eye disease other than RP that impairs visual function (including retinal vascular disease, elevated intraocular 
pressure/glaucoma, severe posterior uveitis, clinically significant macular edema), or media opacity 
precluding visual exam, as well as patients who require other intravitreal therapies 
2. Pseudo-RP, cancer-associated retinopathies (CAR, MAR) excluded as part of differential diagnosis  
3. History of malignancy, end-stage major organ disease, heart failure, significant arrhythmias, stroke or 
transient ischemic attacks, diabetes, immunosuppressive or autoimmune state, major psychiatric disorder, 
epilepsy, COPD, renal failure, or any chronic systemic disease requiring continuous treatment with systemic 
steroids, anticoagulants or immunosuppressive agents. 
4. Known allergy to penicillin or streptomycin. 
5. History of adverse reaction to 
 
6. Unable or unwilling to undergo genetic testing, pupil dilation, topical anesthesia or any protocol-required 
procedure. 
7. Women who are nursing or who are planning to nurse during the 12 months that would follow study 
treatment. 
8. Any circumstance that in the opinion of the investigator, would interfere with participation in, or compliance 
with the study protocol 

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NAME OF COMPANY:jCyte, Inc 
NAME OF FINISHED PRODUCT: 
jCell 
NAME OF ACTIVE INGREDIENT: 
human retinal progenitor cells 
SUMMARY TABLE 
Volume:                       
Page:                            
Reference:                   
(For national Authority Use Only) 
9. Treatment with corticosteroids (systemic, periocular or intravitreal) or any other non-approved, experimental, 
investigational or neuroprotectant therapy (systemic, topical, intravitreal) in either eye within 90 days of study 
enrollment 
10. Cataract surgery within three months prior to enrollment or anticipated to need cataract surgery within a year 
of treatment 
11. Participation in a clinical trial for eye disease within 6 months. 
12. Previous participation in a jCyte clinical trial. 
Test product, dose and mode of administration: 
The investigational product (jCell) is a live suspension of  human retinal progenitor cells (hRPC) suspended in clinical 
grade medium.  The hRPC are of allogeneic human fetal origin. Study subjects randomized to one of the test groups 
will receive a single dose of either 3.0 x 106 jCell or 6.0 x 106 jCell as an intravitreal injection under topical anesthesia 
into the study. 
Reference product, dose and mode of administration: 
There is no reference product, however subjects randomized to the Control group will undergo sham treatment, e.g., a 
mock injection administered as an empty hub without a needle in the study eye. 
Duration of treatment:  
Treatment with jCell consists of a single intravitreal injection. The injection procedure itself takes approximately 2 
minutes. Patients will be released to home on the day of injection.   Subjects randomized to the control group will 
receive a mock injection. All study subjects will be followed for one year from injection (Test) or baseline (Control). 
Criteria for Evaluation: 
Efficacy:  
Best corrected visual acuity (BCVA) will be used as the primary visual function parameter in this study.  Other efficacy 
outcome measures that will be followed in test and control subjects include mobility testing, VA LV-VFQ 48, VF, and 
contrast sensitivity.   
Safety: 
Safety will be assessed on an ongoing basis by adverse events, identification of dose-limiting toxicities (if any), 
physical examinations and vital signs, clinical laboratory values, and anti-drug antibodies. In addition, specific 
ophthalmalogic tests to monitor safety will be performed, including slit lamp and fundus examiniation, B-scans and 
intraocular pressure (IOP).  Additional ophthalmalogic tests (IOP, B-scan, OCT) will also contribute to safety 
monitoring. 
Clinical safety data will be reviewed on an ongoing basis.   
Statistical methods: 
Background and demograhic data will be summarized for all subjects using descriptive statistics. 
Efficacy: 
Primary endpoint and analysis: 
The primary efficacy endpoint will be the 12-month mean change in the study eye of the best corrected visual acuity 
(BCVA), to be measured with the electronic visual acuity testing algorithm (E-ETDRS).  The eligible study subjects 
will be randomized 1:1:1 to either treatment with 3 x 106 jCell, 6 x 106 jCell or to sham treatment (mock injection).  
The primary analyses of the primary endpoint will be based on two pairwise comparisons of the mean change at 12 
months in BCVA for the study eyes between each experimental j-Cell group and the Control group.  Assume the 
standard deviation for the 12-month change in BCVA is 5 letters.  Then, for each pairwise comparison with the control 
group, a randomized trial with 25 patients per arm will have 90% power to detect a true treatment effect of a mean of 5 

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NAME OF COMPANY:jCyte, Inc 
NAME OF FINISHED PRODUCT: 
jCell 
NAME OF ACTIVE INGREDIENT: 
human retinal progenitor cells 
SUMMARY TABLE 
Volume:                       
Page:                            
Reference:                   
(For national Authority Use Only) 
letters, when using a t-test having (one-sided) 0.0125 false positive error rate.  
Secondary endpoints and analyses: 
For assessment of mobility, similar methods will be used as have been reported for the RPE65 studies.  Briefly, 
subjects who are capable of maze navigation will be evaluated for their performance in navigating a mobility course 
under a variety of specified light levels ranging from 0.1 lux (dimmer than a moonless summer night) to 500 lux (a 
brightly lit office) using bilateral testing condition. Each attempt will be recorded, and the videos will be sent to 
independent, centralized, masked graders to assign a score based on speed and accuracy with which the subjects 
navigate the course. The mean improvement in functional vision at specified lux levels, as measured by the change in 
mobility testing using the study eye will be calculated for the three treatment groups. 
Other visual test results will be compared between the 3 treatment groups using analysis of covariance (ANCOVA) 
method with baseline values as covariates. Subjects who cannot perform any specific test at baseline (e.g. mobility) 
will not be considered evaluable for that analysis. 
Similar analytical methods will be used for QOL measures. The total and individual scores from VA LV VFQ-48 will 
be analyzed using linear mixed model, which is designed for longitudinal study with correlated measurements. The 
Pearson Chi-square test with alpha levels adjusted for multiplicity issues will be applied to evaluate the proportion of 
subjects who show specific improvement compared to the proportion of control subjects in any secondary endpoints at 
the pre-specified time of primary analysis. 
 
Safety: 
The safety analyses of AEs and laboratory parameters will include descriptive statistics.  Summaries of treatment-
emergent AEs (TEAE) will be generated by type (AE or SAE), body system and preferred term, severity, and 
relationship to study product.   
 
 

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TABLE OF CONTENTS 
INVESTIGATOR PROTOCOL AGREEMENT ................................................................................. 2 
1.0 
INTRODUCTION........................................................................................................... 10 
1.1 
BACKGROUND ........................................................................................................... 10 
1.1.1 
Retinitis Pigmentosa ......................................................................................... 10 
1.1.2 
Stem /Progenitor Cells ...................................................................................... 10 
1.1.3 
CNS and Retinal Progenitor Cells .................................................................... 11 
1.2 
JCELL (HUMAN RETINAL PROGENITOR CELLS, HRPC) .............................................. 12 
1.3 
RATIONALE FOR THE PROPOSED STUDY .................................................................... 13 
2.0 
OBJECTIVES ................................................................................................................. 15 
2.1 
PRIMARY OBJECTIVE ................................................................................................. 15 
2.2 
SECONDARY OBJECTIVES .......................................................................................... 15 
3.0 
STUDY DESIGN ............................................................................................................. 15 
3.1 
RANDOMIZATION AND MASKING .............................................................................. 15 
3.2 
STUDY POPULATION .................................................................................................. 15 
3.3 
ENDPOINTS ................................................................................................................ 16 
3.3.1 
Efficacy Endpoints ............................................................................................ 16 
3.3.2 
Safety Endpoints ............................................................................................... 16 
3.4 
STUDY DURATION ..................................................................................................... 16 
3.5 
EARLY STUDY TERMINATION .................................................................................... 17 
4.0 
SUBJECT SELECTION ................................................................................................ 17 
4.1 
INCLUSION CRITERIA ................................................................................................. 17 
4.2 
EXCLUSION CRITERIA ............................................................................................... 18 
4.3 
SUBJECT WITHDRAWAL CRITERIA ............................................................................ 19 
4.4 
DISCONTINUATION/WITHDRAWAL PROCEDURES ...................................................... 19 
5.0 
SUBJECT REGISTRATION AND RANDOMIZATION .......................................... 19 
6.0 
TREATMENT PLAN ..................................................................................................... 20 
6.1 
DOSE AND SCHEDULE................................................................................................ 20 
6.2 
TREATMENT COMPLIANCE ........................................................................................ 22 

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6.3 
SUPPORTIVE CARE/PROHIBITED TREATMENTS .......................................................... 22 
7.0 
STUDY EVALUATIONS ............................................................................................... 22 
7.1 
SCREENING ................................................................................................................ 22 
7.2 
BASELINE (SHOULD BE WITHIN 30 DAYS OF TREATMENT) ......................................... 23 
7.3 
BEFORE ADMINISTRATION OF JCELL OR MOCK INJECTION (DAY 0) ........................... 23 
7.4 
AFTER ADMINISTRATION OF A SINGLE INTRAVITREAL DOSE OF JCELL OR MOCK 
INJECTION (DAY 0) ............................................................................................................... 23 
7.5 
FOLLOW UP VISITS .................................................................................................... 24 
7.5.1 
Day 1 (24 hours) ............................................................................................... 24 
7.5.2 
Day 7 (week one) .............................................................................................. 24 
7.5.3 
Day 28 (week four/month 1) +/- 1 day ............................................................. 24 
7.5.4 
Month 3 (Day 90) +/- 4 days............................................................................. 25 
7.5.5 
Month 6 (Day 180) +/- 4 days........................................................................... 25 
7.5.6 
Month 9 (Day 270) +/- 4 days........................................................................... 25 
7.6 
END OF TREATMENT (MONTH 12) OR EARLY TERMINATION VISIT ........................... 26 
8.0 
ASSESSMENTS .............................................................................................................. 26 
8.1 
SAFETY ASSESSMENTS .............................................................................................. 26 
8.1.1 
Adverse Events ................................................................................................. 26 
8.1.2 
Vital Signs ......................................................................................................... 27 
8.1.3 
Clinical Laboratory Tests .................................................................................. 27 
8.1.4 
Blood Samples for Antibody Testing ............................................................... 27 
8.1.5 
RP Gene Mutation Typing and HLA Typing ................................................... 27 
8.1.6 
Ophthalmic AEs ................................................................................................ 27 
8.2 
EFFICACY ASSESSMENTS ........................................................................................... 28 
8.2.1 
BCVA (E-ETDRS) ........................................................................................... 28 
8.2.2 
Visual Field Testing .......................................................................................... 29 
8.2.3 
Autoflourescence .............................................................................................. 29 
8.2.4 
Other Visual Assessments................................................................................. 29 
9.0 
SAFETY CONSIDERATIONS ..................................................................................... 30 

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9.1 
ADVERSE EVENTS ..................................................................................................... 30 
9.2 
SERIOUS AND UNEXPECTED ADVERSE EVENTS ......................................................... 31 
10.0 
STATISTICAL CONSIDERATIONS .......................................................................... 32 
10.1 
STUDY POPULATION .................................................................................................. 32 
10.2 
EFFICACY ANALYSES ................................................................................................ 32 
10.2.1 
Primary Efficacy Endpoint and Analysis .......................................................... 32 
10.2.2 
Secondary Efficacy Endpoints and Analyses ................................................... 32 
10.3 
SAFETY ANALYSES ................................................................................................... 33 
10.4 
OTHER ANALYSES ..................................................................................................... 33 
11.0 
ADMINISTRATIVE CONSIDERATIONS ................................................................. 33 
11.1 
ADVERSE EXPERIENCES ............................................................................................ 33 
11.2 
INSTITUTIONAL REVIEW ............................................................................................ 33 
11.3 
INFORMED CONSENT ................................................................................................. 33 
11.4 
MONITORING PROCEDURE ......................................................................................... 34 
11.5 
REPORTING AND RECORDING OF DATA ..................................................................... 34 
11.6 
CHANGES IN PROTOCOL ............................................................................................ 35 
11.7 
INVESTIGATIONAL PRODUCT AND LABEL CODES ...................................................... 35 
11.7.1 
Investigational Drug.......................................................................................... 35 
11.7.2 
hRPC Reconstitution (Thaw and Culture) and Administration ........................ 35 
11.8 
PRODUCT SECURITY .................................................................................................. 35 
 
 
 

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1.0 
INTRODUCTION 
1.1 
Background 
1.1.1 Retinitis Pigmentosa 
Retinitis pigmentosa (RP) refers to a group of inherited diseases causing retinal degeneration. 
The cell-rich retina lines the back inside wall of the eye. It is responsible for capturing 
images from the environment. People with RP experience a gradual decline in their vision 
because photoreceptor cells (rods and cones) die. Forms of RP and related diseases include 
Usher syndrome, Leber’s congenital amaurosis, rod-cone degeneration, Bardet-Biedl 
syndrome, and Refsum disease, among others. 
In most forms of RP, rods are affected first. Because rods are concentrated in the outer 
portions of the retina and are triggered by dim light, their degeneration affects peripheral and 
night vision. When the more centrally located cones - responsible for color and sharp central 
vision - become involved, the loss is in color perception and central vision. Night blindness is 
one of the earliest and most frequent symptoms of RP.  
RP is typically diagnosed in adolescents and young adults.  The classic clinical sign of the 
disease is the presence of dark deposits in the retina.  The main risk factor is a family history 
of retinitis pigmentosa. It is an uncommon condition affecting about 1 in 4,000 people or 
roughly 100,000 individuals in the US1. Cases of RP are associated with a wide variety of 
known gene mutations, with new mutations still being discovered. The mutations can be 
inherited from one or both parents, or occur spontaneously; the pattern of inheritance can be 
autosomal recessive, autosomal dominant, X-linked or mitochondrial. 
There is no effective treatment for RP; once photoreceptors are lost, they do not regenerate. 
The rate of deterioration of vision varies from person to person, with most people with RP 
legally blind by age 40.  People with RP also often develop cataracts at an early age, or 
swelling of the retina (retinal edema).  Numerous breakthroughs in the treatment of cataract 
and corneal disease have greatly decreased the incidence of blindness from these causes. In 
contrast, treatment of retinal and optic nerve disease is more limited, with these conditions 
now representing major causes of incurable visual loss and a significant unmet medical need. 
1.1.2 Stem /Progenitor Cells 
No restorative treatments for retinal cell loss currently exist, but stem cell treatment has 
emerged as a particularly promising strategy. In addition, the concept of delaying retinal cell 
death through the use of neuroprotective agents has considerable merit and committed 
progenitor cells are useful platforms for delivery of neuroprotective cytokines. Also, the 
possibility of reinvigorating non-functional yet viable cones is an underappreciated yet 
attractive potential clinical target. 

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Cells and tissues of many types survive injection to the subretinal space, in part because this 
location exhibits characteristics often referred to as those of an “immune privileged” site2. 
Both photoreceptors3 and RPE cells4,5 survive beneath the retina, however, failure of donor 
photoreceptors to integrate with surviving host circuitry and failure of donor RPE cells to 
adhere to Bruch’s membrane have thus far frustrated attempts to achieve functional repair 
using these methods. For photoreceptors, there is a fundamental problem that must be 
overcome, namely the physical barrier to neurite outgrowth posed by the outer limiting 
membrane (OLM). In photoreceptor degeneration, the OLM undergoes hypertrophy and 
regenerating neurites are impeded by this barrier6.  Glial hypertrophy has been often been 
implicated in the failure of endogenous regenerative mechanisms to bridge a lesion7, e.g., 
after spinal cord injury8. 
1.1.3 CNS and Retinal Progenitor Cells 
It has been demonstrated that transplanted CNS progenitor cells are not impeded by the 
hypertrophied OLM and cross in large numbers9,10. The ability to migrate into the mature 
retina is characteristic of CNS progenitor cells, which do not simply migrate, but exhibit 
widespread integration into the local cytoarchitecture, with pronounced tropism for areas of 
disease. 
Hippocampal progenitors transplanted to the vitreous of neonatal rats integrated into the 
retina and developed morphologies appropriate to their layer of residence11.  In the 
dystrophic Royal College of Surgeons (RCS) rat, it has been shown that grafted hippocampal 
progenitors developed rod photoreceptor-like morphologies and extended neurites into the 
optic nerve9; however, brain-derived progenitors did not express retina-specific markers. This 
lineage restriction has been overcome using progenitor cells derived from the retina. Retinal 
progenitor cells can be derived from the developing neural retina of rats, mice, pigs, cats and 
humans. 
The first simultaneous sourcing of retinal- and brain-derived neural progenitors from the 
same premature infant occurred in 1999 and both progenitor cell types were found to express 
MHC I antigens, but not MHC II12.  Cultured hRPCs expressed a range of markers consistent 
with CNS progenitor cells.  hRPCs could be distinguished from human brain progenitor cells 
by the expression of retinal specification genes, particularly recoverin. More recently, age 18 
weeks gestation was found to be a suitable age developmental stage for isolation of hRPCs.  
Although rejection is the norm for grafts between individuals of disparate genetic 
background, this tendency is markedly less problematic when placed in an “immune 
privileged” site such as the eye. This does not mean that allogeneic grafts to these sites 
cannot be rejected, but that they benefit from a decreased likelihood of rejection. It has also 
been shown that CNS progenitor cells themselves exhibit properties of cell-specific immune 
privilege. Rat hippocampal progenitor cells were not recognized by human mononuclear cells 
in vitro13 and murine brain progenitor cells survived transplantation to the allogeneic kidney 

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remarkable for nonspecific musculoskeletal abnormalities, myocardial bridge, 
hypercholesterolemia, clavicle fracture, ruptured disc, hypertension, colon polyps, 
esophageal reflux, fracture reduction, coronary artery disease, vitiligo, diabetes (Type II), 
vitamin D deficiency, actinic keratosis, obstructive sleep apnea and anemia.  The study 
investigator reported the event as possibly related to study treatment initially but after further 
testing indicated that the event was unlikely to be related.  
Based on the demonstration of acceptable safety and tolerability in the phase 1/2a study at 
doses up to 3 x 106 hRPC, this phase 2b study is designed as a controlled comparison of the 
changes in visual function and functional vision in subjects who receive a single jCell 
injection in comparison to a comparable untreated control group of subjects with RP.  The 
3.0 x 106 and 6 x 106 cell doses were selected for the phase 2b study based on the results of 
BCVA testing in the phase 1/2a study, which are suggestive of a dose response relationship, 
with best results at the highest dose (3 x 106 jCell). Because of the suggestion of a dose 
response in the phase 1/2a study, and because it is technically feasible to increase the dose to 
6 x 106 cells without significantly increasing the injection volume, it was considered 
reasonable to add one additional dose level in the phase 2b study that would be likely to 
show a dose response difference to the 3 x 106 dose level.  All subjects randomized to the 
Test group will receive an intravitreal injection of either 3 x 106 hRPC or 6 x 106 hRPC, 
depending upon randomization assignment, in the eye that meets the inclusion criterion for 
visual acuity range (study eye).  
In the phase1/2a study, the largest improvement in BCVA was seen in subjects at the highest 
dose level (difference of 9 letter change between the test and control eyes).  The mean 
change in BCVA in the test eye groups compared to the control eyes will be used as the 
primary visual function parameter in this study.  Other efficacy outcome measures that will 
be followed in these subjects at periodic intervals throughout the study include mobility 
testing, VA LV-VFQ 48, VF, and contrast sensitivity.  There are many components to vision; 
visual acuity is a standard and expected endpoint in vision studies, although perhaps not the 
most relevant endpoint in the poor vision subjects in these studies.  The other methods are 
selected based on preliminary assessment of a battery of visual assessment tools in the phase 
1/2a study subjects by low vision experts who have indicated that these are likely to be 
effective methods in subsets of study subjects, depending upon their visual acuity range 
and/or because they have been shown to be endpoints of value in other studies of low vision 
subjects. Data for these endpoints will be compared to the subject’s baseline status and 
changes in the treated groups will be compared to those in the control (sham-treated) group.   
The phase 2b study design is focused on subjects with visual acuity of 20/80 or worse in the 
“study eye”, without macular edema, and whose quality of life is already negatively impacted 
by the disease to a large degree. The cut off of 20/80 for the study eye is important to provide 
a sufficient “margin” for assessment of improvement after treatment.     

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2.0 
OBJECTIVES 
2.1 
Primary Objective 
The primary objective is to assess the changes in visual function as measured by BCVA at 12 
months following a single injection of jCell at one of two dose levels (Test) compared to 
sham-treated controls in a cohort of adult subjects with RP with baseline best corrected visual 
acuity (BCVA) in the study eye equal to or worse than 20/80 and no worse than 20/800 
2.2 
Secondary Objectives 
The secondary objectives of the study are  
• To assess the impact of jCell injection at two dose levels based on visual function 
(VF, CS) and functional vision (mobility, VA LV-VFQ-48)  
• To evaluate the safety and tolerability of jCell injection in subjects with RP  
3.0 
STUDY DESIGN 
This is a prospective, multicenter, randomized, single-masked, three-arm, Phase 2b trial of 
hRPC (jCell) for the treatment of retinitis pigmentosa (RP). Study subjects will be screened 
for eligibility and informed consent will be obtained.  
3.1 
Randomization and Masking 
 Following confirmation of eligibility, up to 85 subjects will be randomized 1:1:1 to one of 
two Test (jCell treatment) arms (3 x 106 jCell dose or 6 x 106 jCell dose) or to the Control 
(sham-treated) study group.  Study subjects will be masked with respect to which treatment 
they are receiving, although study Investigators and those involved in certain aspects of 
treatment and assessment (where injected cells are visualized) cannot feasibly be masked.  
However, site staff that perform key assessments (primary and secondary efficacy endpoints) 
will be masked to the treatment assignment.  Assessors will be told which eye is the study 
eye for each subject but will not know whether that subject is assigned to a Test or Control 
group.   
3.2 
Study Population 
The phase 2b study design is focused on subjects with visual acuity of 20/80 or worse in the 
study eye, without macular edema, and whose quality of life is already negatively impacted 
by the disease to a large degree. The cut-off of 20/80 for the study eye was selected to 
provide a sufficient “margin” for observation of potential benefit, as measured in terms of 
gains in BCVA.     
Subjects randomized to one of the Test groups will receive a single intravitreal injection of 
either 3.0 x 106 or 6 x 106 jCell into the study eye.  Subjects randomized to the Control group 
will not receive treatment, but will undergo a similar procedure that is a mock injection in the 

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study eye.  Subjects in both cohorts will be followed for one year for safety and evidence of 
jCell effects.  
3.3 
Endpoints 
3.3.1 Efficacy Endpoints 
Primary efficacy endpoint: 
• Mean change in BCVA in study eyes in subjects of each Test group compared to 
mean change in BCVA in the control group study eyes using E-ETDRS  
Secondary efficacy endpoints: 
• Changes in Test group in visual field area compared to the Control group, using 
primarily kinetic visual field testing  
• Changes in mobility test scores in the Test group vs the Control group  
• Changes in Test group peak grating contrast sensitivity thresholds compared to the 
Control group  
• Changes in Test group VA LV LFQ-48 total and individual scale scores compared to 
the Control group 
In addition to the assessments listed above, a number of additional visual assessments 
will be conducted including OCT, AF, and ERG.   
3.3.2 Safety Endpoints 
The criteria that will be used to assess safety include:  
• Incidence and severity of treatment-emergent adverse events (TEAE) 
• Immunogenicity (PRA, DRA) 
• Safety visual assessments: slit lamp and fundus exam, IOP, AF, B-scan 
3.4 
Study Duration 
The overall duration of the study is anticipated to be approximately 3.5 years.  The study will 
be considered to have started when the first site is initiated.   
At the end of follow up (12 months) for each study subject, the subject’s treatment 
assignment may be revealed to the subject.  If the subject was assigned to the Control group, 
the subject may elect at that time to cross over to the Test group and receive jCell treatment, 
assuming no safety issues have been noted that may preclude the treatment.  The duration of 
the study is based on the assumption that some or all of the Control subjects will opt to cross 
over after they complete 12 months of follow up.  When this occurs the subjects will revert to 
a new “Day 0” at the time of treatment and will be followed for an additional 12 months 
post-treatment.  The initial part of the study (primary endpoint evaluation) is anticipated to 
take approximately 30 months, including 18 months to enroll and treat all subjects and 12 

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months of follow up on all subjects. The study will be considered to have finished after the 
last subject (including Control subjects who have crossed over) has completed the last 
follow-up visit in the study. 
If deemed necessary or desirable for the purposes of further safety and/or efficacy 
monitoring, an extension study will be offered to subjects treated with jCell for up to 12 
additional months of monitoring.  
3.5 
Early Study Termination 
The Sponsor may terminate this study at any time. Reasons for termination may include but 
are not limited to, the following: 
• The incidence or severity of AEs in this or other studies point to a potential health 
hazard for study subjects. 
• Insufficient subject enrollment. 
• Any information becoming available during the study that substantially changes the 
expected benefit-risk profile of the investigational drug. 
4.0 
SUBJECT SELECTION 
4.1 
Inclusion Criteria  
The following conditions must be met before a subject may be enrolled in the study. 
 
1. Willing to give written informed consent, able to make the required study visits 
and follow study protocol instructions. 
2. Clinical diagnosis of RP confirmed by ERG with visual acuity no better than 
20/80 and no worse than 20/800 in the study eye. 
3. Absence of macular edema. 
4. Willing to consent to gene mutation typing. Mutation typing will be restricted to 
typing for eye disease-related genes known to be involved in inherited retinal 
degenerations and related disorders.  If typing results are already available for the 
subject, the previous results can be recorded and this requirement is waived. 
5. Male or female, age > 18 yr.  
6. Adequate organ function: 
• blood counts (hematocrit, Hgb, WBC, platelets and differential) within 
normal range, or if outside of normal range, not clinically significant as 
judged by the investigator 
• liver function:  alanine transaminase [ALT] and aspartate transaminase 
[AST] ≤2 times the upper limit of the normal range 

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• total bilirubin ≤1.5 times the upper limit of the normal range 
• renal function:  serum creatinine ≤1.25 times the upper limit of the normal 
range 
7. Negative infectious disease screen (hepatitis B, C or HIV).   
8. Willing to provide a blood sample for HLA typing, if not done previously with 
available results 
9. A female patient of childbearing potential must have a negative pregnancy test 
(urine human chorionic gonadotropin) at entry (prior to the first infusion).  Note:  
Women of childbearing potential and men must be advised to use a medically 
accepted method of contraception for at least 4 months following the last 
injection. 
4.2 
Exclusion Criteria 
Patients will be excluded from this study if they meet any of the following criteria: 
1. Eye disease other than RP that impairs visual function (including retinal vascular 
disease, elevated intraocular pressure/glaucoma, severe posterior uveitis, 
clinically significant macular edema), or media opacity precluding visual exam, as 
well as patients who require other intravitreal therapies 
2. Pseudo-RP, cancer-associated retinopathies (CAR, MAR) excluded as part of 
differential diagnosis  
3. History of malignancy, end-stage major organ disease, heart failure, significant 
arrhythmias, stroke or transient ischemic attacks, diabetes, immunosuppressive or 
autoimmune state, major psychiatric disorder, epilepsy, COPD, renal failure, or 
any chronic systemic disease requiring continuous treatment with systemic 
steroids, anticoagulants or immunosuppressive agents. 
4. Known allergy to penicillin or streptomycin. 
5. History of adverse reaction to DMSO. 
6. Unable or unwilling to undergo genetic testing, pupil dilation, topical anesthesia 
or any protocol-required procedure. 
7. Women who are nursing or who are planning to nurse during the 12 months that 
would follow study treatment. 
8. Any circumstance that in the opinion of the investigator, would interfere with 
participation in, or compliance with the study protocol 

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9. Treatment with corticosteroids (systemic, periocular or intravitreal) or any other 
non-approved, experimental, investigational or neuroprotectant therapy (systemic, 
topical, intravitreal) in either eye within 90 days of study enrollment 
10. Cataract surgery within three months prior to enrollment or anticipated to need 
cataract surgery within a year of treatment 
11. Participation in a clinical trial for eye disease within 6 months. 
12. Previous participation in a jCyte clinical trial. 
4.3 
Subject Withdrawal Criteria  
Subjects may withdraw from the study at any time.  Subjects may be discontinued from 
the study for any of the following reasons: 
• Withdrawal of consent by the subject 
• Lost to follow-up 
The investigator may also withdraw a subject at any time at his/her discretion.  The 
sponsor reserves the right to terminate the study or withdraw any subject from the study 
for any reason at any time.   
In addition, the sponsor reserves the right to temporarily suspend or prematurely 
discontinue this study either at a single site or at all sites at any time and for any reason.  
If such action is taken, the sponsor will discuss this with the investigator (including the 
reasons for taking such action) at that time.  The sponsor will promptly inform all other 
investigators and/or institutions conducting the study if the study is suspended or 
terminated for safety reasons, and will also inform the regulatory authorities of the 
suspension or termination of the study and the reason(s) for the action.  If required by 
applicable regulations, the investigator must inform the IRB promptly and provide the 
reason for suspension or termination. 
4.4 
Discontinuation/Withdrawal Procedures 
If a subject is withdrawn from the study (i.e., ceases participation in the study prior to 
completion of the assessments planned in the protocol), the primary reason should be 
recorded in the case report form (CRF). Investigators should make every effort to capture 
withdrawal assessments, which will be recorded in the CRF.   
The investigator will provide or arrange for appropriate follow-up (if required) for subjects 
withdrawing from the study, and will document the course of the subject’s condition. 
5.0 
SUBJECT REGISTRATION AND RANDOMIZATION 
At the time informed consent is obtained, qualified subjects will be assigned a study number 
and randomized.  Subjects will be randomized 1:1:1 to receive jCell at one of two dose levels 
(Test groups, 3.0 x 106 or 6.0 x 106 cells) or to sham-treatment, a mock injection that is 

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performed using essentially the same technique as jCell injection but without a needle 
(briefly pressing an empty syringe against the eye to simulate injection; Control group).  
Randomization will be performed by the sponsor and will occur once a subject is deemed 
eligible and agrees to study participation.  Randomization lists will be maintained by the 
sponsor’s designee and will not be provided to study sites, so that the next assignment will be 
unknown to site personnel at the time a subject is deemed eligible.  Randomization 
assignment will be masked for the study subjects, for certain site personnel performing key 
study assessments (BCVA) as well as for personnel conducting vision testing at a central 
facility.  Investigators and other site personnel will not be masked as they can visualize the 
cells while performing required post-treatment assessments and these observations must be 
documented.   Subjects and study staff will not be masked as to the assignment of the “study 
eye” since only one eye will be treated.  Study Investigators and other unmasked site staff 
will be instructed to take great care not reveal the subject’s treatment assignment or any 
information that might suggest the treatment assignment to the subject during the course of 
the study (e.g. that the cells can be seen or visualized during certain assessments). 
6.0 
TREATMENT PLAN 
6.1 
Dose and Schedule 
All subjects randomized to one of the two Test groups will receive a single intravitreal 
injection of 3.0 x 106 or 6.0 x 106 hRPC, depending upon randomization assignment, into the 
study eye. After topical anesthesia, a single 65 microliter suspension of cells will be 
delivered into the vitreous cavity using a 30 g needle. This treatment does not require 
surgical detachment or manipulation of the retina. The injection procedure itself takes 
approximately 2 minutes.  Following 60 minutes of observation with no serious clinical 
symptoms or manifestations (IOP < 30 mm Hg and vital signs comparable to pre-treatment), 
subjects will be released to home on the day of injection. Please refer to the study procedure 
manual for details of patient preparation and monitoring.  
Subjects randomized to the Control group will undergo a similar procedure in the designated 
study eye but with no injection.  After topical anesthesia, a syringe that replicates the cell 
delivery syringe will be pressed against the subject’s eye in a fashion that mimics the cell 
injection; no injection will actually be given.  Following 60 minutes of observation with no 
serious clinical symptoms or manifestations (IOP < 30 mm Hg and vital signs comparable to 
pre-treatment), subjects will be released to home on the day of injection. Please refer to the 
study procedure manual for details of patient preparation and monitoring. 
The schedule of observations and assessments during the study is summarized in Table 2.    

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Table 2  Schedule of Assessments 
Timepoint 
Scr 
BL* 
Day 0 
Day 1 
Day 75 
Day 285 
 
M36 
M66 
M96 
M12 or Early 
Term 
Written informed consent 
x 
 
 
 
 
 
 
 
 
 
 
Medical history (MH) 
x 
x* 
 
 
 
 
 
 
 
 
x 
Physical examination (PE) 
x 
x* 
 
 
 
 
 
 
 
 
x 
Brief MH and PE 
 
 
 
x 
x 
x 
 
x 
x 
x 
 
Vital signs 
x 
x 
x 
x 
x 
x 
 
x 
x 
x 
x 
Pregnancy test 
x 
x* 
 
 
 
 
 
 
 
 
x 
Safety laboratory tests 1 
x 
x* 
 
 
 
x 
 
x 
x 
 
x 
Infectious disease tests2 
x 
x* 
 
 
 
 
 
 
 
 
 
Urinalysis 
x 
x* 
 
 
 
 
 
x 
x 
 
x 
Blood sample for HLA typing 
x 
 
 
 
 
 
 
 
 
 
 
Blood sample for gene mutation  typing 
Any visit 
 
Slit lamp and fundus exam 
x 
x3 
 
x 
x 
x 
 
x 
x 
x 
x 
BCVA (E-ETDRS or FrACT if worse than 20/800)  
x 
x 
x 
x 
x 
x 
 
x 
x 
x 
x 
ERG 
x7 
 
 
 
 
 
 
 
 
 
 
IOP 
 
x 
x 
x 
x 
x 
 
x 
x 
x 
x 
OCT  
 
x 
 
 
 
 
 
 
x 
 
x 
AF 
 
x 
 
 
 
 
 
 
x 
 
x 
VF 
 
x 
 
 
 
 
 
 
x 
 
x 
B-scan 
 
x 
 
 
x 
 
 
 
x 
 
x 
Contrast sensitivity 
 
x 
 
 
 
 
 
 
x 
 
x 
VA LV-VFQ-48 
 
x 
 
 
 
 
 
 
x 
 
x 
Mobility test 
 
x 
 
 
 
 
 
 
x 
 
x 
Study Drug Injection 
 
 
x 
 
 
 
 
 
 
 
 
Con Meds 
 
 
x 
x 
x 
x 
 
x 
x 
x 
x 
Adverse events 
 
 
x 
x 
x 
x 
 
x 
x 
x 
x 
Blood sample for Ab testing4 
 
x 
 
 
 
x 
 
 
 
 
x 
1 Hematology (minimally Hct, Hgb, CBC with diff, plt), coagulation panel (screening only) and blood chemistries (minimally AP, ALT, AST, BUN, Cr, total bilirubin, serum 
electrolytes, glucose, calcium, phosphate, albumin, total protein and HbA1c) 
2 Hepatitis B and C, HIV     
3 Fundus photographs at baseline 
 
4 Testing for Panel Reactive Antibodies (PRA) and Donor Reactive Antibodies (DRA)  
5 Visit window +/- 1 day          
6 Visit window +/- 4 days 
 
7 ERG can be performed at screening to confirm eligibility.     
*Day -30 to 0.  Does not need to be repeated if Day 0 is within 30 days of screening.       

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6.2 
Treatment Compliance 
Treatment compliance will be assessed via direct observation by the study investigator who 
is responsible for study drug administration or mock injection. The cell dose and exact time 
of injection for the Test subjects will be recorded and the exact time of mock injection for the 
Control subjects will be recorded. 
6.3 
Supportive Care/Prohibited Treatments 
Subjects will receive supportive measures as determined by their needs and according to the 
accepted standards of care. All subjects will be treated with ophthalmic corticosteroid eye 
drops to minimize any inflammation for up to 7 days starting on the day of injection, 
including tapering schedule. If extended beyond 7 days, the reason for longer treatment 
should be documented.  Although Control subjects will not be injected, it is important that 
they also receive a short course of ophthalmic corticosteroid eye drops to maintain the 
masking. The prohibited treatments are any immunosuppressant other than topical steroids as 
noted above. 
7.0 
STUDY EVALUATIONS  
7.1 
Screening 
a. informed consent  
b. documentation of initial diagnosis confirmed by ERG (ERG can be performed at 
screening to confirm eligibility)  
c. consent to mutation typing (if not already done – sample for mutation typing can 
be drawn at any study visit)  
d. complete medical history and physical exam, including vital signs, height, weight 
e. pregnancy test, if female 
f. blood sample for HLA typing  
g. CBC, platelet, differential, hemoglobin, hematocrit 
h. blood chemistries, not limited to, but must include AP, ALT, AST, BUN, Cr, total 
bilirubin, serum electrolytes, glucose, calcium, phosphate, albumin and total 
protein 
i. coagulation function, including PT/INR, PTT 
j. infectious disease screen (Hepatitis B and C, HIV) 
k. urinalysis 
l. slit lamp and fundus exam 
m. BCVA  
n. ERG if needed to confirm eligibility 

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7.2 
Baseline (should be within 30 days of treatment)  
The assessments (indicated by an asterisk*) do not need to be repeated if Day 0 (treatment 
day) is within 30 days of screening. Subjects should be advised to discontinue daily aspirin or 
clopidogrel regimens for 5 days prior to study treatment.   
a. medical history and physical exam*, including height, weight 
b. vital signs 
c. pregnancy test, if female* 
d. hematology [CBC, platelet, differential, hemoglobin, hematocrit]* 
e. blood chemistries*, not limited to, but must include AP, ALT, AST, BUN, Cr, 
total bilirubin, serum electrolytes, glucose, calcium, phosphate, albumin and total 
protein 
f. infectious disease screen* (Hepatitis B and C, HIV) 
g. urinalysis* 
h. slit lamp and fundus exam, including fundus photographs 
i. BCVA (designation of study eye) 
j. IOP 
k. OCT  
l. AF 
m. B-scan 
n. VF 
o. contrast sensitivity 
p. VA LV-VFQ-48 
q. mobility test 
r. blood sample for Ab testing (Test only) 
7.3 
Before administration of jCell or mock injection (Day 0)  
a. vital signs, within 15 minutes prior to injection 
b. BCVA  
7.4 
After administration of a single intravitreal dose of jCell or mock injection (Day 
0) 
a. exact dose and time of injection or mock injection (treatment); any dose 
interruption must be documented 
b. vital signs at 15 and  60 minutes after treatment or until returned to pre-treatment 
levels 
c. intraocular pressure by tonometry post-treatment must be ≤30 mm Hg prior to  
patient release  
d. verify basic vision by lights, hand motions and fingers, depending upon individual 
patient’s baseline prior to patient release 
e. con meds 

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f. adverse events 
7.5 
Follow up Visits  
7.5.1 Day 1 (24 hours)  
a. brief medical history and physical exam, including any changes in vision or light 
perception reported by patient or noted by study staff not specifically assessed 
(for example, patient reports that general vision is “brighter” or study staff reports 
that patient response to certain assessments seems more rapid) 
b. vital signs 
c. slit lamp and fundus exam 
d. BCVA 
e. IOP 
f. con meds 
g. adverse events 
7.5.2 Day 7 (week one) 
a. brief medical history and physical exam, including any changes in vision or light 
perception reported by patient or noted by study staff since prior H & PE 
b. vital signs 
c. slit lamp and fundus exam 
d. BCVA 
e. B-scan 
f. IOP 
g. con meds 
h. adverse events 
7.5.3 Day 28 (week four/month 1) +/- 1 day  
a. brief medical history and physical exam, including any changes in vision or light 
perception reported by patient or noted by study staff since prior H& PE 
b. vital signs 
c. CBC, platelet, differential, hemoglobin, hematocrit 
d. blood chemistries, not limited to, but must include AP, ALT, AST, BUN, Cr, total 
bilirubin, serum electrolytes, glucose, calcium, phosphate, albumin and total 
protein 
e. slit lamp and fundus exam 
f. BCVA 
g. IOP 
h. con meds 
i. adverse events 
j. blood sample for Ab testing 

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7.5.4 Month 3 (Day 90) +/- 4 days 
a. brief medical history and physical exam, including any changes in vision or light 
perception reported by patient or noted by study staff since prior H & PE 
b. vital signs 
c. CBC, platelet, differential, hemoglobin, hematocrit 
d. blood chemistries, not limited to, but must include AP, ALT, AST, BUN, Cr, total 
bilirubin, serum electrolytes, glucose, calcium, phosphate, albumin and total 
protein 
e. urinalysis 
f. slit lamp and fundus exam 
g. BCVA 
h. IOP 
i. con meds 
j. adverse events 
7.5.5 Month 6 (Day 180) +/- 4 days  
a. brief medical history and physical exam, including any changes in vision or light 
perception reported by patient or noted by study staff since prior H & PE 
b. vital signs 
c. CBC, platelet, differential, hemoglobin, hematocrit 
d. blood chemistries, not limited to, but must include AP, ALT, AST, BUN, Cr, total 
bilirubin, serum electrolytes, glucose, calcium, phosphate, albumin and total 
protein 
a. urinalysis 
b. slit lamp and fundus exam 
c. BCVA 
d. AF 
e. B-scan 
f. IOP 
g. VF 
h. contrast sensitivity 
i. VA LV-VFQ-48 
j. mobility test 
k. con meds 
l. adverse events 
7.5.6 Month 9 (Day 270) +/- 4 days  
a. brief medical history and physical exam, including any changes in vision or light 
perception reported by patient or noted by study staff since prior H & PE 

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b. vital signs 
c. slit lamp and fundus exam 
d. BCVA 
e. IOP 
f. con meds 
g. adverse events 
7.6 
End of Treatment (Month 12) or Early Termination Visit  
a. MH and physical exam, including weight and height 
b. vital signs 
c. pregnancy test (if applicable) 
d. CBC, platelet, differential, hemoglobin, hematocrit 
e. blood chemistries, not limited to, but must include AP, ALT, AST, BUN, Cr, total 
bilirubin, serum electrolytes, glucose, calcium, phosphate, albumin and total 
protein 
f. urinalysis 
g. slit lamp and fundus exam  
h. BCVA 
i. B-scan 
j. IOP 
k. OCT 
l. AF 
m. VF  
n. contrast sensitivity 
o. VA LV-VFQ-48 
p. mobility test 
q. con meds 
r. adverse events 
s. blood sample for Ab testing (Test only) 
8.0 
ASSESSMENTS 
8.1 
Safety Assessments 
8.1.1 Adverse Events 
Subjects will be monitored for AEs from the time the subject receives a study number until 
the study end or early termination. Adverse events that occur between clinic visits will be 
elicited by direct, non-leading questioning or will be recorded if offered voluntarily by the 
subject. Further details for AE reporting can be found in Section 9. 

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8.1.2 Vital Signs  
Blood pressure and heart rate, body temperature and respiratory rate will be recorded at 
screening, before and after intravitreal injection and at all follow-up visits. 
8.1.3 Clinical Laboratory Tests 
Blood samples for clinical laboratory tests will be taken as indicated in Table 2. 
Hematology  – full blood count including red blood cell (RBC) count, hemoglobin, 
hematocrit, white blood cell (WBC) count with differential and platelet count; neutrophils, 
lymphocytes, monocytes, eosinophils, basophils. 
Coagulation panel - PT/INR, PTT 
Biochemistry  – alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline 
phosphatase (AP), blood urea nitrogen (BUN), creatinine, total bilirubin, serum electrolytes, 
glucose, calcium, phosphate, albumin and total protein  
Urinalysis – pH, protein, ketones, glucose, bilirubin, blood, urobilinogen, specific gravity by 
dipstick and microscopy if any findings are abnormal. 
Pregnancy A urine sample will be collected for a pregnancy test at screening for female 
subjects of childbearing potential to test for pregnancy. If this is found to be positive, it will 
be followed up with a serum pregnancy test. 
8.1.4 Blood Samples for Antibody Testing 
Blood samples will be collected for testing for Panel Reactive Antibodies (PRA) and Donor 
Reactive Antibodies (DRA) prior to treatment, at 4 weeks post-treatment, and at study 
termination.  Details of preparation/shipping of samples will be provided separately. 
8.1.5 RP Gene Mutation Typing and HLA Typing 
Mutation Typing:  Blood sampling for DNA extraction to determine the RP gene mutation 
may be performed at any study visit. Testing will be performed by a central laboratory.  If a 
subject has previously been typed and a report documenting the type of mutation is available 
or if the subject or parent/legal guardian decline, this test will not be performed.  
HLA Typing: Blood sampling for HLA typing will be done at screening. Testing will be 
performed by a central laboratory.  If a subject has previously been typed and a report 
documenting the subject’s detailed HLA type is available, this test will not be performed. 
Details of preparation/shipping of samples will be provided separately. 
8.1.6 Ophthalmic AEs 
Procedures will be performed at scheduled intervals to specifically assess eye AEs that may 
not be detected by usual AE reporting. Please refer to the study procedure manual for details 
of all ophthalmologic assessment procedures.  

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Slit lamp and fundus exam - The binocular slit-lamp examination provides a stereoscopic 
magnified view of the eye structures in detail, enabling anatomical diagnoses to be made for 
a variety of eye conditions. This assessment will include detection of anterior chamber cell or 
flare; acute cataract development or progression in phakic patients, or vitreous haze. Fundus 
exam allows for inspection of the retina, the cellular graft, and detection of retinal 
detachment. 
B-scan – B-scan is a test that uses high-frequency sound waves to get measurements and 
produce detailed images of the eye. This test may provide information regarding the location 
of the injected cells and the size of the cell clusters at different time points during the study.  
IOP - Intraocular pressure is measured with a tonometer as part of a comprehensive eye 
examination.  Measured values of intraocular pressure are influenced by corneal thickness 
and rigidity 
Spectral Domain Optical Coherence Tomography (OCT) - A clinical OCT machine will 
be used for data collection; the same machine should be used for any given patient at each 
time point.  OCT tests will be analyzed to help define retinal anatomy (AOSLO), and will 
enable monitoring for ocular inflammation after treatment as manifest by cystoid macular 
edema or epiretinal membrane formation or progression. 
8.2 
Efficacy Assessments  
Please refer to the study procedure manual for details of all ophthalmologic assessment 
procedures.  
8.2.1 BCVA (E-ETDRS or FrACT) 
BCVA will be tested at scheduled time points in both the treated and the contralateral eye. 
Visual acuity will be measured with the electronic visual acuity testing algorithm (E-ETDRS) 
for eyes with vision better than 20/800, including all study eyes at baseline.  The Freiburg 
Visual Acuity & Contrast Test (FrACT) using Landolt C’s has a broader range for testing 
and should be used for eyes worse than 20/800.  For any individual eye, the same method 
must be used throughout the study for consistency unless the eye has deteriorated to the point 
where E-ETDRS can no longer be administered. 
Trial frame refraction is the gold standard in obtaining the most accurate refractive error in 
low vision patients.  It involves the use of a trial frame, loose lenses and specialized low 
vision eye charts that are different from the eye charts used in a regular eye examination.  
These special low vision eye charts contain different-sized letters or numbers that can help 
determine the sharpness or clarity of the subject’s distance vision.  Performed properly, the 
examiner obtains not only the refractive error, but additional potentially essential 
information, such as the level and quality of visual acuity, sensitivity to blur, effects of glare 
and the quality of fixation. The optical theory involved is the same as when refracting the 
normal eye, but special adjustments in lens selection, presentation strategy, and “just 

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noticeable difference”, are incorporated for low vision patients.  These adjustments usually 
include large lens increments and special techniques for exploring and refining cylinders.   
The Freiburg Visual Acuity & Contrast Test (FrACT) assesses visual acuity and contrast 
sensitivity. It is a free computer program that uses computer graphics capabilities and 
psychometric methods to assess an individual’s visual acuity in a greater range than 
traditional ETDRS testing.  
8.2.2 Visual Field Testing 
Subjects enrolled in the study may have a severely restricted visual field (e.g., 4 degrees in 
diameter) with or without eccentric islands in the mid to far-periphery in advanced RP, or 
they may have full visual fields but reduced photopic sensitivity in earlier stages of RP.  
Goldmann or kinetic visual field testing is the preferred test to obtain an isopter with an area 
of remaining field(s) of vision from RP using a specified target of V4e for more severe 
subjects and a target of III4e for better seeing subjects. Target size/brightness will be selected 
based on baseline visit for each eye separately. Whatever target size is selected, the same size 
will be used throughout the study on that particular eye.   
8.2.3 Autoflourescence 
Fundus autofluorescence images specifically assess the health of the retinal photoreceptors 
(RODS and CONES) and the retinal layer which provides nutrition to the rods and cones (the 
retinal pigmented epithelium - RPE).   FAF is effective because it can document metabolic 
change from the accumulation of toxic fluorophores in the retinal pigment epithelium.  The 
test may require pupil dilation but does not require injection of any dye. 
8.2.4 Other Visual Assessments 
Mobility testing 
Mobility testing has been performed with different maze configurations, e.g., Spark 
Therapeutics Maze (Maguire et al 2008) and Graz Mobility test (Velikay-Parel et al 2007)].  
A similar maze has been constructed and will be assessed for validity in a subset of subjects 
(dependent upon timing of availability). In general, the mobility test will consist of differing 
but structurally similar configurations of a relatively short maze (e.g. arrows on a printed 
floor mat) with ten obstacles, of various sizes. Subject testing consists of walking through 
several configurations each at a different illumination level as a test of mesopic visual 
function. Each test is videotaped and scored for speed and accuracy (number of errors) by 
independent trained graders and a score, the critical illumination level, is derived that 
represents a significant drop in functional performance (slow walking speed and increase in 
errors). The critical illumination level for each eye is compared over time.  
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A contrast sensitivity test measures the ability of a subject to distinguish between finer and 
finer increments of light versus dark (contrast).  Contrast provides critical information about 
edges, borders, and variations in luminance. Contrast sensitivity is correlated to performance 
on many real world tasks and tests that generate a curve or multiple sensitivity thresholds at 
varying grating sizes (spatial frequency) provide an in-depth view of functional vision.  
Multiple thresholds and varying spatial frequencies will be measured monocularly and 
compared over time. In more severely impaired subjects, only the lower spatial frequencies 
will be able to be tested, while in mild to moderately impaired subjects, several spatial 
frequencies can be assessed resulting in a contrast sensitivity curve. Each spatial frequency is 
tested up to four times (thresholds) so the mean threshold at the peak contrast sensitivity for 
each eye will be compared over time.  
Activities of Daily Living (ADL) 
The Low Vision Visual Functional Questionnaire (LV-VFQ48) is a valid and reliable 
questionnaire that may be administered by telephone to capture changes in patients' self-
report of their difficulty reading and performing other daily living activities affected by 
visual impairment before and after rehabilitation.  In our case, the intervening 
“rehabilitation” would instead be the hRPC treatment. Total instrument scores and separate 
scale scores will be compared over time. 
9.0 
SAFETY CONSIDERATIONS 
9.1 
Adverse Events 
An AE is defined for this study as any untoward medical occurrence in a subject who is 
administered clinical study material. The occurrence of this event does not necessarily have a 
causal relationship with study product.  An AE can therefore be any unfavorable and 
unintended sign (including an abnormal laboratory finding), symptom, or disease temporally 
associated with the use of a study (investigational) product or treatment (e.g. mock injection), 
whether or not related to the study product or treatment. 
Examples of an AE include: 
• Exacerbation of a chronic or intermittent pre-existing condition, including either an 
increase in frequency or intensity of the condition 
• Significant or unexpected worsening or exacerbation of the condition/indication 
under study (RP) 
• A new condition detected or diagnosed after study product administration even 
though it may have been present prior to the start of the study 
• Signs, symptoms, or clinical sequelae of a suspected overdose of either study product 
or a concurrent medication 
• Pre- or post-treatment events that occur as a result of protocol-mandated procedures 

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• Antibody development 
An AE does not include: 
• Medical or surgical procedures (e.g., colonoscopy or biopsy); the medical condition 
that leads to the procedure is an AE 
• Social or convenience hospital admissions where an untoward medical occurrence did 
not occur 
• Day-to-day fluctuations of a pre-existing disease or conditions present or detected at 
the start of the study that do not worsen 
• The disease/disorder being studied (RP), or expected progression, signs, or symptoms 
of the disease/disorder being studied unless more severe than expected for the 
subject’s condition 
All AEs that occur after informed consent is signed will be recorded in the source documents 
and on the appropriate CRF page. The information to be collected includes the nature, date 
and time of onset, intensity (mild, moderate, severe, life-threatening, death), duration, 
causality (relationship to investigational product), and outcome of the event. Even if the 
AE/SAE is assessed by the Investigator as not reasonably attributable to study product, its 
occurrence must be recorded in the source documents and on the appropriate page of the 
CRF. 
Treatment-emergent AEs will be defined as AEs that occur after the study treatment 
(injection or mock injection).  
9.2 
Serious and Unexpected Adverse Events 
A serious adverse event is one which:  
 
•    is fatal or life threatening, 
• 
is permanently or significantly disabling, or 
• 
requires in-patient hospitalization or prolongation of hospitalization 
An unexpected adverse experience is one which: 
• 
is not previously reported with the agents or procedures being undertaken, or 
• 
is symptomatically and pathophysiologically related to a reported toxicity but 
differs because of greater severity or increased frequency. 
Important medical events that may not result in death, be life-threatening, or require 
hospitalization may be considered an SAE when, based upon appropriate medical judgment, 
they may jeopardize the subject and may require medical or surgical intervention to prevent 
one of the outcomes listed in the above definition. 

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The Investigator must report all SAEs and all deaths to the study sponsor or designee 
immediately by telephone and in writing within five (5) days.  A toll free number and an 
SAE reporting form to report such events will be provided.  See also section 11 with respect 
to administrative responsibilities. 
10.0 
STATISTICAL CONSIDERATIONS 
10.1 
Study Population 
The intent-to-treat population comprises all subjects who enroll in the study and who provide 
any post-screening data. The safety population comprises all subjects who receive any study 
treatment. 
10.2 
Efficacy Analyses 
10.2.1 Primary Efficacy Endpoint and Analysis 
The primary efficacy endpoint will be the 12-month mean change in the study eye of the best 
corrected visual acuity (BCVA), to be measured with the electronic visual acuity testing 
algorithm (E-ETDRS).  
The eligible study subjects will be randomized 1:1:1 to either treatment with 3 x 106 jCell, 6 
x 106 jCell or to sham treatment (mock injection).  The primary analyses of the primary 
endpoint will be based on two pairwise comparisons of the mean change at 12 months in 
BCVA for the study eyes between each experimental j-Cell group and the Control group. 
Assume the standard deviation for the 12-month change in BCVA is 5 letters.  Then, for each 
pairwise comparison with the control group, a randomized trial with 25 patients per arm will 
have 90% power to detect a true treatment effect of a mean of 5 letters, when using a t-test 
having (one-sided) 0.0125 false positive error rate.  It is planned to randomize 85 subjects in 
the trial, in order to address a low rate of irregularities in the quality of trial conduct. 
10.2.2 Secondary Efficacy Endpoints and Analyses 
For assessment of mobility, similar methods will be used as have been reported for the 
RPE65 studies.  Briefly, subjects who are capable of maze navigation will be evaluated for 
their performance in navigating a mobility course under a variety of specified light levels 
ranging from 0.1 lux (dimmer than a moonless summer night) to 500 lux (a brightly lit 
office) using bilateral testing condition. Each attempt is videotaped and scored for speed and 
accuracy (number of errors) by independent trained graders and a score, the critical 
illumination level, is derived that represents the level at which a significant drop in functional 
performance is observed (slow walking speed and increase in errors). Other visual test results 
(peak contrast sensitivity, visual field area) will be compared between the 3 treatment groups 
using analysis of covariance (ANCOVA) method with baseline values as covariates. Subjects 
who cannot perform a specific visual test at baseline (e.g. mobility) will be considered non-
evaluable for analysis of that assessment.   

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Similar analytical methods will be used for QOL measures. The total and individual scores 
from VA LV VFQ-48 will be analyzed using linear mixed model, which is designed for 
longitudinal study with correlated measurements. 
The Pearson Chi-square test with alpha levels adjusted for multiplicity issues will be applied 
to evaluate the proportion of subjects who show specific improvement compared to the 
proportion of control subjects in any secondary endpoints at the pre-specified time of primary 
analysis. 
10.3 
Safety Analyses 
Adverse events will be monitored by the investigator and the subject. The safety analyses of 
AEs and laboratory parameters will include descriptive statistics by treatment group.  
Summaries of AEs will be generated by treatment group, type (AE or SAE), body system and 
preferred term, severity, and relationship to study product.    
10.4 
Other Analyses 
Demographics:  Background and demographic data will be summarized for all subjects by 
treatment assignment.   
11.0 
ADMINISTRATIVE CONSIDERATIONS 
11.1 
Adverse Experiences  
All adverse experiences (AE) must be recorded and reported to the sponsor.  Any serious and 
unexpected AE will be reported to the sponsor or designee immediately by telephone and 
subsequently in writing within five (5) days.  The Investigator must also notify the 
institutional IRB/EC. A full report, including clear photocopies of hospital records, 
consultants' reports, autopsy findings where appropriate, and a summary of the outcome by 
the Investigator, including his opinion of study relationship or attribution, will be furnished 
to the study Sponsor or designee as soon as practicable.  It is the sponsor’s responsibility to 
notify the FDA, other regulatory agencies as appropriate, and all clinical sites in compliance 
with regulatory requirements. 
11.2 
Institutional Review  
Prior to implementation of this study, the research protocol and the proposed subject consent 
form must be reviewed and approved by a properly constituted Institutional Review 
Committee operating under the Code of Federal Regulations (21CFR Part 56).  A signed and 
dated statement that they have approved the protocol must be submitted to the sponsor prior 
to the start of the study.  This committee must also approve all amendments to the protocol. 
11.3 
Informed Consent  
Informed consent will be obtained via discussions with the subject, explaining the rationale 
and experimental nature of the system, the duration of the trial, alternate modes of treatment, 

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and prevalent adverse reactions that might occur.  Each subject will receive a copy of the 
signed consent form. 
At the time of the discussions relating to enrollment or at any time during participation in the 
protocol that new information becomes available relating to risks, adverse events, or 
toxicities, this information will be provided orally or in writing to all enrolled or prospective 
subject participants in a timely fashion.  Documentation of communication will be provided 
to the local institutional review board. 
11.4 
Monitoring Procedure  
At the time the study is initiated, the principal monitor and/or co-monitors will thoroughly 
review the protocol and case report forms with the Investigators and their staff.  During the 
course of the study, the principal monitor, the co-monitor or their designated deputies shall 
be available to discuss by telephone or other means, questions regarding adverse reactions, 
removal of subjects from trial, conduct of the study, etc. 
At the time of each monitoring visit, the monitor will review the case report forms of each 
subject in the trial to make certain that data is reported in a timely fashion, that all items have 
been completed and that the data provided are accurate and obtained in the manner specified 
in the protocol.  The subjects' clinical records will be reviewed to confirm that the case report 
form data are consistent with the physician's clinical records.  The monitor will verify the 
adherence to the procedures and schedule as defined in the protocol.  The subject's clinical 
records will be reviewed to determine whether recording of adverse reactions or side effects 
has been omitted on the case report forms.  If this is found to be so, then the case report 
forms will be returned to the Investigator and corrected to include this information. 
At the time of the monitoring visit, it is the responsibility of the participating site to provide 
completed up-to-date case report forms, and to provide ready access to source documents. 
11.5 
Reporting and Recording of Data  
Records -  In the US, federal regulations require that copies of case report forms be retained 
by the Investigator for a period of no less than two (2) years following either the approval of 
Biological License Application or the withdrawal of the Investigational New Drug 
Application.  The Sponsor will advise the Investigator when the two-year period begins.  
Attention of the Investigator is drawn to the fact that he/she may be subject to a field audit by 
FDA or other regulatory inspectors to verify that the study is conducted in accordance with 
the requirements of the protocol, as well as in compliance with Good Clinical Practices. 
Reporting of Data - All information required by the protocol is to be provided, or an 
explanation given for omissions.  A monitor will verify the validity and completeness of the 
forms at each monitoring visit. 

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All data and information on the case report forms are to be neatly recorded in type or legibly 
printed in black ink for ease of duplication, interpretation and analysis before submission to 
the Sponsor designee.  All corrections on the case report forms should be crossed out neatly 
and the new entry initialled and dated by the member of the Investigator's staff making the 
correction.  Prior to forwarding the final case report forms, they should be reviewed for 
completeness, accuracy and legibility by the Investigator. 
Copies of the completed case report forms will be provided by the Sponsor for retention by 
the Investigator. 
11.6 
Changes in Protocol  
There will be no alterations or changes in this protocol without the written consent of the 
sponsor, jCyte.  
11.7 
Investigational Product and Label Codes  
11.7.1 Investigational Drug  
For this study, frozen vials of jCell (hRPC) are provided to the qualified dose preparation 
facility by 
, where the cells were 
manufactured and are currently cryopreserved.  
11.7.2 hRPC Reconstitution (Thaw and Culture) and Administration 
When a study subjects has been scheduled for treatment, the qualified dose preparation site 
will prepare the patient dose according to a standard procedure.  For subjects randomized to a 
Test arm,  frozen cells are thawed and cultured 40-48 hours prior to injection.  Following 
successful culture and testing, 65 µl of cell suspension containing 3 million or 6 million 
human retinal progenitor cells suspended 
 will be 
provided by the dose preparation facility to the clinical site, on ice, with targeted 
administration to the patient within 4 hours of cell harvest. Patients will undergo topical 
anesthesia prior to injection of the cells.  
11.8 
Product Security  
In accordance with Federal and other regulations governing investigational materials, the 
Investigator agrees to keep investigational material in a secure location and to carefully 
control and document its use. The Investigator agrees to document use of investigational 
products as instructed in the labelling and disposal of empty vials, and to return any unused 
investigational product to the Sponsor or its designee. 

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References  
                                                 
1 http://www.blindness.org/index.php?option=com_content&id=50&Itemid=67 
2 Wenkel H, Chen PW, Ksander BR, and Streilein JW. (1999) Immune privilege is extended, 
then withdrawn, from allogeneic tumor cell grafts placed in the subretinal space. Invest 
Ophthalmol Vis Sci 40:3202-3208. 
3 Silverman MS and Hughes SE.  (1989) Transplantation of photoreceptors to light-damaged 
retina. Invest Ophthalmol Vis Sci 30:1684-1690. 
4 Li LX and Turner JE. (1988) Inherited retinal dystrophy in the RCS rat: prevention of 
photoreceptor degeneration by pigment epithelial cell transplantation. Exp Eye Res 47:911-
917. 
5 Lopez R, Gouras P, Kjeldbye H, et al. (1989) Transplanted retinal pigment epithelium 
modifies the retinal degeneration in the RCS rat. Invest Ophthalmol Vis Sci 30:586-588. 
6 Zhang Y, Kardaszewska AK, van Veen T, Rauch U and Perez MT. (2004) Integration 
between abutting retinas: role of glial structures and associated molecules at the interface. 
Invest Ophthalmol Vis Sci 45:4440-4449. 
7 Silver J and Miller JH. (2004) Regeneration beyond the glial scar. Nat Rev Neurosci 5:146-
156. 
8 David S and Lacroix S (2003) Molecular approaches to spinal cord repair. Annu Rev 
Neurosci 26:411-440. 
9 Young MJ, Ray J, Whiteley SJ, Klassen H and Gage FH. (2000) Neuronal differentiation 
and morphological integration of hippocampal progenitor cells transplanted to the retina of 
immature and mature dystrophic rats. Mol Cell Neurosci 16:197-205. 
10 Klassen HJ, Ng TF, Kurimoto Y et al. (2004) Multipotent retinal progenitors express 
developmental markers, differentiate into retinal neurons, and preserve light-mediated 
behavior.  Invest Opthalmol & Vis Sci.45 (11) 4167 – 4173. 
11 Takahashi M, Palmer TD, Takahashi J and Gage FH. (1998) Widespread integration and 
survival of adult-derived neural progenitor cells in the developing optic retina. Mol Cell 
Neurosci 12:340-348. 
12 Klassen H, Aiaeian B, Kirov II, Young MJ and Schwartz PH.  (2004) Isolation of retinal 
progenitor cells from post-mortem human tissue and comparison with autologous brain 
progenitors.  J Neurosci Res. 77(3): 334-343. 
13 Klassen H, Imfeld KL, Ray J, et al. (2003) The immunological properties of adult 
hippocampal progenitor cells. Vision Res 43:947-956. 

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14 Hori J, Ng TF, Shatos M, Klassen H, et al. (2003) Neural progenitor cells lack 
immunogenicity and resist destruction as allografts. Stem Cells 21:405-416.