synopsis
appendix i flow schematic for

*Comprised of post-transplant assessments and post-transplant weekly visits through Day +100, intermittent visits through Day +365 and a telephone call on Day +730.aGvHD = Acute graft-versus-host disease; CMV = Cytomegalovirus; EAC = Endpoint Adjudication Committee; ECG = Electrocardiogram; GvHD = Graft-versus-host disease; HLA = Human Leukocyte Antigen; HCT = Hematopoietic cell transplant; IDMC = Independent Data Monitoring Committee; mPB = Mobilized peripheral blood.  
| An IDMC will convene to assess preliminary safety data  as outlined in the IDMC Charter. At that time, the  Sponsor, in consultation with and based on  recommendations from the IDMC, will decide whether the  study should stop or whether the Phase 2 study can begin.   |                                                                                                                               |                                                                                                         |                               |          |           |
|-----------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|-------------------------------------------------------------------------------------------------------------------------------|---------------------------------------------------------------------------------------------------------|-------------------------------|----------|-----------|
| One unit of  ProTmune  ex vivo  programmed  mPB cells as  the cell  source for the  HCT  procedure.                                                                                                                                                                         |                                                                                                                               |                                                                                                         |                               |          |           |
| Conditioning Regimen per  protocol. GvHD Prophylaxis Regimen per  protocol.                                                                                                                                                                                                 | The study will utilize an EAC to adjudicate the aGvHD  endpoint at Day +100 and Day +180 in accordance with  the EAC charter. |                                                                                                         |                               |          |           |
| Day -53                                                                                                                                                                                                                                                                     | Day -10                                                                                                                       | Day 0                                                                                                   | Post-Transplant  Assessments* | Day +365 | Day +730* |
| Conditioning Period (Day -10 to  Day -1; up to 9 days  prior to Hospital  Admission)                                                                                                                                                                                        | Hospital  Admission (Institutional  Policy)                                                                                   | Key Assessments: - Adverse event monitoring.  - Karnofsky Performance Status. - Acute GvHD assessments. |                               |          |           |
| Screening  Period  (Day -53 to                                                                                                                                                                                                                                              |                                                                                                                               |                                                                                                         |                               |          |           |
| Day -11; up to 6 weeks  prior to the  Conditioning Period)                                                                                                                                                                                                                  | Day of  HCT                                                                                                                   | 2-Year Follow- up                                                                                       |                               |          |           |
| Post-Transplant                                                                                                                                                                                                                                                             | Study Completion                                                                                                              |                                                                                                         |                               |          |           |
| Follow-up                                                                                                                                                                                                                                                                   |                                                                                                                               |                                                                                                         |                               |          |           |
| Key Assessments:                                                                                                                                                                                                                                                            |                                                                                                                               |                                                                                                         |                               |          |           |  
Hospital  
Admission  
(Institutional  
Policy) Key Assessments:  
- Adverse event monitoring. - Karnofsky Performance Status.  
- Acute GvHD assessments. - Chronic GvHD assessments.  
- Peripheral blood monitoring for safety panels, viral  
monitoring, immune reconstitution, chimerism, serum immunoglobulins, and GvHD exploratory  
biomarkers.  
- Bone marrow aspirate.  
- ECG.  
Key Assessments:
- Investigator is required to verify that the subject completed HLA typing.  
- Infectious disease titers and CMV
viremia.  
- Peripheral blood baseline sample for immune reconstitution assays.  
Key Assessments:
- ECG and safety panels on Day 0 prior to HCT.  
- Adverse event monitoring (starting at Conditioning).  
- Karnofsky Performance Status.  
APPENDIX J: CLINICAL LABORATORY ANALYTES Hematology (CBC with differential): Red blood cells Hemoglobin Hematocrit Platelets White blood cells Neutrophils Bands Lymphocytes Monocytes Manual differential includes: Blasts ANC calculation Serum chemistry: Creatinine ALT AST Alkaline phosphatase Blood urea nitrogen Lactate dehydrogenase Sodium Potassium Chloride CO2 Bilirubin (total and direct) Magnesium Calcium Phosphorus