R＆D Meeting 2022
Growth Strategies of Cell Therapy(Regenerative Medicine) Business
November 9, 2022

Kidswell Bio Corporation

Agenda
1. Opening Remarks• Pipelines in Cell Therapy (Regenerative Medicine) Business

Masaharu Tani
President & CEO

2. Our strategies of SHED Platform• Intellectual Property Strategies
Joint patent application on cell therapy for cerebral palsy utilizing
SHED with Tokai National Higher Education and Research System(Nagoya University)•2nd Generation SHED
Collaborative research with Hamamatsu University School of Medicine- Potential application for brain cancer-

Masayuki Kawakami
COOYasuyuki Mitani
Director, Business
Development Dept., Business
Development Div.,Regenerative Medicine UnitLeader

3. Market Potential of SHED
• Potential Market size of Regenerative Medicine Products• Market Potential of SHED- Nervous System and Muscle/ Bone Diseases, Brain Cancer-

Masaharu Tani
President & CEO ）4. Concluding Remarks• Our Vision

Masaharu Tani
President & CEO2

1. Opening Remarks

Pipelines in Cell Therapy (Regenerative Medicine)
Development
ProductTherapeutictargetPartners

Number of patients(Domestic) ※2

Number of patients(Global) ※2

NoneNerve protection,
activation andregeneration

Nagoya University, Tokyo
Medical and Dental University

2,000 patients per
year,30,000 patients in
total

100,000 patients per
year, 1.7 millions
patients in total

Enterectomy,colostomy

Ganglion
regeneration

Mochida Pharmaceutical

100 patientsー

Loss of motor
function andsensation

None

Nerve protection,activation andregeneration

Nagoya University

5,000 patients per
year,100,000 patients in
total

25,000 patients per
year, 500,000
patients in total (US,EU and Japan)

Non-union fractures

Chronic pain, gait
disturbance

SurgeryBoneregeneration

Hokkaido University and Spinal
Injuries Center

100,000 patients peryearーOphthalmologic
disease

※1

※1

※1

Gifu Pharmaceutical University※1※1

Motor function andsensation disorder

Nerve
reconstruction(Autologousnervetransplantation)

Peripheral nerve
regeneration

Oita University

8,000 surgeries peryearーEating and speechdisorderLip arthroplasty+ iliac bone graft

Maxilla boneregeneration

ORTHOREBIRTH

2,000 patients peryear

15 out of 10,000
newborns

Brain cancer

Poor life prognosis

Surgery,
radiation therapy,
chemotherapy

Anticancer,
prevention ofrecurrence

Hamamatsu University Schoolof Medicine

20,000 patients peryear

830,000 patients intotal※1※1※1※1

NanoCarrier, BioMimetics
Sympathies※1※1

Target disease
Pediatric disease

Cerebral palsy

Pediatric disease

Congenital Isolated
Hypoganglionosis
Including Pediatricdisease

Spinal cord injury
1st generation
SHED

Peripheral nerve palsy

Pediatric disease

Cleft lip and palate

Symptom

Quadriplegia andPosture disorder

Intestinalobstruction

2nd generation
SHED

Existing
Treatment※1 Details not disclosed, ※2 KWB research based on public and relevant information

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2. Our Strategies of SHEDPlatform

Intellectual Property Strategy

6

Strengthen Interictal Property Strategies
Accelerate and maximize the growth by combining R&D, Business
Development and Intellectual Property strategies
Business Development

R&D• Proceed R&D strategies based onanalysis of information on the priorpatents• Proceed R&D and intellectual
property strategies based on thepresent state of targeted disease
and issues.

ManagementStrategies• Business collaboration, own patent
license for licensing-out activities andmutual use of patent• Joint patent applications withacademias and development partners

Intellectual Property

Proceed intellectual property strategies effectively combined withR&D and business development strategies• Proceed strategic patent applications and life cycle management of the patents.
• Drive patent applications in USA, Europe and Asia as well as Japan Apply for a substance (cell) patent for differentiating SHED from other cells.
 Apply for a use patent for securing rights of treatment methods of targeted diseases.
 Apply for patents of manufacturing and administration methods for extension of patentterms.
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Joint Patent Application
October 24, 2022

Some research results in the filed patent will be announced atJapan Society for Neonatal Health and Development(Nov. 24 to 26, 2022)

8

Strengthen Interictal Property Strategies
Combined effects of R&D, Business Development andIntellectual Property
BusinessDevelopment

R&D
ManagementStrategies

IntellectualProperty
Proceed intellectual property strategies effectively combined withR&D and business development strategies

92nd Generation SHED10

2nd Generation SHED Development
Launched development of designer cells as reinforced SHEDs to achieve high medicaltreatment goals
Target diseases ofdevelopment※

Development of new gene transfer methods（Ex: NanoCarrier）

Development of new culture methods

Cancer, etc.

ReinforcedSHED(Ex: BioMimetics Sympathies Inc.）

Verification of the effects of various types oftransgenes

Genetic diseases
Degenerative (Nerve)※ in addition to nervous andmuscle/bone diseases in the list oftarget diseases of 1st generation SHED

(Ex: Hamamatsu University School of Medicine,Nagoya Univ., ）Aim to develop reinforced SHED with enhanced therapeutic efficacy
Infection

Lifestyle related

Degenerative（Nerve・Organ）

Autoimmunity

Cancer

Genetic diseases

Pediatric diseases

2nd Generation “Designer Cells”

PermanentCure Treatment

Vaccine(Prevention・Treatment)

1st Generation CellTherapy

Recombinant Cell Therapy (Stem Cell-Based)
Add/Enhance Functions by Genetic Engineering

Non-Recombinant Type ofStem Cells etc.

SymptomaticTreatment

Small, Mid
Molecule Drug

DTxAntibody Drug

NucleicAcidMedicine* Created by Kidswell Bio Corporation referring from Strategic Proposal of Designer Cell by Japan Science and Technology Agency. 11

2nd Generation SHED －Potential application for brain cancer－Brain Cancer•There are primary or metastatic brain cancer. Most
common brain cancer is primary gliomas.

•Low survival rate: Less than 10% of 5-year survival
rate for primary gliomas.

•

Metastatic brain cancer is about 15% of all type ofbrain cancer, half of which is lung cancer brain
metastases.

•Low survival rate: Less than 10 to 30% of 5-year
survival rate for metastatic brain cancer.

Primary Brain Cancer（WHO Grade I-IV）
Glioma 25.2%
All Other 21.2%

Nerve Sheath 10.1%

Pituitary 19.3%

Meningioma 24.4%
Brain tumor registry in Japan 2001-2004

5-year survival rate by type of brain cancer

Primary

MetastaticJpn J Neurosurg (Tokyo) 24: 699-704, 201512

Collaborative Research with Hamamatsu Univ. School of Medicine
Concept of the potential therapeutic application of the next generation SHED for brain cancer①Administration of the SHED
engineered to express an enzyme,
thymidine kinase (TK) to brain

Oishi et al., Mol Ther Methods Clin Dev. 2022

②Confirmed the migratory ability of SHED forglioma cells.
③Systemic administration of ganciclovir(antiviral drug＝GCV )④GCV converted into a toxicant by TK,
and permeated cancer cells
adjacent to SHEDs⑤ Toxicants were permeated cancer cells andcancer cells disappeared one after another(＝bystander effect)

Cancer cells + SHED-TKCancer cells + SHED-TK + GCV

13Collaborative Research with Hamamatsu Univ. School of Medicine

SHED-TK effect in a mouse model of brain cancer

Administered SHED-TK to the opposite side of the brain, where the glioma
implanted, and SHED-TK migrated and accumulated toward the glioma.

No mice were dead in theSHED-TK/GCV group due toextinction of glioma cells.

No mice of lung cancer brain metastases were dead in theSHED-TK/GCV group due to extinction of glioma cells.

Cancer Gene Ther. 2022

Drive further R&D for clinical applications

Mol Ther Methods Clin Dev. 2022

143. Market Potential of SHEDPotential Market size in Regenerative Medicine Products

The Market Size of Regenerative Medicine Products in 2050
Japan: 2.5 trillion yen、 Worldwide:38 trillion yen

Potential Market Size Forecast in Regenerative
Medicine Products (Global)

Potential Market Size Forecast in Regenerative
Medicine Products (Japan)
2012

2020

2030

2050

9 bn

95 bn

1.0 tn

2.5 tnBillion yen

2.5 tn in2050

Others (Hairregeneration)¥

2012

2020

2030

2050

100 bn

1.0 tn

12 tn

38 tn

Trillion yen

38 tnin 2050

Cancerimmunity

1.0 tn in2030

BloodSpleen(Diabetes)

12 tnin 2030

Liver95 bn in2020

Othersincludingdevelopingcountries

JapanKidney
Blood Vessel
Alveolar bone

Europe

1.0 tn in2020

NerveHeartOphthalmus

USACartilage,Skin

2012

2020

2030

2040

2050

Source:METI 「第１回 再生医療・遺伝子治療の産業化に向けた基盤技術開発事業複数課題 プログラム 中間（終了時）評価検討会」 資料⑥
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Market Potential
Nervous System and Muscle/ Bone
Diseases: 1st Generation SHED

Brain Cancer:2nd Generation SHED

Grow from 700 billion to 800-billion-yen market by
2040.

25,000Respiratory
Metabolic Disorders
20,000• Grow more than 2-trillion-yen market by 2027*3• Genetically modified cell medicine (designercells) is expected additional 350-billion-yen
market*4M$)

Immunology

20,000Genetic Disorders
Gastrointestinal
Dermatology
Central Nervous System

15,000

Cardiovascular

15,000*Including Japan, the U.S.
Europe, China and India in
global market (Targeted 10%of the population in China andIndia）
10,00010,000

5,000

5,000

02022 2023 2024 2025 2026 2027 2028 2029 2030 2031 2032 2033 2034 2035 2036 2037 2038 2039 2040 （年）Estimation for market size of allogeneic stem cells*1*1 KWB’s research based on Regenerative medicine and gene therapy market research in 2019 Finalreport from the website of Japan Agency for Medical Research and Development, Global Data fromepidemiological literature and websites from rare diseases (Orphanet, NORD, Japan Intractable Diseases
Information Center, Clinical Development Success Rates 2006-2015, BIO Industry Analysis」 and related
documents from general meeting of Central Social Insurance Medical Council

2021

2022(F)

2023(F)

2024(F)

2025(F)

2026(F)

2027(F) （year）

Sales forecast on potential applicable products forbrain cancer *2（Including all modalities, worldwide total, sales except from brain cancer）*2: Global Data LLC（ Figures for 2022 and beyond are forecasts (F)）*3: 1USD=145円*4: Proportion of brain cancer among new cancer patients 1.4%（CA Cancer J Clin 2015;65:5–29), Assumingly calculated a 10% share of the cell medicine modality （KWB’s forecast）

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4. Concluding Remarks

Our Vision

All for Kids, Kids for All

KIDS WELL, ALL WELL
～ For Comprehensive Healthcare System forChildren as well as Families, and Society ～Focus AreaBusinessDirection

Pediatric diseases
including juvenile diseases,
intractable & rare disease

Targeting diseases which
has less accessibility forsufficient medicaltreatment

All for Kids, Kids for All• Under the declining birthrate and aging population, reducing theburden on children is a major social issue.

Children• Provide new pharmaceuticals and therapeutics to patients sufferingfrom diseases and contribute to the realization of a society wherechildren and adults who support children live happily and brightly.
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All for Kids, Kids for All

Cautionary Statement
This information material is provided for understanding Kidswell Bio
Corporation (“KWB”), not for soliciting investment in KWB shares.

Information provided in this material may contain so-called “forward-lookingstatements.” These statements are based on current expectations, forecasts
and assumptions that are subject to risks and uncertainties, which could cause
actual outcomes and results to differ materially from these statements. Risks
and uncertainties include success rate of R&D projects, new regulations andrules, relations with partners in the future, etc.

This material includes information on pharmaceutical products andregenerative medicine (or related products), etc., which is being developed orlaunched. However, this is not intended to promote our products or provide
medical advices.
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