Brief Overview
Date of the Meeting:Thursday, April 25th, 2024

TOPICS
Update on Regenerative Medicine from eachParticipating Industrial AssociationVenue (or Zoom):

We'll share the latest updates on regulations and trends
in regenerative medicine across Asia.

TOKYO MIDTOWN YAESUCONFERENCE

Points to Consider for Cellular Therapy CMC
toward New Drug Application (NDA)(Please note the change in venue)
4F Main Conference Room
2-2-1 Yaesu, Chuo-ku, Tokyo 104-0028
https://www.yaesu.tokyomidtown.com/en/conference
Past Participants:
Presenter/DiscussantIndustrial Association: China, India, Japan,Korea, Singapore, Taiwan
Regulatory Authority and Agency: India,Japan, Korea, Singapore, Taiwan

ObserverRegulatory Authority: Indonesia, Malaysia,Myanmar, Philippines, Thailand, Vietnam

One of the key sessions at theAPACRM is the discussion
featuring panelists invited from
regulatory authorities andagencies, as well as facilitators
who are APACRM Working
Group (WG) members. We
would greatly appreciate it if
you could register for thisconference via the QR code or
the link provided below.
Registration Form:[English site][Japanese site]

Thissection
willoutlineconsiderations for
manufacturing cellular and tissue-based products,drawing from extensive commercial manufacturingexperience.

Considerations for Conducting Clinical Trials ofRegenerative Medicinal Products
We aim to identify key issues related to clinical trials ofcellular products within regenerative medicine,consolidating cases and concepts from Asian
countries/regions for discussion at the next APACRM.

Considerations for Preparing an NDA for Gene
Therapies
Building upon last year's efforts to identify necessary
data for issuing an Investigational New Drug (IND) for
Adeno-Associated Virus (AAV), this year we will focus
on delineating the data requirements for preparing
NDAs for gene therapies, including CAR-T and AAV. We
will also explore differing perspectives among countries
and regions, aiming to engage in discussions withregulatory authorities and industry stakeholders.

Non-clinical data package for AAV-based Gene
Therapies
Given the recent advancements in the clinicaldevelopment of AAV, we seek to outline the necessary
non-clinical data package required for submitting anIND. This year, we will begin by elucidating Japan'sperspective, facilitating future discussions with othercountries and regions.

Compilation of Case Studies on Cell and Gene
Technology provided to the Patients as Medical
Care or Medical Practice without MarketAuthorization
With 10 years having passed since the implementationof the Act of Safety Regenerative Medicine in Japan, andrevisions scheduled for this year, we will provide insights
into the current situation and showcase examples of howunapproved regenerative medicine is being provided in
Asian countries such as Taiwan.

Agenda for APACRM 2024 (7th APACRM)
Brief Overview of APACRM 2024
Date of the Meeting: Thursday, April 25th, 2024
Venue (or Zoom): TOKYO MIDTOWN YAESU CONFERENCE (Please note the change in venue)
https://www.yaesu.tokyo-midtown.com/en/conferenceTime (JST)Duration

Session

Chair/Speaker/Panelist

13:00-13:03

3 minOpening remarks

Yoshitsugu Shitaka, FIRM

13:03-13:10

7 min

Introduction

Masayuki (Max) Nomura, FIRM

Update on Regenerative Medicine from each Participating Industrial Association

13:10-14:10

60 min10-min presentations from eachcountry/region (Pre-recorded)* on site presentation

Takayuki Nakano, FIRM (Japan)
Eugene, J. WANG, CMBA (China)
Bryan Choi, CARM & RMAF (Korea)
Pawan Kumar Gupta, ABLE (India)
Chia-Ning Shen, BPIPO (Taiwan)
Srinivasan N Kellathur, SAPI (Singapore)*

Educational Lecture
14:10-14:40

30 minPoints to Consider for Cellular Therapy
CMC toward New Drug Application “NDA”

Yukio Mori, FIRM/J-TEC

Topics for future activities
14:40-15:10
15:10-15:20

15:20-16:40(80 min)30 minConsiderations for Conducting Clinical
Trials of Regenerative Medicinal Products

10 minBreak
Points to Consider for Regulations on Marketing Authorization Application (MAA) for AAVbased Gene Therapies and CAR-T Products
10 minBackground and Objectives
Masaaki Miyano, FIRM/Chugai

35 minPanel Discussion 1:
Regulatory review system• Requirement of clinical data
• ERA review at BLA/MAA• Alliance/reference country

35 minPanel Discussion 2:
CMC/GMP topics• Out of specification• In-country testing

16:40-17:10

17:10-17:55

17:55-18:00
18:30-20:00

Shigeaki Hayashi, FIRM/J-TEC

Panelists:
6 country/region regulators: TBD
Alex J. ZHANG, CMBA/Help Tx
Srinivasan N Kellathur, SAPI/Roche
Moderator:
Hirokuni Mizoguchi, FIRM/AstellasRuriko Shinozaki, FIRM/Astellas
Panelists:
Same with panel discussion 1Moderator:
Masaki Fujii, FIRM/Mitsui-soko
Masaaki Miyano, FIRM/Chugai

Non-clinical data package for AAV-based Gene Therapies
Yusuke Kagawa, FIRM/Novartis
30 minPresentation and Discussion
Aki Kito, FIRM/Chugai
ALL
Compilation of Case Studies on Cell and Gene Technology provided to the Patients as
Medical Care or Medical Practice without Market Authorization
Kunihiko Suzuki, FIRM/MEDINET
45 minPresentation and Discussion
James Chieh-liang Lin, MEDIGENALL
5 minClosing
Ken-ichiro Hata, FIRM<Reception> Invite all in-person attendants

Abbreviations: ABLE, Association of Biotechnology Led Enterprises in India; BPIPO, Biotechnology and Pharmaceutical Industries Promotion Officein Taiwan; CARM, Council for Advanced Regenerative Medicine in Korea; CMBA, China Medical Biotech Association in China; RMAF, Regenerative
Medicine Acceleration Foundation in Korea; SAPI, Singapore Association of Pharmaceuticals Industries in Singapore.