Regulatory insights on advanced CAR-T cell products, AAV-based Gene therapies, and medical care/practice in cell and Gene therapies: Report from the 6th Asia partnership conference of regenerative medicine- April 20, 2023

Hiroshi Karasawa; Hirokuni Mizoguchi; Bryan Choi; Chia-Ling Hsieh; Masaaki Miyano; Yuu Moriya; Sasikumar Muthusamy; Koji Takakura; Kunihiko Suzuki; Yoshie Tsurumaki; Takeshi Watanabe; Karen Wen; Tomohiro Yoneda; Ta-Tung Yuan; Masayuki Nomura

doi:10.1016/j.reth.2024.09.015

Regulatory insights on advanced CAR-T cell products, AAV-based Gene therapies, and medical care/practice in cell and Gene therapies: Report from the 6th Asia partnership conference of regenerative medicine- April 20, 2023

Regen Ther. 2024 Oct 29:26:967-980. doi: 10.1016/j.reth.2024.09.015. eCollection 2024 Jun.

Authors

Affiliations

¹ Daiichi Sankyo Co., Ltd., 1-2-58, Hiromachi, Shinagawa-ku, Tokyo, 140-8710, Japan.
² Astellas Pharma Inc., 2-5-1, Nihonbashi-Honcho, Chuo-ku, Tokyo, 103-8411, Japan.
³ Inha University College of Medicine, 100 Inha-ro, Nam-gu, Incheon 22212, South Korea.
⁴ Development Center for Biotechnology, No.107, Ln. 130, Sec. 1, Academia Rd., Nangang Dist., Taipei City, 11571, Taiwan.
⁵ Chugai Pharmaceutical Co., Ltd., 5-1, Ukima 5-Chome, Kita-ku, Tokyo, 115-8543, Japan.
⁶ Takeda Pharmaceutical Company Limited, 26-1, Muraoka-Higashi 2-Chome Fujisawa, Kanagawa, 251-8555, Japan.
⁷ Novartis Healthcare Private Limited, Salarpuria Sattva, Sy. No. 83/1, Plot No.2 Near Inorbit Mall, Raidurg, Hyderabad, 500081, India.
⁸ Astellas Pharma Inc., 21, Miyukigaoka, Tsukuba, Ibaraki, 305-8585, Japan.
⁹ MEDINET Co., Ltd., 6-1-1 Heiwajima, Ota-ku, Tokyo, 143-0006, Japan.
¹⁰ Novartis Pharma K.K., 1-23-1, Toranomon, Minato-ku, Tokyo, 105-6333, Japan.
¹¹ GenomeFrontier Therapeutics, Inc., 23F., No. 95, Sec. 1, Xintai 5th Rd., Xizhi Dist., New Taipei City, 221416, Taiwan.
¹² TFBS Bioscience Inc., 3F., No.103, Ln. 169, Kangning St., Xizhi Dist., New Taipei City, Taiwan.
¹³ Asahi Kasei Corporation, 1-1-2 Yurakucho, Chiyoda-ku, Tokyo, 100-0006, Japan.

Abstract

The 6th Asia Partnership Conference of Regenerative Medicine (APACRM) was held in person with online on April 20, 2023, to promote the regulatory harmonization of regenerative medicine products throughout Asia. Recognizing domestic regulatory guidelines within each country and region and the underlying rationales are important initial steps toward harmonizing regulations. The 6th APACRM featured an open dialog regarding non-clinical evaluation for advanced CAR-T products, regulation of clinical trials for AAV-based gene therapies, and cell and gene therapies provided to patients as medical care/medical practices without market authorization through presentations from the industry and panel discussions with regulatory agencies. The latest updates on regenerative medicine in each country and region are introduced. This paper summarizes the proceedings of the 6th APACRM for public dissemination to foster future discussions.

Keywords: AAV-Based gene therapies; APACRM; Asia; Cell and gene therapy product; Non-clinical; Quality; Regulatory guideline.

Publication types

Review