Chitose Laboratory Corporation (hereinafter, “CHITOSE”), the core company of CHITOSE Group, announced that a  novel Human Amniotic epithelial cell line for gene and cell Therapy (hereinafter, “HAT”) originally established in 2022, is now available for commercial use. HAT cells have the potential to reduce biomanufacturing costs and support the broader adoption of gene therapy by delivering higher yields and improved quality of adeno-associated virus (AAV) vectors compared to conventional cell lines. These findings were published in a high-impact, peer-reviewed journal, marking their recognition by the global scientific community.

Efficient Biomanufacturing to Expand Therapy Access
AAV vectors, derived from non-pathogenic viruses, have emerged as a leading platform in next-generation therapeutics, particularly in gene therapy, due to their capacity to efficiently deliver genetic material, enabling treatment of diseases that were previously considered intractable.

However, the widespread adoption of gene therapy remains hindered by challenges in large-scale production, purification, and quality control of AAV vectors, resulting in prohibitively high treatment costs. Enhancing biomanufacturing efficiency is therefore a critical step toward improving access to genetic therapy.

Key Advantages of the HAT Cells
The newly established HAT cells address these limitations by enabling efficient high-yield production of AAV vectors with minimal impurities, contributing to lowering manufacturing costs. The establishment process and detailed characteristics of HAT cells have been published in Molecular Therapy Methods & Clinical Development, a peer-reviewed high-impact journal focused on transformative research in cell and gene therapy.*1 Some of the key advantages of HAT cells are listed below:

・ High Productivity:
The lead HAT clone achieved high titers across multiple AAV vectors, with AAV5 yields reaching up to 2.7 × 10¹⁴ vector genome (vg) copies per liter of culture supernatant.

・ Improved Product Quality:
HAT cells produced AAV vectors with a significantly higher proportion of full capsids compared to HEK293 cells: 20.6% vs. 6.0% for AAV2 and 27.1% vs. 12.6% for AAV8.

・ Scalability:
Yield and quality of AAV vector production were maintained during scale-up in a 3-liter benchtop bioreactor, suggesting promising scalability under manufacturing conditions.

・ Ethical origin:
Donor material was obtained with prior informed consent for commercial use.

This development was achieved through our proprietary technology to cultivate and select cells with optimal characteristics. The same technology was previously applied to create CHO-MK*2 cells , a commercialized, highly productive cell line designed for the manufacturing of antibody drugs. Following the successful establishment of CHO-MK and HAT cells, CHITOSE will continue its mission to advance the biopharmaceutical industry and improve access to life-saving therapies.

*1 Establishment of a novel human amniotic epithelial-derived cell line, HAT, for high-yield AAV vector production
Hirai, Yugo et al. Molecular Therapy Methods & Clinical Development, Volume 33, Issue 4, 101594
*2 The establishment of CHO-MK was supported by AMED under the Grant Number JP18ae0101054

R&D Structure and Collaboration
The development was carried out by CHITOSE and the Manufacturing Technology Association of Biologics (hereinafter, “MAB”), in collaboration with the National Research Institute for Child Health and Development*3, supported by funding from the Japan Agency for Medical Research and Development (AMED) under the following projects:

Project Focused on Developing Basic Technology Aiming at Industrialization of Regenerative Medicine and Gene Therapy (Development of Manufacturing Technology for Gene Therapy)
— Theme: Development of platform manufacturing technology for gene and cell therapy vectors (JP18ae0201001)
— R&D Representative: Takeshi Omasa

Project Focused on Developing Basic Technology Aiming at Industrialization of Regenerative Medicine and Gene Therapy (Accelerated Development Research Project for Gene Therapy)
— Theme: Integrated development of viral vector production technology (JP24se0123004)
— R&D Representative: Takeshi Omasa

*3 CHITOSE and the National Research Institute for Child Health and Development are MAB association members

Availability and Inquires
CHITOSE is the sole authorized distributor of HAT A2C1 cells for commercial purposes. For inquiries and further information about obtaining the HAT A2C1 cells, please contact us at the email below.

CHITOSE Group Overview
https://chitose-bio.com/
CHITOSE Group is a family of biotechnology companies leading the global bioeconomy. To live in abundance beyond the next millennium using the ability of living things, CHITOSE pursues the possibilities of biotechnology through technological and business development collaborating with its business partners all over the world.

About CHITOSE BIO EVOLUTION PTE. LTD. (head office that oversees CHITOSE Group)
・Established in October, 2011
・Head Office located in Singapore
・CEO: Tomohiro FUJITA, Ph. D.

About CHITOSE Laboratory Corp. (a core company of CHITOSE Group that focuses on business and technological development)
・Established in November, 2002
・Head Office located in Kawasaki-city, Kanagawa Prefecture, Japan
・CEO: Tomohiro FUJITA, Ph. D.
・COO: Rie KUGIMIYA