NIE 2023 | Jia Guodong from iGene Biotechnology: Challenges and Opportunities of CGT — From Process Development to Industrialization

Jia Guodong pointed out that global biotech investment and financing entered a warming phase in 2023. In the sub-field of cell and gene therapy, after an outbreak in the first quarter of 2021, transactions gradually moderated down, but both transaction amounts and volumes increased. In terms of research and development directions, Jia Guodong stated that CGT products have evolved from initial hematological malignancies to a large number of solid tumor projects. At the same time, the application areas are also expanding continuously, including significant layouts in the neurological field. Currently, there have been substantial changes in the basic research and clinical pipeline of the CGT industry. Meanwhile, MNCs are also repositioning their pipelines through mergers and acquisitions.

In terms of commercial progress, Jia Guodong stated that whether the price of products is in the hundreds of thousands of dollars or millions of dollars, a large number of products have seen sales exceed one billion dollars. The commercial attributes of the CGT industry are gradually materializing, and a market worth hundreds of billions has emerged.

Jia Guodong pointed out that CGT products in the confirmatory clinical research phase and commercial production phase will face challenges in capacity, process, regulation, cost, and other aspects.

In terms of production capacity, in recent years, a large amount of capital has flowed into CDMO to fill the capacity gap. Since 2019, investment in the viral vector production industry has increased significantly, and the trend towards capacity expansion has intensified, leading to industry prosperity. This has brought about rapid development of CGT and an expansion of production capacity. However, the capacity that meets the listing requirements for CGT drugs is still insufficient.

In terms of technology, CMC issues have always been of utmost importance. The regulation of CAR-T drugs is becoming increasingly stringent, posing a huge challenge to CMC. Two-thirds of drugs have undergone re-review and re-submission, and from the content of review reports, the reasons for these re-review and re-submission are basically related to CMC. Companies in the CGT field need to take this matter seriously.

In terms of regulation, the emergence of new technologies poses a huge challenge to the regulatory science in the CGT field. Regulatory authorities continuously issue new technical guidelines, which require industry learning and adaptation by regulators to promote flexible production capacity, flexible processes, and comprehensive technical capabilities.

In terms of cost, the commercialization challenge is not only about production and technology, but also about cost control and improving accessibility. Currently, cell therapy in the United States has entered an era of commercialization, with cost and standardization issues gradually becoming the main contradictions. The costs during the CGT production phase can be controlled through localization of materials and equipment, standardization of production processes, scaling up product preparation, automation of the production process, and intelligence.

At the end of his speech, Jia Guodong introduced Harmony Biosciences' CDMO business. Harmony Biosciences has achieved full industrialization support, possesses advanced technology and a large-scale GMP base to empower gene therapy drug development, and continues to expand its CDMO capacity to ensure that gene therapies are faster, better, and more accessible.

He Yuan Biology (Stock Code: 688238) was established in 2013. It is a biotechnology company focusing on the field of gene therapy, dedicated to providing CRO services such as gene therapy vector development and gene function research for basic gene therapy research; it also offers CDMO services including process development and testing, IND-CMC pharmacological research, and GMP production of clinical samples for the research and development of gene therapy drugs, including recombinant viral vector drugs, oncolytic viruses, CAR-T cell treatment products, etc. With the mission of "Empowering Gene Therapy, Jointly Guarding Life and Health," and centered on gene therapy vector research and production, the company will adhere to a customer-centric approach and take providing professional services as its own responsibility. It aims to build an internationally leading gene and cell therapy CXO group enterprise, accelerate the basic research, drug discovery, pharmacological research, clinical, and commercialization processes of gene therapy, promote the development of the gene therapy industry, and benefit human health!