Steminent Biotherapeutics, Inc.

The field of cell and gene therapy (CGT) has seen remarkable progress in recent years, offering both potential and proven treatments for diseases that were once considered incurable. With a global pipeline of approximately 3000 therapies in various stages of development, and 100 products approved in leading markets worldwide, interest and momentum has grown for increased development, broader commercialization and accessibility of CGT products for patients worldwide. In this context, many countries in the Asia-Pacific (APAC) region have emerged as attractive markets within the global CGT field in terms of technology development, clinical trials (second only to the US), and product approvals. However, the region presents unique challenges due to its diverse regulatory landscape. Unlike the US and EU, each with a single regulatory framework, the APAC region features multiple jurisdiction-specific regulatory authorities with varying frameworks and guidances for product development, clinical research, market approval, and patient access. For CGT product developers, including strategies for product development and commercialization in targeted APAC countries can substantially increase the value proposition of their product candidate by adding market approvals and gaining broader patient access. To help expand global patient access to CGT products, the International Society for Cell & Gene Therapy (ISCT) APAC Industry Committee has recognized the need for a better understanding of jurisdiction-specific regulatory frameworks in the APAC region, and has created regulatory roadmaps for key countries in the region. The objective of this initiative is to provide clear, practical guides to these jurisdiction-specific regulatory frameworks for product developers planning to navigate this complex, regional regulatory environment. These jurisdiction-specific roadmaps will highlight regulations for CGT preclinical and clinical development, manufacturing guidance, and marketing authorization pathways for the most highly active CGT jurisdictions within the region. These roadmaps aim to help address the need for standardization of terminologies, definitions, and classifications for CGT products in the APAC region to provide greater clarity for developers. Here, the ISCT APAC Industry Committee presents regulatory roadmaps for Japan, Singapore, and South Korea, and moving forward plans to separately publish similar practical guides for China, Taiwan, India, Australia, New Zealand, and others in the region.

With the increase in cell and gene therapy (CGT) clinical trials in recent years has come a subsequent increase in the number of contract development and manufacturing organizations (CDMOs). Successful transition from development and early-phase clinical trials to commercialization of a CGT product often depends on selecting the best-suited CDMO. However, many CGT companies are small biotech companies that lack expertise in the field or do not have experience selecting and transferring a process to a CDMO.

Given the interest in this topic, a roundtable with CGT developers and CDMO members at the 2023 annual meeting of the International Society of Cell and Gene Therapy Paris discussed these critical aspects of product development, including technical expertise, risk sharing and timing of partnerships.

Here, we'll analyze the considerations discussed by the panel and elaborate on other factors crucial for CGT development.

Cell-based regenerative medicine is an innovative field that can potentially alter the overall survival and quality of life of patients with devastating diseases. Several cell therapy products (CTPs) have been approved within the last two decades, and more are under development. The establishment of an effective developmental strategy in accordance with the regulatory bodies of each country/region is crucial for fast delivery of each respective CTP. In particular, facilitating investigational new drug (IND) approval is important for accelerating the transition from non-clinical to clinical research/trial phases.

Here the authors compared the non-clinical prerequisites for initiating clinical studies in five Asian countries/regions (India, China, Korea, Taiwan and Japan) from an industry viewpoint. The authors first identified the differences and tried to clarify the perspectives/considerations underpinning the different requirements.

The authors' findings revealed that differences in regulations and development experiences, especially with CTPs, have led to clear differences in the non-clinical study package and its corresponding study design.

By sharing experiences of the research and development of CTPs among Asian countries/regions and including not only industry but also regulatory authorities, we will be able to expedite cross-border IND approval and eventually contribute to the early delivery of innovative CTPs to many Asian patients.