BIO Asia-Taiwan Kicks Off with the Latest Cell and Gene Therapy Developments

The first day of BIO Asia-Taiwan featured sessions on cell and gene therapies (CGTs). An in-depth session began with an enthusiastic overview of their transformative potential to enhance patient care. Presenters emphasized Asia’s growing role in the global CGT market and explored innovative treatments for various diseases. A comprehensive review of the gene therapy landscape highlighted rapid growth, significant market potential, and challenges like manufacturing and regulatory hurdles, accessibility, and affordability. The session also showcased the game-changing potential of induced pluripotent stem cells (iPSCs) for off-the-shelf therapies, focusing on reprogramming and engineering. It covered the complexities of clinical trials in CGT development and provided investor insights on reimbursement acceptance, stressing the need for differentiation and innovation in a competitive market.

Updates on Cell and Gene Therapies 

Chien Tsun Kuan opened the session with enthusiasm, expressing excitement for the advancements in CGTs and their potential to improve patient health. He emphasized the importance of capitalizing on Asia’s potential in the global CGT market and hoped the conference would provide a valuable platform for discussing various diseases and their treatments.

Dr. John Tsai, CEO of Forcefield Therapeutics and Executive Partner at Syncona, provided a comprehensive overview of the gene therapy landscape. He highlighted the significant growth in CGT over the last 15-20 years, with notable growth in the past 2-3 years. Tsai projected the market could reach $52 billion by 2053, far exceeding earlier estimates of $10-20 billion. Currently, 10% of the drug pipeline in clinical trials comprises CGT, reflecting its growing importance.

However, Dr. Tsai identified several challenges that have obstructed the progress of gene therapy, including technical difficulties in CMC manufacturing, AAV capsid usage and dosage requirements, as well as regulatory and logistical challenges. Furthermore, market access and pricing have been identified as significant hurdles. Nevertheless, advancements like the accelerated approval of gene therapies, exemplified by the approval of Elevidys, have propelled the field forward. Tsai concluded by stressing the need for a broader impact of gene therapy for its success and envisaged an evolving treatment landscape driven by these therapies.

Driving the Next Generation of Innovative and Affordable Cell Therapies across Indications

Alex Huang, VP and Head of Cell Therapy at BeiGene, focused on the making of cancer treatments both more accessible and affordable. He noted that while durable efficacy has been observed in cell therapies, it is not guaranteed. To this end, Huang emphasized the potential of induced pluripotent stem cells (iPSCs) as a game-changer for off-the-shelf cell therapy, essentially enhancing access and affordability.

To succeed with iPSCs, Huang outlined three key areas: reprogramming, engineering, and differentiation and expansion. Here he noted that acquiring a well-characterized, proprietary GMP iPSC clone collection is crucial for source material. He stated that “we need to strengthen the efficacy and persistence of the iPSC-Derived Effector Cells”, which is vital for the engineering process. Huang highlighted that Beigene’s feeder-free protocol allows for sufficient expansion, thereby addressing one of the critical challenges in cell therapy development.

Clinical Trials Design and Execution Crucial for Success; Regulatory Frameworks and Manufacturing Pivotal Considerations

Following, Chia-Hua Maggie Ho, a speaker from Amarex discussed the clinical landscape of CGT development, emphasizing the regional variations in therapy focus. Here she focused on the key considerations in CGT development, which include patient population and recruitment, country and site selection, logistics and manufacturing, and long-term follow-up. Ho stressed the importance of early investment in site selection to find the target population and the need for a sustainable patient identification and genetic screening process. A pragmatic study design and the “light touch” method can help reduce patient burden during the 15-year follow-up required by the FDA for cell and gene therapies.

Following, speaker David Chang of TBMC highlighted the company’s commitment to advancing cell therapy through innovative approaches. He emphasized that cell therapy represents a highly meaningful modality for future medical progress, stating that “”cell therapy is truly a meaningful modality to move forward in.” Further, Chang outlined TBMC’s goal to develop a fully integrated, end-to-end automated cell manufacturing platform, showcasing how automation is essential for efficiency and progress in this field, adding that “automation is the way to go.” He also introduced TBMC’s biomaterial platform, which aims to harness combinatorial technology derived from mesenchymal stem cells (MSCs) in conjunction with advanced biomaterials. 

The Perspectives from Investors on Cell and Gene Therapy

To end the session, Gur Roshwalb, Partner at aMoon, shared insights from an investor’s perspective on CGT. He highlighted that reimbursement for cell therapies has been widely accepted, which is crucial for their continued growth. However, the challenge lies in the fact that many platforms are targeting the same diseases, making differentiation and innovation essential for success, stating that “if you really want to be rewarded you have to come up with real differentiation/innovation.” Moreover, Roshwalb went on to stress the importance of demonstrating efficacy to both physicians and patients.

Roshwalb also pointed out the declining valuations for CGT, which is problematic for investors. To address this, the industry needs to solve manufacturing, regulatory, efficacy, and reimbursement issues. He mentioned that gene therapy in ophthalmology, areas of immune escape, and the CNS are promising fields. Roshwalb concluded by emphasizing the need for more innovation in cell therapy, moving beyond CD19 and BCMA to explore new targets like T-regs.

Key Take-Aways: Collaboration, Innovation, Regulatory Alignment, Strategic Investments

The session concluded with moderator remarks highlighting the importance of collaboration across all sectors to enhance the development of CGT. The speakers collectively underscored the potential of CGT to revolutionize treatment landscapes, provided challenges are addressed through innovation, regulatory alignment, and strategic investments.

Author

Bernice Lottering