Investor Temperature Check: What’s Hot and What’s Not in CGT?
Summary of Key Points from Investor Round Table on May 30th, 2024
by Victoria Zellmer, PhD
Business Development Manager
US Central Territory – MaxCyte
Panelists:
Jason Bock, Ph.D., CTMC
Nagisa Sakurai, Ph.D., MBA, Astellas Venture Manager LLC
Phil Vanek, Ph.D., Gamma Bioscience
Bryan Poltilove, MBA, BroadOak Capital Partners
What’s Hot: Panelists were asked to discuss what types of cell & gene therapy (CGT) tools/technologies/products they are most enthusiastic about investing in at this time.
- Allogeneic Cell Therapies are High Risk, High Reward. Panelists discussed shared excitement over novel allogeneic cell therapies with an emphasis on hypoimmune engineering from induced pluripotent stem cells (iPSCs) (though not yet validated in human) – considered high risk for high reward and worth persistent shots on goal, with success continually measured by quality of clinical data.
- Second- and Third-Wave Tumor-Infiltrating Lymphocytes (TILs) Remain Worthy of Investor Attention. Significant market opportunity exists for TILs given that solid tumors compose >90% of all cancers and TILs have demonstrated favorable treatment outcomes in refractory disease. Panelists were optimistic that the FDA’s approval of Iovance Biotherapeutics’ lifileucel, a first wave non-engineered autologous cell therapy, would establish the regulatory pathway for second-wave TIL therapies involving cell engineering, specifically modifications by virus, CRISPR, etc. and ultimately third-wave combinatorial TIL therapies.
- Continued Momentum for Cell Therapy in Autoimmune Indications. The autoimmune disease market continues to expand due to rising prevalence and awareness, and cell therapies show potential for antigen-specific targeting of pathogenic cells without depletion of protective immunity. Given manufacturing, scalability, and hospital/outpatient infrastructure challenges, panelists were aligned that allogeneic cell therapies are most practical in this context. The regulatory pathway and commercial hurdles surrounding allogeneic cell therapies in autoimmune disease are evolving, as questions about the sufficiency of clinical data continue to arise.
- Renewed Interest in Autologous Therapies Calls for Cell Engineering & Process-Enabling Technology Around Scalability, Cost, and Automation. Manufacturing challenges (including exorbitant costs) shifted early cell therapy away from autologous therapies in favor of allogeneic; however, efficacy of autologous therapies has piqued investor interest and will require novel tools and platforms to improve scalability and reduce cost by increasing potency, thereby reducing COGs. Innovative strategies that streamline quality and product development while reducing interventions deserve further consideration. Particularly, panelists recognized the need for higher fidelity gene modification tools, including analytics that deliver improved accuracy and shorter development time horizons.
What’s Not Hot: Panelists were asked to discuss what types of cell & gene therapy (CGT) tools/technologies/products they are least enthusiastic about investing in at this time.
- Lack of Clarity Facing Exosomes Presents Investment Challenge. Therapeutic potential and commercial viability for exosomes are hindered by unresolved questions about cell type selection, regulatory pathway, and manufacturing scalability, creating a challenging investment landscape.
- Tempered Excitement Toward In Vivo CAR-T Therapy. Achieving a therapeutic number of CAR-T cells in an in vivo context remains challenging and inefficient. In vivo CAR-T therapy should not be viewed as a replacement for current therapies, but rather in supplement. Clinically significant data will be the key to securing investor buy-in.
- Cell Therapy Products Targeting CD19, CD20, CD22 or BCMA are Risky Business. Allogeneic autoimmune indications aside, significant commercial risks exist for those seeking to pursue these targets, including stiff competition and market saturation. Many candidates in this drug class are further threatened by hospital infrastructure challenges that will require a substantial overhaul to address.
- Undifferentiated Technology is Unappealing to Investors. The field is oversaturated with undifferentiated, ordinary platforms that maintain status quo. Panelists reiterated the need for process-enabling tools and platforms that reduce COGs and enable efficient tech transfer.
Closing Summary
When identifying therapies, tools, and technologies that will advance cell therapy and attract capital, it is clear investors are seeking clinically significant data and differentiated platforms that address the high costs associated with cell therapy manufacturing. Throughout the discussion, the panelists acknowledged the impact healthcare infrastructure and supply chain have on the future of cell therapy. As the cell therapy landscape continues to evolve, innovative patient-centered strategies that reduce manufacturing costs and logistical challenges will be critical for realizing the full potential of cell therapy.
The summary provided reflects general trends and opinions in the field of cell and gene therapy investment and should not be considered financial or investment advice.
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