Key insights from the symposium included:
1. Consistency and capacity are priorities for CAR-T cell commercial product launches.
Variable quality in manufacturing chimeric antigen receptor T-cell (CAR-T) therapies can lead to out-of-spec (OOS) products that create challenges for hospitals, physicians, and patients. From the outset, companies have been designing modular processes built to expand manufacturing while controlling costly OOS production.
Deployment planning extends to the yearlong onboarding process for treatment centers. Companies are also focused on the extensive data collection needed to monitor the rollout of these emerging therapies.
2. Encouraging outlook for SCD and TDT genome editing therapies.
Several companies have cell therapies in development for sickle cell disease (SCD) and transfusion-dependent thalassemia (TDT), establishing an encouraging outlook for the future of genome editing to address these globally widespread diseases. Current players—and especially genome editing companies—are vying to claim the best approach to in vivo delivery. Targeted lipid nanoparticles that can specifically deliver editing enzymes and guide RNAs to the hematopoietic stem cells in the bone marrow may be the next wave of in vivo therapeutics for SCD and TDT.
3. TCR therapy represents huge opportunity in treating tumors.
T-cell receptor (TCR) therapy companies are engineering T-cells to recognize and precisely eliminate different tumor antigens or receptors using novel platforms. TCR therapy offers considerable promise in treating solid tumors with transformative and durable benefits for patients, particularly when leveraged earlier in the treatment journey.
Perspectives differ on the frequency and significance of Human Leukocyte Antigen (HLA) downregulation, but companies are exploring innovative ways to address other tumor-specific mechanisms of resistance.
Challenges in effectively developing and delivering TCR therapies include manufacturing costs and scalability, regulatory hurdles, safety and efficacy evaluation, data collection to validate results, and durability and quality of life assessment.
4. Novel cell therapies lead the charge for a diabetes cure.
One of the innovative technologies in development to address diabetes uses an implanted cell pouch device filled with iPSC-derived Islet-like clusters (ILCs) which is showing success in achieving durable insulin independence. A similar delivery approach uses a unique cell line derived from the human pancreas, with cells housed in an implanted omentum pouch rather than a device.
Other methods to control Type 1 diabetes include implanting almost fully differentiated cyT49 human stem cells that mature into insulin-secreting beta-like cells which are engineered to avoid destruction by the patient’s immune system. Anticipation is high for new data over the next 18 months that will demonstrate the potential for durable functional cures for diabetes.
5. Large pharma has a vital role to play.
Lowering the cost of manufacturing and increasing patient access are critical steps that will allow cell therapy to fulfill its unprecedented potential, drastically changing the course of many serious diseases. Pharmaceutical companies are well positioned to achieve these goals, given their capacity to scale and reduce cost of goods sold.
Shorter culture times for autologous CAR-T may yield a more nascent cell phenotype and significantly lower manufacturing costs and door-to-door time, as CAR-T moves beyond malignancies and expands to autoimmune diseases.
6. Venture capital is bullish on cell therapy’s future.
Investors in therapeutics see an encouraging outlook—for patients and investors alike—with continued innovation in the cell therapy space as well as more regulatory approvals and commercial launches. Especially rich areas for development include:
- High unmet medical needs for cell therapy in autoimmune diseases
- Promising preclinical solid tumor therapies, including TCR and CAR-T approaches
- In vivo gene therapy that could offer functional cures or long-term benefits
For effective market penetration and widespread adoption, cell therapy products must show clinical benefit and value to justify their pricing, moving toward value-based pricing and risk-sharing models. Collaboration between scientific founders, academic centers, and cell therapy companies provides the formula for commercially viable breakthroughs.
