Cabaletta Bio (CABA) H.C. Wainwright 4th Annual BioConnect Investor Conference summary

Industry context and scientific advances

• CD19-directed autologous CAR T therapy showed transformative results in autoimmune disease, first demonstrated in a lupus patient who achieved remission after a single dose, sparking widespread industry interest and innovation in cell therapy for autoimmunity.

• The field has since seen a surge in efforts to replicate these results, with multiple modalities and companies entering the space.

Manufacturing innovation and scalability

• Traditional autologous CAR T manufacturing was not scalable or affordable for large autoimmune indications due to high capital requirements, cost of goods, and hospital-based administration challenges.

• Adoption of automated, industrialized manufacturing through a partnership with Cellares enables production of tens of thousands of doses annually, with strong comparability to manual processes.

• This approach reduces costs, capital investment, and logistical barriers, making outpatient administration feasible due to improved safety.

Product design and clinical differentiation

• Rese-cel was designed as a fully human, weight-adjusted, redosable product specifically for autoimmune patients, closely mirroring the academic protocols that produced the original breakthrough results.

• Safety profile allows for outpatient treatment, expanding access and improving reimbursement models.

• Pediatric indications, such as juvenile myositis, are being targeted, with potential for priority review vouchers.