Generation Bio (GBIO) TD Cowen 45th Annual Healthcare Conference summary

Strategic focus and platform overview

• Developing a proprietary ctLNP platform for selective siRNA delivery to T cells, targeting T cell-driven autoimmune diseases with high precision and minimal off-target effects.

• Lead program, MY25, targets undruggable T cell intracellular proteins, with an IND planned for the second half of 2026 and a lead target announcement mid-year.

• Cash runway extends into the second half of 2027, supporting preclinical and early clinical development.

• Platform enables highly selective delivery to T cells, opening new therapeutic opportunities in autoimmunity and addressing a large, underserved market segment.

• The modular ctLNP platform can be adapted to target other immune and non-immune cell types, expanding therapeutic opportunities beyond T cells.

Differentiation and technology innovation

• Combines T cell selectivity with the ability to target intracellular proteins using siRNA, a novel approach in the autoimmune space.

• ctLNP system dials out non-specific biodistribution and dials in cell-specific targeting using biological ligands, avoiding liver and spleen clearance for prolonged circulation.

• Achieves dramatic reduction in off-target delivery and extends LNP half-life to hours in primates.

• Over 90% of targeted T cells in humanized mice transduced with a single dose, with negligible off-target uptake.

• Platform is positioned as superior to traditional LNPs and siRNA conjugates, which are ineffective for T cell delivery due to rapid clearance and lack of specificity.

siRNA delivery and therapeutic potential

• Overcomes historical challenges of siRNA delivery to T cells by enabling potent, durable knockdown of target genes, with robust, dose-dependent, and persistent effects in vitro and in vivo.

• Durability of knockdown suggests potential for monthly or six-weekly dosing in humans.

• siRNA offers a reversible, potent, and precise alternative to gene editing or CAR-T approaches.

• The platform's modularity allows for rapid adaptation to new targets and indications, supporting a broad pipeline strategy.

• The company is prioritizing indications with high unmet need and clear clinical pathways, such as autoimmune hepatitis, IBD, vasculitis, and dermatological conditions.