Generation Bio (GBIO) 24th Annual Needham Virtual Healthcare Conference summary

Strategic focus and technology overview

• Developing first-in-class targeted siRNA therapeutics for T-cell-driven autoimmune diseases using a proprietary, highly selective, and reducible LNP delivery system.

• LNP system avoids non-specific biodistribution, minimizing off-target effects by reducing liver and spleen uptake to less than 0.1% of the dose and extending half-life in primates to about eight hours.

• Demonstrated efficient and selective delivery to T-cells, including CD8, CD4, and NK cells, with minimal uptake in non-target cells.

• siRNA payloads show potent, persistent knockdown in preclinical models, with anticipated dosing intervals of once monthly or every two months.

• Lead target and candidate indications to be announced mid-year, focusing on diseases with unmet needs and therapeutic ceilings.

Market context and competitive positioning

• Selective delivery beyond the liver is seen as a key enabler for the future of genetic medicines, overcoming limitations of traditional AAV and LNP systems.

• Differentiation is based on the ability to dial out non-selective clearance and achieve true on-target delivery, not just ligand attachment.

• Safety profile in preclinical studies is favorable, with no liver toxicity or accelerated blood clearance upon redosing.

• Focus on T-cell targets is driven by the lack of existing technologies for selective intracellular knockdown in T-cells, offering a non-depleting approach that preserves T-cell state.

• siRNA is chosen for its stability, predictable pharmacology, reversibility, and compatibility with the delivery system; differentiation is mainly on the delivery side.

Collaborations, manufacturing, and financials

• Ongoing collaboration with Moderna focuses on T-cell selective LNPs for mRNA delivery, with no major business changes expected in the near term.

• Open to additional partnerships in oncology and other immune cell types, with potential new deals as data matures.

• Proprietary enzymatic manufacturing process for DNA offers speed, purity, and efficiency, supporting global therapy access.

• Strong financial position with $185 million in cash and runway projected into the second half of 2027, aiming for first in-human experience within this period.

• Advances in delivery technology are expected to unlock new applications for genetic medicines in larger disease areas.