Editas Medicine (EDIT) investor relations material

Conference overview

• The session focused on the next wave of technologies and programs in genome editing, highlighting advances in ex vivo and in vivo editing, delivery innovations, and commercial challenges.

• Panelists included CEOs and CTOs from leading genome editing companies, each presenting their technology platforms and clinical pipelines spanning rare to prevalent diseases.

• Companies are leveraging diverse editing tools (CRISPR, base, prime, epigenome editing) and delivery systems (LNPs, AAVs, PDVs) to address a broad range of indications.

• The discussion emphasized the coexistence of multiple editing technologies, with strategic selection based on target, differentiation, and clinical need.

• Lessons from first-wave therapies include the importance of delivery optimization, indication choice, and the need for business model innovation.

Technology and delivery strategies

• Delivery systems are critical, with LNPs and novel protein delivery vehicles enabling tissue-specific and efficient in vivo editing.

• LNPs are not uniform; their composition, targeting moieties, and ionizable lipids significantly impact safety, efficacy, and tissue tropism.

• Platform approaches allow for rapid expansion into new indications, leveraging validated formulations to streamline regulatory and manufacturing processes.

• Moving beyond the liver, companies are exploring CNS delivery using AAVs, balancing risk and opportunity in challenging indications.

• Non-viral delivery is seen as essential for global access due to lower cost of goods and broader scalability.

Commercial and business case

• One-time gene editing therapies face unique commercial challenges, especially in rare diseases where patient pools shrink as treatments succeed.

• Demonstrating transformational efficacy and high responder rates is key to commercial viability and payer acceptance.

• Cost of goods, pricing strategy, and margin optimization are central to building sustainable business models, with a shift toward low-cost, scalable delivery systems.

• Companies are increasingly disciplined in indication selection, focusing on areas with clear unmet need and commercial potential.

• The next five years are expected to bring multiple approvals, launches, and a maturing payer landscape, validating the business case for gene editing therapeutics.