CRISPR Therapeutics (CRSP) Jefferies 2024 Global Healthcare Conference summary

Platform and pipeline overview

• Commercialized Casgevy for severe sickle cell disease and beta thalassemia, demonstrating strong clinical data.

• Five clinical programs across seven trials and ten preclinical programs announced, aiming for a sustainable R&D engine.

• Casgevy is a 60/40 partnership with Vertex, with global commercialization led by Vertex.

• Growing interest in CRISPR-based therapies, especially among severe patients and those with high disease burden.

• Expansion into Middle East and Asia, with U.S. launch as primary focus.

Commercial launch and patient access

• 25 of 75 global centers activated for Casgevy, with increasing patient cell collections expected.

• Time from cell collection to treatment is influenced by patient scheduling, with opportunities for process optimization.

• Early adopters are diverse, often highly educated or with severe comorbidities, and eager for a one-time curative therapy.

• Medical tourism and payer ability in Middle East and Asia present additional market opportunities.

In vivo gene editing and pipeline expansion

• Two in vivo gene editing programs in clinic: CTX310 (ANGPTL3) and CTX320 (Lp(a)), targeting cardiovascular risk factors.

• Lp(a) program targets a large U.S. population, aiming for a one-shot therapy with durable effect.

• Initial clinical data expected this year, with focus on severe subpopulations for regulatory approval.

• Learnings from competitors like Intellia inform dosing and delivery strategies.