CRISPR Therapeutics (CRSP) TD Cowen 46th Annual Health Care Conference summary

Strategic direction and business segments

• Transitioning from a technology-focused to a disease-focused model, emphasizing cardiovascular and autoimmune franchises.

• By mid-2027, expects data from six programs beyond Casgevy across more than six indications.

• Cardiovascular pipeline includes CTX310, CTX611, LPA, and CTX340 for refractory hypertension.

• Autoimmune focus includes CTX320 and upcoming siRNA targets, with additional work in type 1 diabetes and oncology.

Casgevy performance and outlook

• Patient initiations grew from 100 in 2024 to over 300 in 2025, with expectations for further growth in 2026.

• Revenue inflection anticipated as more sites become productive and patient demand remains strong.

• Time from referral to treatment ranges from six months (thalassemia) to a year (sickle cell), with low dropout rates.

• 2025 expected to be peak spend year for the franchise, with profitability anticipated but not yet guided.

• Investments in gentler conditioning and in vivo HSC delivery aim to expand the addressable market.

In vivo and cardiovascular pipeline updates

• CTX310 for ANGPTL3 showed 80% reduction in ANGPTL3, 50% LDL, and 60% triglyceride reduction at highest doses.

• Potential for fast-track approval in homozygous familial hypercholesterolemia; regulatory meetings will clarify registrational path.

• CTX320 and CTX321 for Lp(a) reduction await HORIZON data to determine prioritization; CTX321 shows higher potency in preclinical models.

• Gene editing seen as optimal for early intervention in Lp(a) due to lifelong risk.

• Safety profile for in vivo programs remains strong, with minor, self-resolving LFT increases.