CRISPR Therapeutics (CRSP) 46th Annual William Blair Growth Stock Conference summary

Company overview and strategic direction

• Over a decade in gene editing, now transitioning to a broader biotech leader with a diversified pipeline in cardiovascular, autoimmune, oncology, and siRNA therapies.

• Five clinical readouts expected by late 2026, with two additional programs entering the clinic and data anticipated in the next 12-18 months.

• Moving from a technology-focused to a modality-agnostic, therapeutic area-driven company, aiming for significant market cap growth.

CASGEVY launch and commercial progress

• CASGEVY is the first globally approved CRISPR therapy, with over 500 patients initiated and more than 100 infused, supported by a growing network of 75+ ATCs.

• Targeting a potential market of 60,000 patients across the US, EU5, and Middle East, with a price of $2.2 million per treatment.

• Revenue recognition is ramping up as foundational infrastructure is established, with a low dropout rate among patients.

• Expansion into pediatric populations is underway, with an sBLA submitted and priority review status granted.

Pipeline and clinical development

• Active programs in cardiovascular (CTX310, CTX611), autoimmune (zugo-cel), and oncology, with a focus on both permanent and transient gene editing solutions.

• siRNA franchise established via a partnership with Sirius, targeting Factor XI for anticoagulation, with a phase II readout expected in the second half of the year.

• In vivo CAR-T and HSC programs leverage internal manufacturing and gene editing expertise, aiming for differentiation through delivery and durability.