CRISPR Therapeutics (CRSP) Jefferies Global Healthcare Conference 2026 summary

Pipeline overview and strategic focus

• Commercialization of CASGEVY is progressing well, with strong revenue trajectory and global market expansion.

• Three next-generation assets (CTX310, zugo-cel, CTX611) are expected to be phase III-ready by late 2024 or early 2025.

• Additional assets targeting refractory hypertension, Alpha-1 Antitrypsin, and Lp(a) are advancing, with clinical entry or data readouts anticipated this year.

• In vivo HSC editing and LNP-based CAR Ts represent future growth areas, aiming for broader patient reach and simpler administration.

• The pipeline is described as broad and rich, with multiple catalysts expected in the next 12 months.

CTX310 (ANGPTL3) program updates

• Expansion phase includes diverse patient populations: severe hypertriglyceridemia, refractory hypercholesterolemia, mixed dyslipidemias, and homozygous familial hypercholesterolemia.

• High-dose levels achieve nearly 100% liver editing, resulting in up to 80% ANGPTL3 reduction.

• ANGPTL3 shows synergy with PCSK9 therapies and may offer a superior safety profile compared to APOC3.

• Phase III design will be informed by ongoing phase I-B data and regulatory discussions by year-end.

• Accelerated approval may be possible using LDL as a biomarker in certain populations.

Lp(a) and Alpha-1 Antitrypsin programs

• Advancement of Lp(a)-lowering assets (CTX320, CTX321) depends on outcomes from the Novartis HORIZON trial, with CTX321 targeting up to 90% reduction.

• Alpha-1 Antitrypsin program leverages SyNTase editing, aiming for best-in-class efficacy and potentially curative outcomes.

• Preclinical models show up to 5x improvement in AAT production; clinical entry is planned for mid-2024.

• Regulatory path de-risked by competitor progress, with a large patient population supporting commercial potential.