Crispr Therapeutics: Betting on One-Shot Gene Editing Success

Crispr Therapeutics AG, a gene-editing company, has a diversified pipeline supported by a strong $2.1B cash position. The company has made progress since my previous article, and I will provide an update on their current developments.

Crispr Therapeutics AG (NASDAQ:CRSP), a leading gene-editing company, has made significant strides since the last update. The company's diversified pipeline, supported by a robust $2.1B cash position, continues to attract investor interest.

CASGEVY Commercial Rollout
Crispr's commercial rollout of CASGEVY, an FDA-approved gene therapy for sickle cell disease, has shown promising results. In the first quarter of 2025, CASGEVY generated $14.2M in sales. The company, in partnership with Vertex Pharmaceuticals (VRTX), has activated over 65 authorized treatment centers worldwide, with cell collections initiated for over 90 patients and a referral pipeline of twice that number. While the initial patient numbers are modest, management frames this as a medical device rollout rather than a drug launch, highlighting the infrastructure establishment and payer support challenges.

Cardiovascular Franchise
Crispr's cardiovascular franchise, particularly the CTX-310 and CTX-320 programs, holds immense commercial potential. CTX-310, a gene-editing therapy targeting LDL cholesterol and triglycerides, demonstrated nearly 80% reduction in both metrics from a single injection. CTX-320, targeting LP(a), showed promising results in a Novartis (NVS) trial, validating its potential. Both therapies are one-time gene editing solutions priced at approximately $150K, offering a significant value proposition over current lipid-lowering therapies with recurring costs and adherence challenges.

Sirius Therapeutics Partnership
Crispr recently partnered with Sirius Therapeutics to gain access to their siRNA technology, represented by SRSD107. This long-acting siRNA selectively inhibits Factor XI, reducing the risk of thrombotic events and bleeding. A Phase I study showed over 93% FXI reduction and sustained effects for up to six months. A Phase II trial is launching in total knee arthroplasty patients, with commercialization rights for the U.S. market.

Risks and Considerations
While Crispr's progress is encouraging, several risks must be considered. CASGEVY's uptake is still in the early stages, and its commercial success will impact the company's gene therapies' outlook. The company's other promising programs, including cardiovascular, allogeneic CAR-T, and siRNA therapies, are in early stages and lack extensive patient data. Despite these risks, Crispr's strong cash position and pipeline diversity make it an attractive investment.

Conclusion
Crispr Therapeutics AG continues to make steady progress, with notable advancements in its gene-editing therapies and strategic partnerships. While risks remain, the company's diversified pipeline and strong cash position position it well for future growth. Investors should closely monitor the company's progress and consider the potential upside in its gene-editing therapies.

References
[1] https://seekingalpha.com/article/4794789-crispr-therapeutics-betting-on-a-one-shot-future