CRISPR Therapeutics Eyes CASGEVY Growth, Year-End Pipeline Catalysts

Key Points

• CASGEVY adoption could accelerate following its FDA expansion to patients ages 2 and older, with pediatric treatment starts and potentially shorter conditioning regimens viewed as key growth drivers.
• CRISPR Therapeutics expects significant year-end catalysts, including Phase 1b data for CTX310 in severe hypertriglyceridemia, Phase 2 results for Factor XI therapy CTX611, and additional data from its zugo-cel autoimmune program.
• The company is advancing earlier-stage gene-editing technologies, including in vivo editing, broader tissue delivery and whole-gene insertion, with additional clinical data expected in 2027.

CRISPR Therapeutics NASDAQ: CRSP CEO Sam Kulkarni outlined the company’s commercial and clinical priorities during a Morgan Stanley discussion, highlighting continued adoption of CASGEVY and expected data updates across several pipeline programs by the end of 2026.

Kulkarni said the company’s strategy rests on several layers of potential value: the commercial launch of CASGEVY, three later-stage assets that could be positioned for pivotal studies next year, early clinical programs, and research efforts in areas including in vivo cell editing.

CASGEVY launch and pediatric expansion

CASGEVY, which CRISPR Therapeutics markets with Vertex Pharmaceuticals for sickle cell disease and transfusion-dependent beta thalassemia, recently received an FDA label expansion for patients ages 2 years and older. Kulkarni described the pediatric indication as a “great tailwind,” citing Vertex’s most recently disclosed quarterly CASGEVY revenue of approximately $76 million and more than 100 patient initiations per quarter.

He said the pediatric population could support additional treatment starts because more children’s hospitals have experience with the required procedures and because earlier intervention may help patients avoid disease-related organ damage.

According to Kulkarni, many initial launch bottlenecks—including reimbursement processes, hospital contracting and manufacturing capacity—have largely been addressed. However, the treatment journey can still take six to nine months or longer from initiation to treatment. He said delays are not primarily related to manufacturing, but can reflect exchange transfusions before cell collection and patient scheduling needs.

Kulkarni also pointed to potential gentler conditioning regimens as a future growth driver. He said a regimen that could reduce hospital stays to roughly three days or less, compared with two or three weeks, could broaden access among more moderate patients. He added that CRISPR Therapeutics is pursuing in vivo hematopoietic stem cell editing, which he said could ultimately expand treatment beyond the current markets in the U.S. and Western Europe.

CTX310 data expected in severe hypertriglyceridemia

The company expects to report Phase 1b data for CTX310 in severe hypertriglyceridemia by year-end. CTX310 is designed as a one-time CRISPR-Cas9 treatment targeting ANGPTL3, a protein associated with lipid metabolism and cardiovascular risk.

Kulkarni said data presented at the European Society of Cardiology meeting showed reductions of up to 80% in ANGPTL3 at higher dose levels, along with reductions of nearly 50% in LDL cholesterol and triglycerides in a mixed Phase 1a population. He said the company observed that ANGPTL3 reductions remained at approximately 80% one year after treatment in the data presented.

The Phase 1b study is evaluating more defined patient populations, including severe hypertriglyceridemia, mixed dyslipidemia and refractory hypercholesterolemia. Kulkarni said the immediate focus is severe hypertriglyceridemia, where CRISPR Therapeutics aims to assess triglyceride reduction and additional biomarkers such as LDL cholesterol, hemoglobin A1C and hepatic fat fraction.

He also said ANGPTL3 may be complementary to PCSK9 therapies, citing Phase 1a patients who were already receiving PCSK9 treatment and experienced additional LDL reductions. The company plans to use the Phase 1b results to discuss potential pivotal trial designs with regulators.

Factor XI and autoimmune programs

CRISPR Therapeutics also expects year-end top-line Phase 2 data for CTX611, an siRNA therapy targeting Factor XI that is being developed with Sirius Therapeutics. The ongoing study is in patients undergoing total knee arthroplasty and compares CTX611 with enoxaparin.

Kulkarni said the program seeks to provide anticoagulation with less bleeding risk than existing direct oral anticoagulants. He said prior Phase 1 studies showed up to 95% reduction in Factor XI. The company expects the Phase 2 update to include high-level comparisons of venous thromboembolism rates between treatment arms, while more detailed bleeding information is expected later.

Potential future settings discussed included secondary stroke prevention, atrial fibrillation patients unable to use direct oral anticoagulants, cancer-associated thrombosis and surgical applications. CRISPR Therapeutics is responsible for Phase 3 development and potential commercialization of CTX611, according to Kulkarni.

For zugo-cel, its allogeneic CAR-T program, the company is prioritizing B-cell-driven autoimmune diseases. Kulkarni said CRISPR Therapeutics is conducting a basket trial across indications that include systemic sclerosis and myositis, with additional data expected by year-end. The company expects those results to help determine whether one or more indications could advance toward pivotal development.

Earlier pipeline and technology priorities

Kulkarni said clinical data from the company’s alpha-1 antitrypsin deficiency program and CTX340 for refractory hypertension are expected in 2027. CTX340 targets angiotensinogen, and Kulkarni said changes in blood pressure could potentially be observed within days after gene editing.

Beyond individual programs, he said CRISPR Therapeutics is working to broaden delivery beyond the liver to areas such as the hematopoietic and immune systems, kidneys, central nervous system and muscle. The company is also pursuing more advanced editing approaches, including whole-gene insertion.

Kulkarni said the company is increasingly using artificial intelligence in regulatory writing, operational activities and product design. He said AI tools are helping the company evaluate mutations, optimize editing strategies and potentially accelerate advancement of programs into the clinic.

About CRISPR Therapeutics (NASDAQ:CRSP)

CRISPR Therapeutics AG NASDAQ: CRSP is a biotechnology company focused on developing gene-editing medicines using CRISPR/Cas9 technology. The company's programs are designed to modify disease-associated genes in human cells, with the goal of treating serious conditions across hematology, oncology, autoimmune disease and regenerative medicine.

Its most advanced product is Casgevy (exa-cel), an autologous cell therapy developed in collaboration with Vertex Pharmaceuticals. Casgevy uses CRISPR/Cas9 gene editing to modify a patient's blood stem cells and is approved in several markets for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia.

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