CRISPR Therapeutics Enters 'Second Phase' as CASGEVY Momentum Builds, Pipeline Data Looms

Key Points

• CRISPR Therapeutics says it is entering a “second phase” after the CASGEVY launch, shifting focus from commercialization to a broader pipeline with data expected from six assets over the next 12 to 18 months.
• CEO Sam Kulkarni said CASGEVY momentum is building, with patient initiations rising sharply through Vertex and future revenue expected to follow after a lag in the treatment and reimbursement process.
• The company highlighted several potential growth drivers, including a pediatric label expansion for CASGEVY and promising early data across cardiovascular, autoimmune, oncology and rare disease programs such as CTX310 and zugo-cel.

CRISPR Therapeutics NASDAQ: CRSP Chief Executive Officer Sam Kulkarni said the company is entering a “second phase” as it moves beyond the initial launch of CASGEVY and prepares for data from multiple pipeline programs over the next 12 to 18 months.

Speaking at a Bank of America fireside chat hosted by analyst Alec Stranahan, Kulkarni said the company’s first 11 years were centered on developing CASGEVY for sickle cell disease and beta thalassemia and bringing the therapy to patients. With that program now commercialized through partner Vertex, he said CRISPR Therapeutics is shifting more attention to a broader portfolio that includes cardiovascular, autoimmune, oncology and rare disease programs.

Kulkarni said the company expects six assets to generate data in the next 12 to 18 months. He described the pipeline as including CTX310, an ANGPTL3-targeting program for LDL cholesterol and triglyceride reduction; zugo-cel, an allogeneic CAR T cell therapy being developed for autoimmune disease and oncology; CTX611, a long-acting siRNA approach to blood thinning; CTX340, a hypertension program targeting angiotensinogen; an Lp(a) program; and an alpha-1 antitrypsin rare disease program.

CASGEVY Launch Gains Momentum, CEO Says

Kulkarni said CASGEVY’s commercial rollout is “gaining a lot of momentum,” though he emphasized that the launch differs from a typical pharmaceutical launch because of the time required between patient initiation, cell collection, manufacturing, infusion and revenue recognition.

He said Vertex, which is leading commercialization, initiated about 100 patients in 2024, more than 300 patients in 2025 and has now initiated more than 500 patients. Kulkarni said the growth in patient initiations should translate into future revenue as patients move through the treatment funnel, though he noted there can be a lag of two to three quarters from initiation to revenue recognition.

“It’s a certainty that it all falls through because you’re not seeing patients drop out of the journey,” Kulkarni said. “It’s just a matter of time.”

Stranahan noted that CASGEVY generated $43 million in the first quarter. Kulkarni said CRISPR Therapeutics does not see major headwinds for the product at this stage and said Vertex is executing well on supply chain and patient handling. He said the company feels “comfortable about the trajectory of the product.”

Pediatric Label and Reimbursement Seen as Tailwinds

Kulkarni pointed to several potential tailwinds for CASGEVY, including a pediatric label expansion. The current U.S. label covers patients ages 12 and older, and the company has submitted for an expansion to patients ages 5 and older. Kulkarni said treating younger patients could help prevent vascular and organ damage associated with disease progression.

He also said the pediatric expansion could bring more children’s hospitals into the treatment network, potentially increasing center activation and treatment velocity.

Outside the United States, Kulkarni said CASGEVY is the only available option in certain markets. He also highlighted a reimbursement agreement in Germany, describing it as a significant achievement given prior challenges faced by a competitor in that market.

Kulkarni also discussed “gentler conditioning” as a future potential expansion of CASGEVY’s life cycle. He said CRISPR Therapeutics has not provided guidance on when such an approach might be available, but said it could meaningfully broaden the addressable population if it achieves results comparable to the current busulfan conditioning regimen.

Cardiovascular Programs Target Large Markets

On CTX310, Kulkarni said the company presented data last year showing reductions of approximately 50% in LDL cholesterol or triglycerides after treatment. He said the therapy uses lipid nanoparticle delivery and described the early safety profile as favorable, with limited and self-resolving liver enzyme elevations observed.

For homozygous familial hypercholesterolemia, Kulkarni said the regulatory bar could be relatively low if the therapy can show additional LDL reduction on top of agents such as PCSK9 inhibitors. For severe hypertriglyceridemia, he said CRISPR Therapeutics needs more patient data before engaging regulators on a potential registrational path.

Kulkarni also discussed CTX340, which targets angiotensinogen for hypertension. He said a gene-editing approach could provide consistent blood pressure reduction, in contrast to therapies that may wear off toward the end of a dosing period. He said reducing systolic blood pressure by 10 to 15 millimeters of mercury could be clinically meaningful, while still allowing physicians to adjust other medications.

Separately, Kulkarni said the company’s collaboration with Sirius Therapeutics on a Factor XI siRNA program reflects a “right tool for the job” approach. He said CRISPR Therapeutics does not want to permanently edit Factor XI because anticoagulation may be needed for defined periods or specific patient populations. He said the company sees potential indications including secondary stroke prevention, atrial fibrillation patients not eligible for DOACs and peripheral artery disease after revascularization.

Zugo-cel Advances in Autoimmune Disease and Oncology

Kulkarni described zugo-cel as a potential best-in-class allogeneic CD19 CAR T therapy, citing what he called autologous-like efficacy with the convenience and cost-of-goods profile of an allogeneic product.

In oncology, he said CRISPR Therapeutics previously showed a nearly 70% complete response rate, with at least two patients beyond 12 months at the time of the data cut. The company is also studying zugo-cel in combination with the BTK inhibitor pirtobrutinib, based on evidence that BTK inhibitors may potentiate CAR T therapies.

In autoimmune disease, Kulkarni said the company’s goal is to become a leading player. CRISPR Therapeutics has dosed patients in lupus, myositis and scleroderma through the AID-500 trial and has expanded into immune thrombocytopenia and warm autoimmune hemolytic anemia. He said the company has also opened an IND for neuroimmune indications, citing evidence that zugo-cel can enter the central nervous system and eliminate B cells in the spine or brain.

Kulkarni said CRISPR Therapeutics had dosed 14 patients in its autoimmune program as of its first-quarter update and expects to provide additional updates as development progresses.

About CRISPR Therapeutics NASDAQ: CRSP

CRISPR Therapeutics AG is a biopharmaceutical company specializing in the development of gene-editing therapies based on the CRISPR/Cas9 platform. The company applies its proprietary technology to modify genes in human cells, aiming to create durable treatments for a range of serious diseases. Its research and development efforts focus on both ex vivo and in vivo applications, enabling targeted correction or disruption of disease-causing genes.

Among its lead programs is CTX001, an ex vivo edited cell therapy designed to treat sickle cell disease and transfusion-dependent β-thalassemia in collaboration with Vertex Pharmaceuticals.

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