CRISPR Therapeutics NASDAQ: CRSP CEO Sam Kulkarni told investors at the Needham Healthcare Conference that the company is moving into what he described as a “second phase” following the launch of Casgevy, with a broader pipeline spanning cardiovascular, autoimmune disease, oncology, regenerative medicine, and rare disease programs.
Kulkarni, speaking with Needham Senior Analyst Gil Blum, said the company now has “potentially six programs that would read out in the next six-12 months,” as it looks to “parlay” from a first approved product into multiple clinical readouts using both ex vivo and in vivo gene editing approaches.
Casgevy launch: building an installed base and tracking leading indicators
Blum noted Casgevy generated over $100 million in sales last year, with a “sizable chunk” in the fourth quarter, and asked what to expect in 2026. Kulkarni said the launch differs from traditional medicines because it requires a growing network of specialized sites capable of delivering the therapy.
“This requires an installed base build,” Kulkarni said, describing authorized treatment centers as both a practical requirement and a competitive moat. He said there are now “over 75 authorized treatment centers around the world” established by partner Vertex Pharmaceuticals in approved jurisdictions.
Kulkarni pointed to patient initiations, collections, and infusions as key markers, calling initiations “a leading indicator” for eventual infusions. He said “over 100 patients” were initiated in the last quarter and argued that, if those initiations progress through collection and infusion, Casgevy represents a “multibillion-dollar opportunity.”
On the timing of profits and how revenue will flow through CRISPR Therapeutics’ financial statements, Kulkarni said the company has not commented on when the Casgevy franchise will become profitable. He added that collaboration expense currently includes investments beyond the marketed product, including work on “gentler conditioning” and “in vivo editing for sickle cell and thalassemia,” making it difficult to disaggregate profitability. Still, he said the company is “confident that the franchise will turn profitable in the not-too-distant future” and could meaningfully support enterprise-wide spending.
On access, Kulkarni said coverage has been “very heartening” across regions, citing “the CMMI pilot” in the U.S. and what he characterized as broad coverage outside the U.S. He framed Casgevy as an option that can let patients “return to normalcy” compared with alternatives such as haplo transplant when matches are unavailable or medicines like hydroxyurea that do not change underlying disease.
In vivo gene editing: pushing back on the chronic-therapy argument
Addressing a common investor concern—whether gene editing should be limited to severe or fatal diseases—Kulkarni argued the question differs from what physicians are asking. He said gene editing’s durability, lack of compliance burden, and more consistent pharmacologic effect could make it preferable to chronic therapies in some settings.
Using a media example of a father-son duo with severe lipid disorders, Kulkarni said that after “one shot of gene editing,” lipid measures looked “much better than anything they’ve seen before,” and that permanent editing avoids a “sawtooth effect” associated with dosing intervals in some chronic medicines.
He also suggested pricing dynamics could favor gene editing over time, contending it could be priced around “four or five years’ worth of siRNA therapy” compared with potentially “25 years of siRNA therapy” for some patients. Kulkarni said the debate may shift from “why gene editing” to “why not gene edit,” while noting that uptake will likely depend on safety and how adoption expands from more severe to broader patient populations.
Platform priorities: extrahepatic delivery and gene correction/insertion
Kulkarni said CRISPR Therapeutics is increasing investment in platform capabilities as others reduce spending. He highlighted two areas of focus:
- Extrahepatic delivery: Kulkarni said the company has shown “promising animal data for bone marrow editing with LNPs” and is working on conjugated LNPs for other organs, “including CNS delivery.”
- Gene correction and gene insertion: He said the platform is moving from gene disruption toward correcting single base-pair anomalies and ultimately “insert entire genes into various tissues or various cell types.”
He argued these advances could open a range of opportunities across rare diseases affecting muscle, the central nervous system, and other organs.
Cardiovascular franchise: ANGPTL3, Lp(a), hypertension, and factor XI
Kulkarni described cardiovascular as one of two major franchises the company is building. On CTX310, which targets ANGPTL3, he outlined how the addressable market could expand as data accumulate, beginning with smaller, more straightforward populations and potentially moving into broader dyslipidemia groups.
He cited multiple patient segments, including:
- Homozygous familial hypercholesterolemia (HoFH): “about 1,000-2,000 patients in the U.S.” as a potential path for phase III and approval.
- Severe hypertriglyceridemia (sHTG): “about 3 million” people in the U.S., with “nearly a third” experiencing acute pancreatitis; Kulkarni suggested a subset of about “1 million” patients could potentially support an approval path with pancreatitis reduction as an endpoint.
- Mixed dyslipidemias: including heterozygous FH (“about 1 million” patients), refractory hypercholesterolemia, and other groups that could amount to “several million more” potentially eligible patients.
He added that ANGPTL3 functions like a “built-in bispecific” by addressing both LDL and triglycerides.
On near-term disclosures, Kulkarni said the company previously presented dose escalation data for 15 patients at the American Heart Association meeting and expects additional data in the second half of the year from a phase 1b effort intended to evaluate effects in specific subpopulations and help select a phase III dose. He said the company plans to take the data to regulators and referenced the potential for designations such as RMAT to facilitate development discussions.
On lipoprotein(a), Kulkarni called Novartis’ pelacarsen HORIZON readout “one of the most anticipated” in pharma and said the field is awaiting whether pharmacologic Lp(a) reduction translates to reduced cardiovascular events. He said CRISPR Therapeutics has two Lp(a)-lowering assets, CTX320 (dose escalation completed) and CTX321 (being advanced in parallel), and that decisions on potency selection and phase II cutoff levels will be informed by HORIZON results and subgroup analyses.
For treatment-resistant hypertension, Kulkarni said the company is “on track” to start a clinical trial in the first half of the year for CTX340, which targets angiotensinogen. He referenced siRNA trial data from Roche and Alnylam showing systolic blood pressure reductions “anywhere from 9%-19%” across subpopulations, and said gene editing could offer a consistent baseline effect without end-of-dose variability. When asked about the bar for early clinical results, he said “high single digits or low double-digit” systolic reductions would be meaningful for patients with significantly elevated blood pressure.
Kulkarni also discussed CTX611, the company’s siRNA program targeting factor XI in anticoagulation. He said factor XI is not a target the company would want to “permanently edit,” making siRNA more appropriate for use “when needed,” such as after surgeries, potentially in secondary stroke prevention, or in conditions like atrial fibrillation. He described a planned approach including a total knee arthroplasty (TKA) study, where DVT prevention is a key endpoint and where current options such as enoxaparin still leave a “one in five chance” of DVTs. Kulkarni said the TKA trial can help position the therapy versus other factor XI approaches and inform phase III strategy, and noted the company has guided to data in the second half of 2026.
Autoimmune and oncology: zugo-cel expansion and upcoming updates
Kulkarni said CRISPR Therapeutics is committed to an autoimmune franchise and highlighted zugo-cel as a program spanning autoimmune disease and oncology, including a combination strategy with pirtobrutinib in oncology. Commenting on Allogene’s newly discussed non-futility data in frontline DLBCL with MRD positivity, Kulkarni said the paradigm suggests cell therapies can reach “places that the biologics may not,” and argued a similar concept could apply to autoimmune disease.
Blum asked about cytokine release syndrome (CRS) risk in autoimmune settings, noting roughly 70% grade 3 CRS in oncology studies. Kulkarni said he does not expect the same CRS levels in autoimmune disease because B-cell burden is lower than in cancer, and because the doses being explored in autoimmune disease are lower than in oncology.
On autoimmune indications, Kulkarni said the company is expanding its “rheum basket” from initial focus areas—SLE, myositis, and scleroderma—into ITP and AIHA, and is evaluating additional indications “particularly in the CNS realm.” He said the company plans a zugo-cel update in the second half of this year across autoimmune and oncology and hopes to have regulatory clarity by then on what pivotal trials could look like in some indications.
In regenerative medicine, Kulkarni said the company is advancing CTX213, an iPSC-derived multi-edited cell program, but has not specified a clinical start date; he said it is “more likely to be next year.” He added that the company believes its immune-evasion and robustness edits may be best in class, while acknowledging multiple companies are pursuing similar approaches and that performance will be determined by data.
Looking ahead, Kulkarni said the company has “seven assets outside of Casgevy” and expects data for “at least six in the next 12 months.” He also noted a rare disease program, CTX460 for alpha-1 antitrypsin deficiency, is “going into the clinic soon.” Kulkarni said the company believes it has a strong balance sheet and operational efficiency that allows investment across multiple areas as others retrench.
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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