01
Its flagship, Casgevy (exagamglogene autotemcel), is the world's first approved CRISPR/Cas9 gene-editing medicine: a one-time therapy for sickle cell disease and transfusion-dependent beta thalassemia, developed and commercialized with Vertex Pharmaceuticals (which books the sales; CRISPR keeps ~40% of the economics). Casgevy is approved in 39 countries across North America, Europe, and the Middle East, priced at ~$2.2M per treatment, and in July 2026 the FDA expanded its US label to children as young as two. Commercial uptake has been slower than hoped - constrained by cell-collection bottlenecks - but cumulative patient starts have surpassed 500, Vertex booked $76M of Casgevy sales in Q2 2026, and CEO Sam Kulkarni said at Morgan Stanley on September 14, 2026 that initiations now exceed 100 per quarter with most early reimbursement and manufacturing bottlenecks addressed. Beyond Casgevy, CRISPR is pivoting toward in vivo liver gene editing. Lead cardiovascular program CTX310 presented one-year Phase 1a durability data at ESC Congress on August 28, 2026: at the highest dose, mean LDL fell 53% and triglycerides 48% with no treatment-related serious adverse events, and Phase 1b work in severe hypertriglyceridemia is due by year-end. The pipeline also includes allogeneic CAR-T zugo-cel/CTX112 (autoimmune disease and cancer, including a Lilly pirtobrutinib combination), CTX213 (deviceless beta-cell replacement for type 1 diabetes), and CTX611 (SRSD107), a long-acting Factor XI siRNA with Sirius now in Phase 2 versus enoxaparin with topline data still due in H2 2026. Q2 2026 cash and investments were $2,364.4M against a narrowed $91.2M net loss. The stock closed at $52.96 on September 14, 2026 (market cap about $5.16B).
marketbeat.comUpdated Sep 15, 2026
Analysis · 5 min read · Aug 25, 2026The long view on CRISPR Therapeutics: strategy, technology and what to watch02
2026
Convert the 500+ started patients into infusions and grow revenue as the authorized-treatment-center network expands. Casgevy is now described as approved in 39 countries. Revenue booked by Vertex reached $76M in Q2 2026, up 78% quarter-over-quarter and 151% year-over-year. At Morgan Stanley on September 14, 2026, Kulkarni said initiations now exceed 100 per quarter, that most early reimbursement, contracting and manufacturing bottlenecks have been addressed, and that the remaining 6-to-9-month treatment journey is driven more by exchange transfusions and scheduling than by manufacturing. The pediatric ages-2+ label is a tailwind. The next official sales figure is expected with Vertex's early-November Q3 2026 results.
H2 2026
TargetH2 2026 (Phase 1b, Lp(a) update)
Having presented one-year CTX310 (ANGPTL3) Phase 1a follow-up at the European Society of Cardiology Congress on August 28, 2026 (15 participants; highest-dose mean LDL -53% and triglycerides -48% through 12 months; no treatment-related serious adverse events) - simultaneously published in NEJM - CRISPR still owes a separate CTX310 Phase 1b clinical update by year-end 2026, with Kulkarni at Morgan Stanley on September 14 saying the immediate focus is severe hypertriglyceridemia (triglycerides plus biomarkers such as LDL, HbA1c and hepatic fat) to inform pivotal-trial discussions. The company also continues progressing the Lp(a)-lowering program CTX321.
H2 2026
Report additional autoimmune and hematologic data for zugo-cel in H2 2026, advancing its off-the-shelf CAR-T toward registrational studies in B-cell-driven autoimmune disease and B-cell cancers. At Morgan Stanley on September 14, 2026, CEO Sam Kulkarni said CRISPR is prioritizing the rheumatology basket (including systemic sclerosis and myositis), with additional data expected by year-end to help decide whether one or more indications can move toward pivotal development. The pipeline still spans three parallel Phase 1 autoimmune basket trials - rheumatology, hematology, and an autoimmune-neurologic basket covering progressive MS, NMOSD, MOGAD, autoimmune encephalitis, and stiff person syndrome - plus a combination study of zugo-cel with Lilly's pirtobrutinib in aggressive B-cell lymphomas.
H2 2026
Report topline Phase 2 data for CTX611 (SRSD107), the long-acting Factor XI siRNA co-developed with Sirius Therapeutics, in patients undergoing total knee arthroplasty versus enoxaparin. Kulkarni said at Morgan Stanley on September 14, 2026 that the year-end update should include high-level venous-thromboembolism rate comparisons, with more detailed bleeding information later, and that CRISPR will lead Phase 3 development and potential commercialization outside Greater China. No Phase 2 readout has been published as of September 15, 2026.
2026
TargetSunday, November 8, 2026, 10:30 a.m. ET (Poster Session A, abstract 0225)
2026
TargetMonday, November 9, 2026, 8:00 a.m. CST (AHA LBS.05); embargoed until then
2027
At Morgan Stanley on September 14, 2026, Kulkarni said clinical data from CTX340 (in vivo AGT editing for refractory hypertension) and CTX460 (SyNTase editing for alpha-1 antitrypsin deficiency) are expected in 2027, after both programs entered Phase 1 in 2026. He said blood-pressure changes on CTX340 could potentially be observed within days after gene editing.
03
CRISPR Therapeutics presented a late-breaking Phase 1a durability update for CTX310, its in vivo gene-editing therapy targeting ANGPTL3, at the European Society of Cardiology Congress in Munich on August 28. Across 15 participants with average follow-up of at least 60 days, the company reported no treatment-related serious adverse events and no grade 3-or-higher liver-transaminase changes, with a separate Phase 1b clinical update still due in the second half of 2026.
CRISPR Therapeutics reported second-quarter 2026 results on August 3, ending the quarter with $2,364.4 million in cash and investments and narrowing its net loss to $91.2 million from $208.5 million a year earlier. The company said it initiated Phase 1 trials for CTX340, an in vivo editing candidate for refractory hypertension, and CTX460, the first candidate from its new SyNTase editing platform for alpha-1 antitrypsin deficiency, broadening its in vivo pipeline beyond the cardiovascular program CTX310.
The U.S. FDA on July 1 approved expanded use of Casgevy (exagamglogene autotemcel) - the world's first approved CRISPR/Cas9 gene-editing medicine, developed by Vertex Pharmaceuticals with CRISPR Therapeutics - for patients ages 2 and older with sickle cell disease or transfusion-dependent beta thalassemia, lowering the prior age floor of 12. Vertex said the expansion makes roughly 5,500 additional U.S. children newly eligible for the one-time therapy. CRISPR shares rose about 8% and Vertex about 6% on the news; CRISPR retains roughly 40% of Casgevy's economics under the partnership.
04
The world's first approved CRISPR/Cas9 gene-editing medicine (exagamglogene autotemcel). A one-time therapy that edits the BCL11A gene in a patient's own blood stem cells to reactivate fetal hemoglobin, treating sickle cell disease and transfusion-dependent beta thalassemia. Partnered with Vertex Pharmaceuticals.
An in vivo gene-editing therapy delivered by lipid nanoparticles that knocks out the ANGPTL3 gene in the liver to lower triglycerides and LDL cholesterol - a potential one-time treatment for severe dyslipidemia and cardiovascular risk, and the lead of CRISPR's in vivo pipeline.
A long-acting, double-stranded siRNA that targets Factor XI (FXI) messenger RNA to inhibit FXI protein expression, aiming to provide anticoagulant and antithrombotic protection with less bleeding risk than existing anticoagulants, potentially dosed as infrequently as semi-annually. Co-developed with Sirius Therapeutics (50/50 cost and profit share; CRISPR leads U.S. commercialization, Sirius leads Greater China) across a range of thromboembolic indications including atrial fibrillation, venous thromboembolism, and stroke prevention.
An allogeneic, gene-edited anti-CD19 CAR-T cell therapy (zugocaptagene geleucel) being developed for autoimmune diseases such as lupus as well as relapsed/refractory B-cell cancers, using CRISPR editing to make an off-the-shelf product.
A next-generation, deviceless beta-cell replacement candidate for type 1 diabetes, made of unencapsulated precursor islet cells derived from gene-edited, hypoimmune induced pluripotent stem cells (iPSCs), designed for direct administration without an encapsulation device or chronic immunosuppression.
05
($10M collaboration revenue plus grants), beating consensus; Casgevy sales booked by Vertex ($76M in Q2 2026, up 151% YoY), with CRISPR's ~40% economics flowing through the collaboration line. Vertex's Q3 2026 results (which would carry the next Casgevy sales figure) are not expected until early November 2026.
Vertex count as of the July 2026 pediatric-label expansion; locator still lists the network as independently operated ATCs with no later disclosed total
39 countries across North America, Europe, and the Middle East (company count, treating EU member states separately); named authorizations include the US, Canada, UK, EU, Switzerland, Saudi Arabia, Bahrain, Qatar, UAE, and Kuwait. >60,000 eligible patients (~37,000 in North America/Europe, >23,000 Middle East)
$557.2M remaining of a $600.0M 2025 ATM as of June 30, 2026 (Jefferies Open Market Sale Agreement).
AHA Scientific Sessions late-breaking oral Nov. 9, 2026, 8:00 a.m. CST; slides after the session
06
Operating status
Commercial (via partner), pre-profit
Casgevy remains commercial via Vertex (Q2 2026 Vertex-booked sales $76M; CRISPR about 40% economics). Vertex Q3 is scheduled Nov. 2, 2026 after the close; CRISPR has not posted Q3 and StockAnalysis still estimates Nov. 9. Official newsroom newest item is the Oct. 8 AHA late-breaker for CTX310 Phase 1b sHTG on Nov. 9, 8:00 a.m. CST; data embargoed until then. ACR Convergence abstract 0225 remains a Nov. 8 poster slot, not posted slides. CTX611 Phase 2 TKA topline still unpublished. Pipeline page still lists Casgevy, zugo-cel, CTX310, CTX611, CTX321, CTX340, CTX460 and CTX213. Oct. 9 close $53.39, market cap about $5.16B on 96.69M shares.
Casgevy (Vertex collaboration)
Vertex books all Casgevy product revenue (~$116M globally in 2025); CRISPR is entitled to ~40% of the program's net profits and shares ~40% of its costs, recognized through the net collaboration line rather than as product revenue.
Non-recurring cash from the Vertex partnership, including the $900M upfront paid in 2021 and a $200M payment triggered at Casgevy's first approval.
A small stream of research grant revenue (roughly $1–4M per year) recognized as GAAP revenue.
07
How past announced dates turned out.
1/1announced date hit on time
Move new in vivo programs into the clinic
Target 2026 · Resolved Aug 3, 2026
CRISPR Therapeutics confirmed in its Q2 2026 business update that it initiated Phase 1 trials for both next-wave in vivo editing candidates: CTX340 for refractory hypertension and CTX460 (the first candidate from its new SyNTase editing platform) for alpha-1 antitrypsin deficiency.
08
2026
ACR abstract 0225 posts zugo-cel Phase 1 SSc numbers
Meeting abstract 0225 on acrabstracts.org reports Phase 1 zugo-cel data in refractory systemic sclerosis as of a 27 April 2026 cutoff: 6 subjects with at least one month of follow-up (median 2 months, range 1-3). Median modified Rodnan Skin Score fell 10.5 points (36 versus 25.5). Four of six met CRISS-25 by day 28. Two of six had Grade 2 or lower CRS; no Grade 3 or higher CRS and no ICANS were reported. One pulmonary embolism was a serious adverse event considered possibly related. CRISPR still schedules the poster for 8 November 2026 and said slides post after the session. This is an abstract-level table, not the poster and not a registrational readout.
Schedules CTX310 Phase 1b sHTG late-breaker at AHA
On October 8, 2026 CRISPR Therapeutics said Phase 1b data for CTX310 in severe hypertriglyceridemia will be presented as a late-breaking oral at AHA Scientific Sessions 2026 on Monday, November 9, 2026 at 8:00 a.m. CST (session LBS.05). The company said the abstract and presentation remain embargoed until then and that slides will post on crisprtx.com after the session. This is a scheduled readout, not published efficacy tables.
Schedules zugo-cel Phase 1 systemic sclerosis poster at ACR Convergence
On October 2, 2026 CRISPR Therapeutics said a poster of Phase 1 data for zugocabtagene geleucel (zugo-cel), an allogeneic CD19 CAR T, in refractory systemic sclerosis will be presented as abstract 0225 in Poster Session A at ACR Convergence 2026 on Sunday, November 8, 2026 at 10:30 a.m. ET. The company said a copy will be posted after the session. This is a conference slot, not a published efficacy table.
CTX310 one-year durability data presented at ESC Congress
Presents late-breaking Phase 1a one-year follow-up for CTX310 (ANGPTL3 editing) at the European Society of Cardiology Congress in Munich on August 28, 2026, simultaneously published in the New England Journal of Medicine. Across 15 participants, a single-course treatment produced durable lipid lowering through 12 months: at the highest dose, mean reductions were 79% for ANGPTL3 (max 89%), 48% for triglycerides (max 78%), and 53% for LDL (max 84%), with no treatment-related serious adverse events. Cleveland Clinic, which ran the first-in-human trial, reported 52.5% LDL and 47.8% triglyceride lowering at the top dose after one year. A separate CTX310 Phase 1b update in severe hypertriglyceridemia and refractory hypercholesterolemia remains due in H2 2026.
Zugo-cel expands to a third autoimmune basket trial
Initiates a third Phase 1 basket trial for zugo-cel (CTX112) in autoimmune-neurologic disease - covering progressive multiple sclerosis, NMOSD, MOGAD, NMDAR/LGI1 autoimmune encephalitis, and stiff person syndrome - alongside the ongoing rheumatology basket (lupus, systemic sclerosis, inflammatory myositis) and hematology basket (ITP, warm autoimmune hemolytic anemia), broadening the off-the-shelf CAR-T program well beyond its original oncology and lupus scope.
Diabetes pipeline transitions to next-gen CTX213
Reports that durable Phase 1 data from CTX211 (detectable C-peptide 12 months post-implantation) informed a transition to CTX213, a next-generation deviceless beta-cell replacement candidate made of unencapsulated, edited iPSC-derived precursor islet cells, which has shown compelling preclinical efficacy and is progressing toward the clinic.
Zugo-cel plus pirtobrutinib combination study with Lilly
Initiates a combination study evaluating zugo-cel (CTX112) alongside Lilly's pirtobrutinib in aggressive B-cell lymphomas under the companies' clinical-supply collaboration, broadening zugo-cel's oncology development beyond monotherapy ahead of H2 2026 readouts.
Initiates CTX340 and CTX460 Phase 1 trials
Reports in its Q2 2026 business update (August 3, 2026) that it has initiated Phase 1 trials for CTX340, an in vivo editing candidate for refractory hypertension, and CTX460, the first candidate from its new SyNTase editing platform, targeting alpha-1 antitrypsin deficiency - broadening the in vivo pipeline beyond the cardiovascular program CTX310.
2026
FDA expands Casgevy to children ages 2+
In July 2026 the FDA expands Casgevy's US label to patients as young as two years old with sickle cell disease or transfusion-dependent beta thalassemia, adding roughly 5,500 more eligible US children.
$600M convertible notes strengthen balance sheet
Raises ~$585M net through $600M of convertible senior notes due 2031, lifting cash and investments to ~$2.44B and extending its runway across a catalyst-heavy pipeline.
2025
zugo-cel (CTX112) update and Lilly deal
Provides a broad update on zugo-cel (CTX112), its allogeneic CAR-T, with strong autoimmune and lymphoma data (90% overall response rate, 70% complete response in oncology) and a clinical-supply agreement with Eli Lilly.
Positive in vivo cardiovascular data (CTX310)
Reports positive Phase 1 data for CTX310, an in vivo lipid-nanoparticle therapy that edits ANGPTL3 in the liver, showing deep and durable triglyceride and LDL lowering - validating CRISPR's move into one-time in vivo gene editing. Data presented at the AHA meeting and published in NEJM.
First patient dosed in CTX611 (SRSD107) Phase 2 trial
CRISPR Therapeutics and Sirius Therapeutics announce (September 22, 2025) that the first patient has been dosed in a European Phase 2 trial of SRSD107 (CTX611) for prevention of venous thromboembolism in patients undergoing total knee arthroplasty.
Sirius Therapeutics siRNA collaboration (CTX611/SRSD107)
Announces a multi-target collaboration with Sirius Therapeutics (May 19, 2025) to co-develop and co-commercialize SRSD107 (CTX611), a long-acting Factor XI-targeting siRNA for thromboembolic disorders, plus an option on up to two further siRNA targets. CRISPR pays $25M cash and $70M in equity upfront, the companies split costs and profits 50/50, and CRISPR leads U.S. commercialization while Sirius leads Greater China. CRISPR also owes Sirius up to $300M in per-target milestones plus a shared $87.5M milestone pool and tiered royalties. Phase 1 data showed peak FXI activity reductions of >93%, sustained up to 6 months.
2024
2023
2021
2019
First patient dosed with exa-cel (CTX001)
Doses the first patient with CTX001 (later Casgevy) in transfusion-dependent beta thalassemia - one of the first company-sponsored CRISPR/Cas9 therapies to enter the clinic - with landmark results published later that year in the New England Journal of Medicine.
2016
2015
2014
2013
09
$2.1Bdisclosed across 7 rounds with a stated amount
Cumulative disclosed raise · dated rounds
Series A
2014
Versant Ventures
Series A top-up / Series B
2015
SR One, Celgene, NEA, Abingworth
IPO (NASDAQ: CRSP)
2016
Public markets; Bayer
Follow-on offering
2020
Public markets (6.43M shares at $70)
Registered direct offering
2024
EcoR1 Capital and SR One (co-leads), other institutional investors
Convertible senior notes (due 2031)
2026
Debt investors (1.73% coupon)
ATM (2025 program)
Oct 2025
$600M authorized; $557.2M remaining as of June 30, 2026
Public markets via Jefferies LLC Open Market Sale Agreement
