CRISPR Therapeutics Highlights Strategic Priorities and Anticipated 2026 Milestones

Rhea-AI Summary

CRISPR Therapeutics (Nasdaq: CRSP) outlined 2026 strategic priorities and timing for multiple clinical and commercial milestones. The company enters 2026 with approximately $2 billion in cash, cash equivalents, and marketable securities and reported CASGEVY revenue exceeding $100M in 2025 with a nearly three-fold increase in patient initiations versus 2024. Key 2026 items include global CASGEVY commercialization and pediatric regulatory submissions in H1 2026, CTX310 and Lp(a) program updates in H2 2026, topline Phase 2 CTX611 data in H2 2026, and planned trial starts for CTX460 (mid-2026) and CTX340 (H1 2026). Multiple oncology, autoimmune, in vivo editing and siRNA programs advance through the clinic.

Positive

• $2.0B in cash and marketable securities at start of 2026
• CASGEVY revenue exceeded $100M in 2025
• Nearly 3× increase in patient initiations and first cell collections in 2025 vs 2024
• CTX320 (LPA) showed up to 73% reductions in dose escalation
• CTX310 advanced to Phase 1b after Phase 1 showed deep, durable TG and LDL reductions
• Planned 2026 starts or updates: CTX611 Phase 2 topline (H2), CTX460 trial (mid-2026), CTX340 trial (H1 2026)

Negative

• None.

News Market Reaction

+2.71%

1 alert

+2.71% News Effect

On the day this news was published, CRSP gained 2.71%, reflecting a moderate positive market reaction.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

Cash & securities: approximately $2 billion CASGEVY revenue 2025: >$100M CASGEVY patients infused: more than 60 patients +5 more

8 metrics

Cash & securities approximately $2 billion Starting 2026 balance sheet

CASGEVY revenue 2025 >$100M 2025 global CASGEVY sales

CASGEVY patients infused more than 60 patients Patients receiving CASGEVY infusions in 2025

CTX320 LDL(a) reduction up to 73% Dose escalation phase of CTX320 clinical trial

SLE patient remission 9 months First SLE patient maintained drug-free DORIS remission through month 9

SLEDAI-2K score 0 at month 2 Second SLE patient after CAR-T therapy with sustained B cell depletion

CASGEVY approved age 12 years and older SCD or TDT indications in multiple geographies

CASGEVY pediatric range ages 5–11 Planned global regulatory submissions in first half of 2026

Market Reality Check

Price: $53.51 Vol: Volume 2,053,155 is 1.27x...

normal vol

$53.51 Last Close

Volume Volume 2,053,155 is 1.27x the 20-day average of 1,621,590, indicating elevated trading interest pre‑announcement. normal

Technical Shares at $53.84 trade above the 200-day MA of $51.95, yet remain 31.4% below the 52-week high of $78.48 and 79.23% above the 52-week low of $30.04.

Peers on Argus

CRSP fell 4.54% with elevated volume, while peers showed mixed moves: MRUS -7.08...

CRSP fell 4.54% with elevated volume, while peers showed mixed moves: MRUS -7.08%, TGTX -7.26%, PTCT -0.39%, RNA +0.06%, LEGN +2.12%. The momentum scanner did not flag a coordinated sector move, suggesting the reaction was more stock-specific than a broad biotech rotation.

Common Catalyst Multiple peers (e.g., PTCT, LEGN) also issued J.P. Morgan Healthcare Conference updates, pointing to conference-driven news flow across the group.

Historical Context

5 past events · Latest: Jan 05 (Neutral)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Jan 05	Conference presentation	Neutral	+2.6%	J.P. Morgan Healthcare Conference presentation announcement with webcast details.
Dec 22	Clinical update	Positive	+3.7%	Strong zugo-cel efficacy and remission data in autoimmune disease and lymphoma.
Nov 10	Earnings and business	Negative	-1.0%	Wider quarterly loss and high R&D and collaboration expenses despite CASGEVY progress.
Oct 10	Preclinical data	Positive	-2.5%	Promising CTX460 SyNTase AATD data with high gene correction in models.
Oct 02	Partnership news	Positive	+10.1%	Monash Boston Hub launch featuring collaborations including CRISPR Therapeutics.

Pattern Detected

Recent news often aligned with share moves, with only one notable divergence on positive preclinical data.

Recent Company History

Over the last few months, CRISPR Therapeutics has reported several key updates. On Nov 10, 2025, Q3 results highlighted cash and securities of $1,944.1M and continued CASGEVY momentum. Preclinical CTX460 data on Oct 10, 2025 showed >90% mRNA correction in AATD but shares fell, marking a divergence. A Monash collaboration headline on Oct 2, 2025 coincided with a 10.07% gain. The current 2026 milestone-rich outlook builds directly on these clinical, commercial, and partnership developments.

Market Pulse Summary

This announcement outlines a broad 2026 roadmap, with CASGEVY already exceeding $100M in 2025 revenu...

Analysis

This announcement outlines a broad 2026 roadmap, with CASGEVY already exceeding $100M in 2025 revenue and the company starting the year with approximately $2 billion in cash and securities. Multiple programs, including CTX310, CTX611, zugo‑cel, and CTX460, have key data or trial initiations expected in 2026 across cardiovascular, autoimmune, oncology, and rare diseases. Investors may monitor clinical readouts, regulatory submissions for CASGEVY in ages 5–11, and continued execution across the diversified pipeline.

Key Terms

siRNA, Lp(a), Phase 2 clinical trial, IND/CTA-enabling studies, +4 more

8 terms

siRNA medical

"We also added a siRNA pillar through our partnership with Sirius"

Small interfering RNA (siRNA) is a short strand of genetic material that binds to and destroys the messenger RNA that carries instructions for making a specific protein, effectively switching that gene off. Investors care because siRNA is a platform for precise medicines: successful trials or approvals can create high-value drugs, while delivery challenges, manufacturing complexity, patent positions and regulatory risk can sharply affect a biotech company's prospects.

Lp(a) medical

"Updates from the Lp(a) program are expected in 2026."

Lp(a), or lipoprotein(a), is a blood particle that carries cholesterol and a specific protein; think of it as a delivery truck that can drop off harmful cargo along artery walls. High Lp(a) levels are linked to increased risk of heart disease and stroke, so changes in testing, treatments, or clinical results for Lp(a) can affect healthcare costs, drug prospects, and investor expectations for companies working on cardiovascular diagnostics or therapies.

Phase 2 clinical trial medical

"CTX611 ... is in an ongoing Phase 2 clinical trial in patients"

A phase 2 clinical trial is a research study that tests a new medical treatment or drug to see if it is effective and safe for a specific condition. It involves a larger group of people than earlier trials and helps determine whether the treatment should move forward to more extensive testing. For investors, successful phase 2 results can signal potential for future approval and commercial success, while setbacks may indicate challenges ahead.

IND/CTA-enabling studies regulatory

"CTX321 is currently in IND/CTA-enabling studies, with an Lp(a) program update"

Ind/CTA-enabling studies are the preclinical experiments and safety tests a drug or therapy must pass before regulators allow human trials. Think of them as a building inspection and stress test that shows a candidate is safe enough to try in people; passing these studies unlocks the formal applications to start clinical testing, which is a major milestone that reduces technical and regulatory risk and drives investor value and timelines.

CAR-T medical

"following CAR-T therapy. The second SLE patient with a baseline"

CAR-T is a type of cancer therapy that reprograms a patient’s own immune cells to seek and destroy specific cancer cells, like teaching guard dogs a new scent to track intruders. It matters to investors because CAR-T treatments can command high prices, drive strong revenue for successful developers, and carry regulatory and manufacturing risks that can sharply affect a company’s valuation and long-term growth prospects.

C-peptide medical

"CTX211 were promising, demonstrating detectable C-peptide levels 12 months"

C‑peptide is a short protein fragment released at the same time the pancreas produces insulin; because it lingers in the blood longer than insulin itself, clinicians measure C‑peptide levels as a clear sign of how much natural insulin a person still makes. For investors, C‑peptide matters because it’s used as a measurable outcome in diabetes drug and device trials, in diagnostic tests, and by regulators to judge treatment benefit — results that can affect clinical success, approvals, and market value.

angiopoietin-related protein 3 (ANGPTL3) medical

"CTX310, targeting angiopoietin-related protein 3 (ANGPTL3), into Phase 1b"

A liver-made protein that helps control levels of cholesterol and other fats in the blood; think of it as a thermostat that affects how the body stores and clears those fats. It matters to investors because therapies that block or change this protein’s action can lower cardiovascular risk and become valuable drugs or diagnostics, affecting the prospects and valuation of companies working on treatments or tests tied to blood-fat management.

Factor XI (FXI) medical

"CTX611 ... targeting Factor XI (FXI), is in an ongoing Phase 2"

Factor XI (FXI) is a naturally occurring protein in blood that helps amplify the clotting process, making small clots grow into larger ones when the body needs to stop bleeding. Investors care because FXI is a promising drug target for new anticoagulant therapies that aim to prevent harmful clots like strokes or deep vein thrombosis while lowering the risk of bleeding—like turning down a dimmer to reduce sparks without cutting power completely.

AI-generated analysis. Not financial advice.

 -2026 is poised to be a data- and milestone-rich year across the portfolio-

-CASGEVY^® launch acceleration continues, supporting multi-billion-dollar potential-

-Broad pipeline supported by leading gene-editing and siRNA platforms across cardiovascular, autoimmune, oncology and rare diseases-

-Starting 2026 with a strong balance sheet with approximately $2 billion in cash, cash equivalents, and marketable securities-

ZUG, Switzerland and BOSTON, Jan. 12, 2026 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today highlighted its strategic priorities and anticipated 2026 milestones.

"Entering 2026, CRISPR Therapeutics is well positioned, with CASGEVY^® gaining momentum, and multiple programs with encouraging data advancing rapidly through clinical trials across a diverse set of therapeutic areas,” said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. “Across the portfolio, clinical data demonstrated the transformative potential of CTX310™ for patients suffering from severe cardiovascular disease, as well as zugo-cel in oncology and autoimmune diseases. We also added a siRNA pillar through our partnership with Sirius and are advancing our lead siRNA asset, CTX611™ targeting Factor XI through the clinic. Beyond these clinical stage programs, we have multiple additional assets in preclinical development that leverage our best-in-class advanced editing platform. Together, our broad portfolio, strong balance sheet and effective operating model reinforce our confidence as we move into the next phase."

Anticipated Key Milestones in 2026
CRISPR Therapeutics anticipates several key milestones across its portfolio:

• Continued global commercialization and adoption of CASGEVY, with ongoing quarterly updates.
• Global regulatory submissions for CASGEVY in patients ages 5 – 11 are expected to be initiated by Vertex in the first half of 2026.
• Updates from CTX310^® are expected in the second half of 2026.
• Updates from the Lp(a) program are expected in 2026.
• Top-line Phase 2 clinical data from CTX611 in patients undergoing total knee arthroplasty (TKA) are expected in the second half of 2026.
• Updates across autoimmune disease and immuno-oncology for zugocabtagene geleucel (zugo-cel; formerly CTX112™) are expected in the second half of 2026.
• The Company expects to initiate a clinical trial for CTX460 in alpha-1 antitrypsin deficiency (AATD) in mid-2026.
• The Company expects to initiate a clinical trial for CTX340 in refractory hypertension in the first half of 2026.
  
  

Portfolio Highlights and Outlook

Hemoglobinopathies and CASGEVY (exagamglogene autotemcel [exa-cel])

• CASGEVY now approved in the U.S., the United Kingdom, the EU, the Kingdom of Saudi Arabia, the Kingdom of Bahrain, Qatar, Canada, Switzerland, the United Arab Emirates, and Kuwait for patients 12 years and older with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).
• CASGEVY exceeded $100M in revenue in 2025, reflecting more than 60 patients receiving CASGEVY infusions.
• Achieved a nearly three-fold increase in patient initiations and first cell collections in 2025 compared to 2024.
• Regulatory submissions for CASGEVY in patients aged 5-11 years with SCD and TDT are expected in the first half of 2026.
• CASGEVY awarded a Commissioner’s National Priority Voucher for pediatric submissions in SCD and TDT, enabling accelerated regulatory review once submissions are complete.
• Continued development of next-generation conditioning approaches with the potential to expand addressable patient populations for SCD and TDT with CASGEVY.
• Continued development of a lipid nanoparticle (LNP) based in vivo approach for editing hematopoietic stem cells (HSCs), with the potential to address a broader patient population in SCD and TDT.
  
  

In Vivo Liver Editing

CRISPR Therapeutics continues to advance a diversified portfolio of in vivo gene editing programs leveraging its proprietary LNP delivery platform.

• In 2025, CRISPR Therapeutics presented data for CTX310, demonstrating deep and durable reductions of triglycerides (TG) and low-density lipoprotein (LDL) following a single-course intravenous infusion, with a well-tolerated safety profile. Based on the positive Phase 1 results, the Company has advanced CTX310, targeting angiopoietin-related protein 3 (ANGPTL3), into Phase 1b clinical trials, prioritizing development in severe hypertriglyceridemia (sHTG) and refractory hypercholesterolemia. The Company expects to provide an update in the second half of 2026.
• CTX320™, targeting LPA, has demonstrated reductions of up to 73% in the dose escalation phase of the clinical trial. In parallel, the Company is advancing a next-generation LPA program, CTX321™, incorporating an updated guide RNA that demonstrates approximately two-fold greater potency in preclinical testing, while utilizing the same LNP delivery system. CTX321 is currently in IND/CTA-enabling studies, with an Lp(a) program update expected in 2026.
• In addition to its clinical-stage programs, CRISPR Therapeutics continues to advance several preclinical in vivo gene editing candidates, including:
  • CTX460™, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), is the first investigational candidate to emerge from the Company’s SyNTase™ editing platform. The Company expects to initiate a clinical trial for CTX460 in mid-2026.
  • CTX340™, targeting angiotensinogen (AGT) for refractory hypertension, is currently in IND/CTA-enabling studies. The Company expects to initiate a clinical trial for CTX340 in the first half of 2026.
    
    

siRNA-based Programs
CRISPR Therapeutics’ small interfering RNA (siRNA)-based portfolio includes clinical-stage programs in cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics.

• CTX611 (SRSD107), a long-acting siRNA targeting Factor XI (FXI), is in an ongoing Phase 2 clinical trial in patients undergoing total knee arthroplasty (TKA). The Company expects to provide an update in the second half of 2026.
  
• CRISPR Therapeutics will lead global Phase 3 development of CTX611, excluding Greater China. The program targets a range of thromboembolic and clotting-related indications and represents a multi-billion-dollar market opportunity, including arterial fibrillation (AF), venous thromboembolism (VTE), ischemic stroke, cancer-associated thrombosis (CAT), chronic kidney disease (CKD), peripheral vascular disease (PVD), chronic coronary artery disease (CAD).
  
  

Autoimmune Disease and Immuno-Oncology
Zugocabtagene geleucel (zugo-cel; formerly CTX112™) continues to advance in both autoimmune disease and hematologic malignancies.

• In autoimmune disease, Phase 1 clinical trials are ongoing across multiple indications, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis and a second Phase 1 trial in immune thrombocytopenia purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA). The first patient with SLE, refractory to 9 prior therapies with a baseline Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score of 8, has maintained drug-free DORIS clinical remission through month 9 following CAR-T therapy. The second SLE patient with a baseline SLEDAI-2K score of 8, has sustained B cell depletion with SLEDAI-2K score of 0 through month 2 following CAR-T therapy. The Company expects to provide updates in the second half of 2026.
  
• The Phase 1/2 clinical trial of zugo-cel in B-cell malignancies is ongoing. The Company expects to provide updates in the second half of 2026. CRISPR Therapeutics has also established a collaboration and clinical supply agreement with Lilly to evaluate zugo-cel together with pirtobrutinib in aggressive B-cell lymphomas, further expanding the program’s development in oncology.
  
  

Regenerative Medicine
CRISPR Therapeutics continues to advance its regenerative medicine portfolio, including its efforts in diabetes. Clinical data generated from CTX211™ were promising, demonstrating detectable C-peptide levels 12 months after implantation. These data have informed the Company’s approach to hypoimmune cell engineering, supporting a transition to a next-generation candidate, CTX213™. CTX213 has demonstrated compelling preclinical efficacy and is progressing towards the clinic. The Company expects to provide additional updates as development progresses.

About CASGEVY^® (exagamglogene autotemcel [exa-cel])

CASGEVY^® is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world. 

About the CRISPR Therapeutics - Vertex Collaboration for CASGEVY 

CRISPR Therapeutics and Vertex established a strategic research collaboration in 2015 to discover and develop therapies using CRISPR/Cas9 technology to address the underlying genetic causes of human disease. CASGEVY is the first approved therapy to emerge from this collaboration. Under an amended collaboration agreement, Vertex leads global development, manufacturing, and commercialization of CASGEVY and shares program costs and profits worldwide 60/40 with CRISPR Therapeutics. Vertex is the manufacturer and exclusive license holder of CASGEVY. 

About In Vivo Programs

CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) delivery platform to enable gene editing in the liver using both CRISPR/Cas9 and its novel, proprietary SyNTase™ editing technologies. The Company’s in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (ANGPTL3)) and CTX320 (directed towards LPA, the gene encoding apolipoprotein(a) (apo(a)), a major component of lipoprotein(a) [Lp(a)]). Both are validated therapeutic targets for cardiovascular disease. CTX310 and CTX320 are being developed for patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company’s research and preclinical development candidates include: CTX460™, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), CTX340™, targeting AGT for the treatment of refractory hypertension and CTX321, targeting LPA for the treatment of patients with elevated lipoprotein(a).

About CTX611 (SRSD107)

CTX611 is a novel double-stranded siRNA, designed to target the human coagulation FXI messenger RNA and inhibit FXI protein expression. Through modulation of the intrinsic coagulation pathway, CTX611 is intended to provide anticoagulant and antithrombotic effects. Supported by clinical experience to date, CTX611 is being developed as a long-acting FXI inhibitor with the potential to support infrequent, including semi-annual, subcutaneous administration.

About Zugocabtagene Geleucel (zugo-cel; formerly CTX112) 

Zugocabtagene geleucel (zugo-cel) is a wholly-owned, allogeneic chimeric antigen receptor (CAR) T cell therapy product candidate targeting Cluster of Differentiation 19 (CD19), in development for both autoimmune and immuno-oncology indications. Zugo-cel is an off-the-shelf allogeneic CAR-T that utilizes CRISPR Cas9 for targeted gene knockout and CAR insertion for immune evasion and enhanced T effector cell potency. Zugo-cel is given following a standard lymphodepletion regimen without the need for HLA matching. Zugo-cel is being investigated in ongoing clinical trials in adult patients with systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis and in adult patients with relapsed or refractory B-cell malignancies.

About the CRISPR Therapeutics and Sirius Therapeutics Collaboration

CRISPR Therapeutics and Sirius Therapeutics entered into a strategic collaboration in 2025 to develop and commercialize novel small interfering RNA (siRNA) therapies for thromboembolic disorders and other serious diseases. The lead program, CTX611, is a long-acting siRNA targeting FXI. Under the agreement, the companies will co-develop CTX611 and share costs and profits equally. CRISPR Therapeutics will lead commercialization in the U.S., while Sirius will lead in Greater China. The collaboration also provides CRISPR Therapeutics with the option to license up to two additional siRNA programs.

About CRISPR Therapeutics 
Founded over a decade ago, CRISPR Therapeutics is a leading gene editing company focused on developing transformative medicines for serious diseases. The Company has evolved from a pioneering research-stage organization into an industry leader, marking a historic milestone with the approval of CASGEVY^® (exagamglogene autotemcel [exa-cel]), the world’s first CRISPR-based therapy, approved for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia. CRISPR Therapeutics is advancing a broad and diversified pipeline across hemoglobinopathies, oncology, regenerative medicine, cardiovascular and autoimmune, and rare diseases. The Company continues to expand its leadership in gene editing through the development of SyNTase™ editing, a novel and proprietary gene-editing platform designed to enable precise, efficient, and scalable gene correction. To accelerate and expand its impact, CRISPR Therapeutics has established strategic collaborations with leading biopharmaceutical partners, including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit www.crisprtx.com.

CRISPR THERAPEUTICS^® standard character mark and design logo, SyNTase™, CTX112™, CTX211™, CTX213™, CTX310^®, CTX320™, CTX321™, CTX340™, CTX460™ and CTX611™ are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY^® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners. 

CRISPR Special Note Regarding Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements made by Dr. Kulkarni in this press release, as well as regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, manufacturing capabilities, status of such studies and trials, potential expansion into new indications and expectations regarding data, safety and efficacy generally; (ii) data included in this press release, as well as the ability to use data from ongoing and planned clinical trials for the design and initiation of further clinical trials; (iii) CRISPR Therapeutics strategy, goals, anticipated financial performance and the sufficiency of its cash resources; (iv) plans and expectations for the commercialization of, and anticipated benefits of, CASGEVY, including anticipated patient access to CASGEVY; (v) regulatory submissions and authorizations, including timelines for and expectations regarding regulatory agency decisions; (vi) the expected benefits of its collaborations; and (vii) the therapeutic value, development, and commercial potential of gene editing technologies and therapies, including CRISPR/Cas9 and SyNTase, as well as other technologies. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading “Risk Factors” in its most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

This press release also contains information regarding our industry, our business and the markets for certain of our product candidates, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Unless otherwise expressly stated, we obtained this industry, business, market and other data from market research firms and other third parties, including medical publications, government data and similar sources. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information. This press release discusses investigational therapies and is not intended to convey conclusions about efficacy or safety as to those investigational therapies or uses of such investigational therapies. There is no guarantee that any investigational therapy will successfully complete clinical development or gain approval from applicable regulatory authorities. 

Investor Contact: 
+1-617-307-7503 
ir@crisprtx.com 

Media Contact: 
+1-617-315-4493 
media@crisprtx.com

FAQ

What 2026 milestones did CRSP announce for CASGEVY commercialization?

CRSP expects continued global commercialization and quarterly updates, with pediatric regulatory submissions for ages 5–11 initiated by Vertex in H1 2026.

When will CRSP report topline data for CTX611 (CRSP:CRSP)?

Top-line Phase 2 clinical data for CTX611 in total knee arthroplasty patients are expected in the second half of 2026.

What cash position did CRSP report entering 2026 and why does it matter to investors?

CRSP reported approximately $2 billion in cash, cash equivalents and marketable securities, supporting clinical development and commercial launch activities.

What updates are expected for the in vivo programs CTX310 and Lp(a) in 2026 for CRSP?

CRSP expects an update on CTX310 in H2 2026 and an Lp(a) program update (including CTX321 IND/CTA progress) during 2026.

Which CRSP preclinical programs are scheduled to start clinical trials in 2026?

CRSP expects to initiate CTX460 for AATD in mid-2026 and CTX340 for refractory hypertension in the first half of 2026.

How is CRSP advancing siRNA programs and what is the development plan for CTX611?

CRSP added an siRNA pillar via a partnership with Sirius; CRSP will lead global Phase 3 development of CTX611 (excluding Greater China) targeting multiple thromboembolic indications.