This section highlights FDA-related milestones and regulatory updates for drugs developed by CRISPR Therapeutics (CRSP).
Over the past two years, CRISPR Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
CTX112, CTX310, SyNTase, and SRSD107. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
CTX112 FDA Regulatory Events
CTX112 is a drug developed by CRISPR Therapeutics for the following indication: In relapsed or refractory (R/R) CD19-positive B-cell malignancies.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- CTX112
- Announced Date:
- December 22, 2025
- Indication:
- In relapsed or refractory (R/R) CD19-positive B-cell malignancies
Announcement
CRISPR Therapeutics provided updates on zugocaptagene geleucel (zugo-cel), formerly known as CTX112™, its investigational allogeneic CAR T targeting CD19, in development for autoimmune disease and hematologic malignancies.
AI Summary
CRISPR Therapeutics updated progress on zugocaptagene geleucel (zugo-cel), an off‑the‑shelf, CRISPR‑edited allogeneic CAR T that targets CD19. In autoimmune trials, four patients treated at the 100 million‑cell dose showed rapid, deep B‑cell depletion lasting at least 28 days and all had meaningful clinical improvement by Day 28. The first systemic lupus erythematosus (SLE) patient, refractory to many prior therapies, remained in DORIS remission through Month 6. Treatment at the 100M dose has been well tolerated with no high‑grade CRS or ICANS observed. A new Phase 1 basket trial was started for refractory ITP and warm autoimmune hemolytic anemia, and more autoimmune updates are expected in the second half of 2026.
In blood cancers, the recommended Phase 2 dose (RP2D) is 600 million cells. At that dose in relapsed/refractory large B‑cell lymphoma, zugo‑cel showed 90% overall response (9/10) and 70% complete response (7/10), with 67% (2/3) of evaluable patients remaining in CR at 12 months. Peak CAR T expansion was about 1,700 cells/µL (≈4× higher vs 300M). At the RP2D, rates of Grade 3 CRS, ICANS, and serious infections were 17%, 17%, and 8%, respectively. CRISPR also announced a collaboration with Lilly to test zugo‑cel plus pirtobrutinib in aggressive B‑cell lymphomas.
Read Announcement- Drug:
- CTX112
- Announced Date:
- December 22, 2025
- Indication:
- In relapsed or refractory (R/R) CD19-positive B-cell malignancies
Announcement
CRISPR Therapeutics today provided updates on zugocaptagene geleucel (zugo-cel), formerly known as CTX112™, its investigational allogeneic CAR T targeting CD19, in development for autoimmune disease and hematologic malignancies.
AI Summary
CRISPR Therapeutics provided updates on zugocaptagene geleucel (zugo-cel, formerly CTX112), an investigational allogeneic CAR T therapy targeting CD19 for autoimmune diseases and blood cancers. In autoimmune trials, four patients treated at the 100 million cell dose showed rapid, deep B‑cell depletion lasting at least 28 days, with repopulating B cells shifting toward a naive repertoire. All four had significant clinical improvement at Day 28, and the first systemic lupus erythematosus (SLE) patient — refractory to nine prior therapies — remained in DORIS remission through Month 6. The 100M dose has been well tolerated with no high‑grade CRS or ICANS observed. A Phase 1 basket study in refractory ITP and warm autoimmune hemolytic anemia (wAIHA) has been initiated.
In relapsed/refractory large B‑cell lymphoma, the recommended Phase 2 dose is 600 million cells. At that dose the ORR was 90% (9/10) and CRR 70% (7/10); among patients with 12‑month follow‑up, 67% (2/3) remained in complete response. Peak CAR T expansion reached ~1,700 cells/µL at 600M, about four times higher than at 300M. Grade 3 CRS and ICANS rates were 17% each and serious infections 8% at the RP2D (n=12). CRISPR also announced a collaboration with Lilly to test zugo‑cel plus pirtobrutinib, with more data expected in H2 2026.
Read Announcement- Drug:
- CTX112
- Announced Date:
- December 9, 2024
- Indication:
- In relapsed or refractory (R/R) CD19-positive B-cell malignancies
Announcement
CRISPR Therapeutics today presented data from the Company's ongoing Phase 1/2 dose escalation clinical trial evaluating the safety and efficacy of CTX112™, a next-generation CD19 allogeneic CAR T cell therapy, in relapsed or refractory (R/R) CD19-positive B-cell malignancies at the 2024 American Society of Hematology (ASH) Annual Meeting.
AI Summary
At the 2024 American Society of Hematology Annual Meeting, CRISPR Therapeutics shared promising early data from its ongoing Phase 1/2 dose escalation trial of CTX112™, a next-generation CD19 allogeneic CAR T cell therapy, for relapsed or refractory CD19-positive B-cell malignancies. The study, which used a standard lymphodepletion protocol, showed that CTX112 has a well-tolerated safety profile and produced encouraging clinical responses, including complete remissions, in heavily pre-treated patients. Dose-dependent efficacy was noted, with higher dose levels resulting in greater CAR T cell expansion and potentially longer-lasting responses.
Additionally, the favorable safety findings and robust responses support CTX112’s potential as an off-the-shelf treatment option. These results bolster hopes of addressing unmet needs in aggressive B-cell malignancies and pave the way for further clinical development and optimization.
Read Announcement
CTX310 FDA Regulatory Timeline and Events
CTX310 is a drug developed by CRISPR Therapeutics for the following indication: Treatments for Cardiovascular Diseases.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- CTX310
- Announced Date:
- November 8, 2025
- Indication:
- Treatments for Cardiovascular Diseases
Announcement
RISPR Therapeutics announced positive Phase 1 data from its ongoing clinical trial evaluating CTX310®, an investigational, in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3. A single-course treatment with CTX310 produced dose-dependent, durable reductions in circulating ANGPTL3 with a mean reduction from baseline of -73% (maximum -89%), a mean reduction in triglycerides (TG) of -55% (maximum -84%) and a mean reduction of low-density lipoprotein (LDL) of -49% (maximum -87%) at the highest dose.
AI Summary
CRISPR Therapeutics reported positive Phase 1 results for CTX310, an investigational in vivo CRISPR/Cas9 therapy that edits ANGPTL3 in the liver. A single IV course produced dose-dependent, durable reductions in ANGPTL3 (mean −73%, up to −89%), triglycerides (mean −55%, up to −84%) and LDL cholesterol (mean −49%, up to −87%) at the highest dose. In participants with baseline triglycerides >150 mg/dL, therapeutic doses produced a mean 60% TG reduction.
CTX310 was generally well tolerated: no treatment-related serious adverse events and no ≥Grade 3 liver transaminase elevations were reported. Adverse events were mostly mild to moderate and resolved. The data were presented at the AHA Scientific Sessions and published in The New England Journal of Medicine. CRISPR Therapeutics is advancing CTX310 into further studies, positioning a one-time gene-editing approach as a potential durable treatment for severe or refractory lipid disorders.
Read Announcement- Drug:
- CTX310
- Announced Date:
- September 9, 2025
- Indication:
- Treatments for Cardiovascular Diseases
Announcement
CRISPR Therapeutics announced that a late-breaking oral presentation highlighting the Company's Phase 1 clinical data of its investigational CRISPR/Cas9 in vivo gene editing therapy, CTX310™, targeting angiopoietin-related protein 3 (ANGPTL3) for cardiovascular and cardiometabolic disease, will be presented at the American Heart Association (AHA) Scientific Sessions 2025, taking place November 7 – 10, 2025, in New Orleans, Louisiana.
AI Summary
CRISPR Therapeutics announced that it will share Phase 1 clinical data for its investigational in vivo CRISPR/Cas9 therapy, CTX310™, at the American Heart Association Scientific Sessions 2025 in New Orleans, November 7–10. CTX310 is designed to edit the ANGPTL3 gene in the liver to reduce risk factors for cardiovascular and cardiometabolic disease.
The data will be featured in a late-breaking oral presentation titled “First-in-Human Phase 1 Clinical Trial of a CRISPR-Cas9 Gene Editing Therapy Targeting ANGPTL3” on Saturday, November 8, at 8:30 a.m. CST (Abstract #4392851). Results will remain under embargo until the session begins. After the presentation, CRISPR Therapeutics will post slides and details at www.crisprtx.com. This event highlights the company’s progress in applying CRISPR gene editing to treat serious heart and metabolic conditions.
Read Announcement- Drug:
- CTX310
- Announced Date:
- June 26, 2025
- Indication:
- Treatments for Cardiovascular Diseases
Announcement
CRISPR Therapeutics announced updates across its in vivo cardiovascular disease programs.
AI Summary
CRISPR Therapeutics has provided important updates on its in vivo cardiovascular disease programs. The company presented new Phase 1 clinical data for CTX310™, which targets the ANGPTL3 gene. The trial showed dose-dependent reductions in triglycerides (up to 82%) and low-density lipoprotein (up to 86%), with a favorable safety profile. The complete Phase 1 data for CTX310™ is set to be shared at a medical meeting later this year.
In addition, progress is underway with CTX320™, which targets the LPA gene. The dose-finding study continues, with updated results now expected in the first half of 2026. CRISPR Therapeutics is also advancing CTX340™, a preclinical program targeting angiotensinogen for treating refractory hypertension, and it is moving towards IND/CTA filings. Overall, these developments highlight the company’s commitment to advancing innovative gene-based therapies for serious cardiovascular diseases.
Read Announcement
SyNTase FDA Regulatory Events
SyNTase is a drug developed by CRISPR Therapeutics for the following indication: Gene Editing Technology.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- SyNTase
- Announced Date:
- October 10, 2025
- Indication:
- Gene Editing Technology
Announcement
CRISPR Therapeutics today reported new preclinical data from its novel SyNTase™ gene editing platform for the treatment of Alpha-1 Antitrypsin Deficiency (AATD).
Read Announcement- Drug:
- SyNTase
- Announced Date:
- October 1, 2025
- Indication:
- Gene Editing Technology
Announcement
CRISPR Therapeutics To Present Preclinical Data On Alpha-1 Antitrypsin Deficiency Utilizing Novel SyNTase Gene Editing Technology
AI Summary
CRISPR Therapeutics announced that its abstract on SyNTase gene editing for Alpha-1 Antitrypsin Deficiency (AATD) was accepted for oral presentation at the European Society of Gene and Cell Therapy Annual Congress. The new SyNTase platform combines compact Cas9 proteins and engineered polymerases for more precise, efficient gene correction.
In cell studies, SyNTase achieved up to 95% correction of the AATD mutation (SERPINA1-E342K) with under 0.5% off-target effects. In a humanized mouse model, a single low-dose intravenous injection of lipid nanoparticle–encapsulated editors drove efficient gene repair and showed a good safety profile.
In a custom rat model, the approach delivered over 70% mRNA correction and more than three-fold increase in serum AAT, surpassing protective levels. These results offer proof-of-concept for a potentially best-in-class, single-dose treatment for AATD and highlight SyNTase’s promise for monogenic diseases.
Read Announcement
SRSD107 FDA Regulatory Events
SRSD107 is a drug developed by CRISPR Therapeutics for the following indication: for Thromboembolic Disorders.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- SRSD107
- Announced Date:
- September 22, 2025
- Indication:
- for Thromboembolic Disorders
Announcement
CRISPR Therapeutics announced that the first patient has been dosed in a Phase 2 clinical trial of SRSD107, a next-generation, long-acting Factor XI (FXI) siRNA for the prevention of venous thromboembolism (VTE) in patients undergoing total knee arthroplasty (TKA). SRSD107 is being co-developed by CRISPR Therapeutics and Sirius Therapeutics as part of a strategic collaboration to advance innovative treatments for cardiovascular and clotting-related diseases.
AI Summary
CRISPR Therapeutics and Sirius Therapeutics announced that the first patient has been dosed in a Phase 2 clinical trial of SRSD107, a next-generation, long-acting Factor XI (FXI) siRNA. The study will evaluate SRSD107’s ability to prevent venous thromboembolism (VTE) in patients undergoing total knee arthroplasty (TKA). This collaboration aims to develop safer, more convenient anticoagulant therapies with less bleeding risk and the potential for infrequent dosing.
The randomized, multicenter, global trial will assess SRSD107’s safety, efficacy and pharmacological profile and help guide dose selection for pivotal studies. By selectively inhibiting FXI—a key driver of pathological thrombosis—SRSD107 is designed to reduce clot formation while preserving normal blood-clotting function. Earlier Phase 1 trials showed over 93% reduction in FXI levels and sustained effects up to six months after a single dose.
With its sustained, reversible action and semi-annual dosing potential, SRSD107 could offer a differentiated approach for patients at high thrombotic risk, including those undergoing major orthopedic surgery.
Read Announcement
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