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 and CTX310. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
CTX112 - FDA Regulatory Timeline and 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 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.
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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:
- 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.
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