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Sep 30, 2023
CRISPR Therapeutics Q3 2023 Earnings Report
Reported financial results and provided a business update.
Key Takeaways
CRISPR Therapeutics reported its Q3 2023 financial results, showing a net loss of $112.2 million compared to a net loss of $174.5 million for the third quarter of 2022. The company's cash, cash equivalents, and marketable securities totaled $1,739.8 million as of September 30, 2023. Significant progress was noted across its clinical pipeline, especially with exa-cel awaiting FDA decision.
Advisory Committee meeting completed for exa-cel for severe sickle cell disease, with a PDUFA target action date of December 8, 2023.
Exa-cel assigned PDUFA target action date of March 30, 2024 for transfusion-dependent beta thalassemia.
Clinical trials are ongoing for CAR T product candidates, CTX110 and CTX112, targeting CD19 in B-cell malignancies.
Phase 1 clinical trial initiated for CTX310, targeting angiopoietin-related protein 3 (ANGPTL3).
CRISPR Therapeutics
CRISPR Therapeutics
Forward Guidance
CRISPR Therapeutics is advancing its pipeline with key milestones expected in the near term, particularly the PDUFA date for exa-cel and the progression of in vivo programs. They are also advancing additional programs utilizing in vivo delivery to address both rare and common diseases.
Positive Outlook
- Potential approval of exa-cel could bring a transformative therapy to patients living with sickle cell disease.
- Advancing multiple in vivo programs directed towards cardiovascular indications and beyond.
- CTX310 and CTX320 have the potential to shift the treatment paradigm for ASCVD with a single-dose, potentially life-long durable editing approach.
- Received a new grant from the Bill & Melinda Gates Foundation to research in vivo gene editing of hematopoietic stem and progenitor cells (HSPCs).
- Advancing additional programs utilizing in vivo delivery to address both rare and common diseases.
Challenges Ahead
- Efficacy and safety results from ongoing clinical trials, including of exa-cel, will not continue or be repeated in ongoing or planned clinical trials or may not support regulatory submissions.
- The FDA or other regulatory authorities may not approve exa-cel on a timely basis or at all.
- Adequate pricing or reimbursement may not be secured to support continued development or commercialization of exa-cel following regulatory approval.
- Clinical trial results may not be favorable.
- Future competitive or other market factors may adversely affect the commercial potential for its product candidates.