CRISPR Therapeutics AG reports developments in gene-based medicines built around CRISPR/Cas9 gene editing, cell therapy, in vivo editing and siRNA programs. Recurring updates cover CASGEVY, developed with Vertex for severe sickle cell disease and transfusion-dependent beta thalassemia, as well as pipeline work in cardiovascular disease, autoimmune disease, oncology, rare disease and Type 1 diabetes.
Company news also includes financial results, business updates, investor conference presentations, manufacturing and platform disclosures, collaborations such as Sirius Therapeutics, and capital actions involving common shares or convertible senior notes.
CRISPR Therapeutics (CRSP) reported its Q3 2022 financial results, highlighting a net loss of $174.5 million, an increase from $127.2 million in Q3 2021. Collaboration revenue fell to $0.1 million, down from $0.3 million the previous year. R&D expenses surged to $116.6 million, attributed to advancements in clinical programs. The company is advancing its gene-editing therapies, with multiple submissions expected for exa-cel, a therapy for sickle cell disease and beta thalassemia, with FDA review starting in November 2022.
CRISPR Therapeutics announced the resignation of its Chief Operating Officer, Lawrence Klein, effective year-end 2022, to pursue external opportunities. During his tenure, Klein significantly contributed to the company’s strategic direction and leadership. The company is currently searching for a successor while preparing for the regulatory filing of its lead program, exa-cel, with multiple other clinical programs also in progress. CEO Samarth Kulkarni expressed gratitude for Klein's contributions and highlighted the company's solid position for future success.
CRISPR Therapeutics (Nasdaq: CRSP) announced participation in the Society for Immunotherapy of Cancer's 37th Annual Meeting from November 8-12, 2022. Key presentations include:
- Oral Presentation: CTX130 allogeneic CRISPR-Cas9 CAR T cells in advanced clear cell renal cell carcinoma, scheduled for November 10 at 5:37 PM ET.
- Poster Presentation: CRISPR/Cas9 gene-edited anti-CD83 CAR-T cells in GvHD and AML models, on November 10 from 9:00 AM to 9:00 PM.
The company focuses on gene-based medicines for serious diseases using CRISPR/Cas9 technology.
CRISPR Therapeutics (Nasdaq: CRSP) announced participation in the BMO Biopharma Spotlight Series on October 6, 2022, at 9:00 a.m. ET. Members of its senior management team will share insights on gene editing and therapy. A live webcast will be accessible on the company's Investors page, and a replay will be available for 14 days post-event. CRISPR Therapeutics specializes in transformative gene-based medicines for serious diseases, utilizing its proprietary CRISPR/Cas9 technology across various therapeutic programs.
CRISPR Therapeutics (Nasdaq: CRSP) announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to its CTX130™ CAR T cell therapy for treating Mycosis Fungoides and Sézary Syndrome. This designation acknowledges the therapy's potential based on positive clinical data. RMAT designation can expedite development, providing intensive FDA guidance and opportunities for accelerated approval. CTX130, targeting CD70, is evaluated in ongoing trials for safety and efficacy in various malignancies, having also received Orphan Drug status.
Vertex Pharmaceuticals and CRISPR Therapeutics have announced a rolling review of exa-cel by the U.S. FDA for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), with submission starting in November 2022 and completion expected by Q1 2023. Submissions to the EMA and MHRA are also on track for Q4 2022. Exa-cel received multiple designations, including Fast Track and Orphan Drug status. The therapy aims to edit patients' stem cells for improved disease outcomes.
CRISPR Therapeutics (Nasdaq: CRSP) announced its participation in two upcoming investor conferences in September 2022. The first event is the Citi 17th Annual BioPharma Conference on September 7 at 8:50 a.m. ET, followed by the Morgan Stanley 20th Annual Global Healthcare Conference on September 12 at 11:40 a.m. ET. Both presentations will be webcast live and archived for 14 days on the company's website. CRISPR Therapeutics focuses on gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 gene editing technology.
CRISPR Therapeutics announced positive results from its Phase 1 COBALT™-LYM trial for CTX130, an allogeneic CAR-T cell therapy targeting CD70, focusing on treating peripheral T-cell lymphoma (PTCL) and cutaneous T-cell lymphoma (CTCL). At Dose Level 3 and above, the overall response rate was 70%, with a complete response rate of 30%. The safety profile was favorable, with no dose limiting toxicities reported. The trial involved 19 patients, demonstrating significant clinical benefits and responses across various disease compartments.
Vertex Pharmaceuticals and CRISPR Therapeutics unveiled promising new data on exa-cel for treating transfusion-dependent beta thalassemia and severe sickle cell disease. The study involved 75 patients, with up to 37.2 months of follow-up. Key findings include:
- 42 out of 44 patients with beta thalassemia were transfusion-free post-treatment.
- All 31 patients with sickle cell disease experienced cessation of vaso-occlusive crises.
The safety profile aligns with myeloablative conditioning and stem cell transplant protocols.
CRISPR Therapeutics announced participation in the Goldman Sachs 43rd Annual Global Healthcare Conference on June 15, 2022, at 9:20 a.m. PT. Senior management members will present insights on the company's advancements in gene-based medicines for serious diseases, leveraging the CRISPR/Cas9 technology. A live webcast of the presentation will be accessible on their Investors page, with a replay available for 14 days post-event. CRISPR Therapeutics continues to develop therapies across various sectors, including oncology and rare diseases, collaborating with major partners like Bayer and Vertex Pharmaceuticals.