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.
Vertex Pharmaceuticals and CRISPR Therapeutics announced upcoming oral presentations of clinical data for exagamglogene autotemcel (exa-cel) at the 2022 ASH Annual Meeting. This investigational therapy targets sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Key presentations include efficacy and safety data from Phase 3 trials, with plans for regulatory submissions in the U.S., U.K., and EU by early 2023. The studies highlight the quality of life impacts for patients undergoing treatment.
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CRISPR Therapeutics (Nasdaq: CRSP) presented promising data on CTX130™, a CAR-T cell therapy targeting CD70 for relapsed renal cell carcinoma (RCC), showing a 77% disease control rate in heavily pretreated patients. The trial revealed a durable complete response in one patient, indicating potential for durable remissions. Additionally, preclinical findings on anti-CD83 CAR-T cells suggest their effectiveness against acute myeloid leukemia (AML). The ongoing development of next-generation CAR-Ts, including CTX131™, enhances their potency. These results could pave the way for innovative treatments in oncology.
CRISPR Therapeutics (Nasdaq: CRSP) announced a poster presentation on its investigational therapy CTX110® at the ASH 2022 Annual Meeting, scheduled for December 10-13, 2022. The poster will showcase preliminary results from the ongoing Phase 1 CARBON trial, which involves 32 patients with Large B-Cell Lymphoma (LBCL). Updates on the safety and efficacy of CTX110®, a gene-edited CAR-T therapy targeting CD19, will be highlighted. The study has received Regenerative Medicine Advanced Therapy designation from the FDA.
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.