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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 significant advancements in its product pipeline, including completed regulatory submissions for exa-cel for transfusion-dependent beta thalassemia and sickle cell disease in Europe, with a U.S. BLA submission expected by Q1 2023. The company is also advancing clinical trials for CAR T therapies CTX110 and CTX130, along with new candidates CTX112 and CTX131. Financially, CRISPR ended 2022 with $1.87 billion in cash but reported a net loss of $650.2 million for the year, marking a significant contrast to the $377.7 million net income in 2021.
CRISPR Therapeutics (Nasdaq: CRSP) announced participation in two upcoming investor conferences in February 2023. The SVB Securities Global Biopharma Conference will take place on February 15, 2023, at 1:40 p.m. ET, followed by Citi’s 2023 Virtual Oncology Leadership Summit on February 22, 2023, at 1:00 p.m. ET. These sessions will feature live webcasts available on the company's website with replays accessible for 14 days post-event. CRISPR Therapeutics focuses on developing gene-based medicines for serious diseases using the CRISPR/Cas9 platform, establishing partnerships with companies like Bayer and Vertex Pharmaceuticals.
CRISPR Therapeutics (Nasdaq: CRSP) announced that its senior management will participate in B. Riley Securities’ 3rd Annual Oncology Conference on January 18, 2023, at 10:30 a.m. ET. The event will showcase the company's commitment to developing transformative gene-based therapies for serious diseases using its proprietary CRISPR/Cas9 platform. Interested parties can register for a live webcast or replay on the company's website under the 'Events & Presentations' section.
CRISPR Therapeutics (Nasdaq: CRSP) has appointed Alex Harding, M.D., M.B.A., as Senior Vice President and Head of Business Development, effective January 5, 2023. Dr. Harding has significant experience in biopharma, previously serving at Remix Therapeutics, where he was pivotal in raising over $150M and forming strategic collaborations. His expertise is expected to support CRISPR's growth and enhance its business development operations, as stated by CEO Samarth Kulkarni. CRISPR focuses on gene-based therapies utilizing its CRISPR/Cas9 platform for serious diseases.
CRISPR Therapeutics has provided an update from its Phase 1 CARBON trial for CTX110, a CAR T cell therapy targeting CD19+ B-cell malignancies. At the recent ASH Annual Meeting, data confirmed that CTX110 can lead to long-term complete remissions in heavily pre-treated Large B-cell Lymphoma patients, with three patients maintaining remission for over two years. The trial suggests a positive safety profile and beneficial effects of consolidation dosing, prompting the initiation of a Phase 2 registrational trial in collaboration with regulatory agencies.
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.