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 (Nasdaq: CRSP) announced participation in the Guggenheim Healthcare Talks 2021 Oncology Day on February 12, 2021, at 3:30 p.m. ET. Senior management team members will represent the company during this event. A live webcast is available on the company's Investors website, with a replay accessible for 14 days post-event. CRISPR Therapeutics focuses on developing transformative gene-based medicines utilizing its proprietary CRISPR/Cas9 technology, with initiatives across various disease areas such as oncology and hemoglobinopathies.
CRISPR Therapeutics (Nasdaq: CRSP) appointed Philippe Drouet as Chief Commercial Officer on February 1, 2021. Drouet, with over 20 years in global pharmaceutical marketing, is expected to drive the company’s commercialization efforts. His previous roles include Senior VP at Merck, where he led the launch of Keytruda, and President of Hospira’s U.S. division. The CEO emphasized Drouet's leadership will be crucial as CRISPR advances its gene-based therapies across oncology and hematology.
CRISPR Therapeutics (CRSP) announced a grant from the Bill & Melinda Gates Foundation to support research on CRISPR/Cas9-based therapies for HIV. The funding aims to enhance in vivo gene editing capabilities, allowing broader access to transformative treatments, particularly in resource-limited settings. This grant builds upon CRISPR's expertise in editing hematopoietic stem cells and aligns with its commitment to global health.
CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals reported promising data for CTX001, a CRISPR/Cas9 gene-editing therapy, at the ASH Meeting. In beta thalassemia, all seven patients were transfusion independent after 3 to 18 months post-infusion. For sickle cell disease, three patients experienced no vaso-occlusive crises within 3 to 15 months. The New England Journal of Medicine published a peer-reviewed article detailing these findings. CTX001 is being advanced through Phase 1/2 trials and has received several FDA designations to expedite its development.
CRISPR Therapeutics and Vertex Pharmaceuticals will host an investor webcast on December 9, 2020, at 8:00 a.m. ET. The event will focus on clinical data from ongoing Phase 1/2 trials of CTX001, a gene-editing therapy for sickle cell disease and beta thalassemia, which will be presented at the ASH Meeting. Notable speakers include representatives from both companies and Dr. Haydar Frangoul. The webcast will be accessible via the companies' websites, and an archived version will be available for 30 days.
The collaboration aims to develop gene-based treatments using CRISPR/Cas9 technology.
CRISPR Therapeutics (Nasdaq: CRSP) announced participation at the Piper Sandler 32nd Annual Virtual Healthcare Conference on December 2, 2020, at 9:30 a.m. ET. Senior management will represent the company, which specializes in gene-based medicines for severe diseases using its CRISPR/Cas9 technology. A live webcast will be available on the company’s website, with a replay accessible for 14 days post-event. CRISPR Therapeutics holds collaborations with Bayer, Vertex Pharmaceuticals, and ViaCyte, and operates headquarters in Zug, Switzerland, and a U.S. subsidiary in Cambridge, Massachusetts.
CRISPR Therapeutics (Nasdaq: CRSP) has announced its participation in the Jefferies London Healthcare Conference on November 19, 2020, at 12:00 p.m. ET. The management team will engage in a virtual event, with a live webcast accessible on the company’s website under 'Events & Presentations'. A replay will be available for 14 days post-presentation. Known for its innovative CRISPR/Cas9 gene editing technology, CRISPR Therapeutics aims to develop transformative medicines for serious diseases, collaborating with partners like Bayer and Vertex Pharmaceuticals.
CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals announced the acceptance of data from two ongoing Phase 1/2 clinical trials of CTX001, a CRISPR/Cas9 gene-editing therapy for severe hemoglobinopathies, for oral presentation at the annual ASH Meeting in December 2020. The trials focus on transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD). CTX001 aims to provide a one-time curative therapy by elevating fetal hemoglobin levels. The therapy has received multiple FDA designations, emphasizing its potential in treating these serious conditions.
CRISPR Therapeutics reported significant advancements in its clinical trials and financial results for Q3 2020. The company achieved positive results from its Phase 1 CARBON trial for CTX110, showing a 50% complete response rate. It received Rare Pediatric Disease designation for CTX001 targeting sickle cell disease and Orphan Drug Designation for CTX120. Financially, cash increased to $1.4 billion, but total collaboration revenue dropped to $0.1 million from $211.9 million year-over-year, with a net loss of $92.4 million.
CRISPR Therapeutics announced positive results from the Phase 1 CARBON trial evaluating CTX110, an allogeneic CAR-T cell therapy for CD19+ B-cell malignancies. As of the September 28, 2020 data cutoff, a 50% complete response rate was observed at Dose Level 3, with both responders maintaining their status. The therapy exhibited a favorable safety profile, with no dose-limiting toxicities reported. Management expressed confidence in the potential of CTX110 for broader patient access. The trial aims to assess multiple dose levels and the company is working towards further data releases in the coming year.