Crispr Therapeut Stock Price, News & Analysis

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.

Vertex Pharmaceuticals and CRISPR Therapeutics announced additional abstracts for the 2022 European Hematology Association (EHA) Congress. The highlight is the late-breaking abstract on the efficacy and safety of exa-cel for beta-thalassemia and sickle cell disease, scheduled for oral presentation on June 12. Three real-world economics abstracts will also be presented on June 10, detailing the economic burden of these diseases in the U.S. Exa-cel is being investigated as a one-time therapy for these patients.

CRISPR Therapeutics (Nasdaq: CRSP) will host an Innovation Day on June 21, 2022, at 2:00 PM ET, focusing on its advanced research in genetic engineering and cell therapy. The event will showcase the company's innovative platform technologies and new discovery programs. A live webcast will be available on the company's website, with the recording archived for 30 days post-event. CRISPR Therapeutics is committed to developing transformative gene-based medicines targeting serious diseases, leveraging its proprietary CRISPR/Cas9 technology.

CRISPR Therapeutics (Nasdaq: CRSP) has appointed Dr. Phuong Khanh (P.K.) Morrow as Chief Medical Officer, effective May 23, 2022. Dr. Morrow brings over a decade of experience in global drug development, particularly in oncology, and will oversee the company's clinical development and regulatory operations. The leadership change aims to enhance CRISPR’s innovative gene-editing therapies. CEO Samarth Kulkarni emphasized her expertise will be essential in advancing their clinical strategy and building the organization.