CRISPR Therapeutics

CRISPR Therapeutics Expands into siRNA Therapies with $95M Sirius Partnership for Thromboembolic Disorders

5 days ago

• CRISPR Therapeutics is diversifying beyond gene editing through a strategic $95 million partnership with Sirius Therapeutics to develop SRSD107, a long-acting siRNA therapy targeting Factor XI for thromboembolic disorders. • In Phase 1 trials, SRSD107 demonstrated impressive efficacy with over 93% reduction in Factor XI levels and doubled blood clotting time, with effects lasting up to six months from a single dose. • The collaboration includes a 50-50 cost and profit-sharing structure for SRSD107 development, with CRISPR leading U.S. commercialization and retaining rights to license two additional siRNA targets.

NKure and CRISPR Therapeutics Partner to Develop Off-the-Shelf CAR-T Therapy for Cancer in India

15 days ago

• Bengaluru-based NKure Therapeutics and CRISPR Therapeutics have formed a strategic partnership to co-develop CTX112, a next-generation allogeneic CAR-T therapy for B-cell malignancies in India. • CTX112 utilizes CRISPR/Cas9 gene-editing technology on healthy donor cells to create an off-the-shelf product that could potentially reduce treatment costs to one-third of current autologous CAR-T therapies in India. • The companies plan to seek CDSCO approval in June to conduct Phase II clinical trials in India, enrolling approximately 25 patients, with a marketable product expected within two years.

FDA's Cell and Gene Therapy Champion Peter Marks Departs, Leaving Industry at Critical Juncture

2 months ago

• Peter Marks, head of FDA's Center for Biologics Evaluation and Research since 2016, has resigned, leaving cell and gene therapy developers without their biggest regulatory advocate during a challenging investment period. • Under Marks' leadership, the FDA approved dozens of cell and gene therapies including the first gene therapy, first cellular treatment for cancer, and first CRISPR gene editing medicine, establishing flexible regulatory frameworks for these novel treatments. • Despite concerns about regulatory uncertainty following Marks' departure, incoming FDA Commissioner Marty Makary has signaled support for conditional approval pathways for rare disease treatments where randomized controlled trials aren't feasible.

Isomorphic Labs to Launch Clinical Trials for AI-Designed Drugs in 2025

4 months ago

• Isomorphic Labs, a Google DeepMind spinoff, anticipates initiating clinical trials for AI-designed drugs by the end of 2025, potentially revolutionizing drug discovery. • CEO Demis Hassabis aims to drastically reduce the drug discovery timeline from a decade to mere weeks or months using AI-driven approaches. • Isomorphic Labs has established strategic research collaborations with Eli Lilly and Novartis, signaling growing confidence in AI's role in pharmaceutical R&D. • The company's platform leverages DeepMind's AlphaFold AI to model molecular structures and predict interactions, accelerating the identification of promising drug candidates.

T-Cell Lymphoma Pipeline Shows Promise with Novel Therapies and Regulatory Support

7 months ago

• The T-Cell Lymphoma drug pipeline is advancing with key players like Eisai, Takeda, and Rhizen Pharmaceuticals developing innovative therapies. • Immunomodulatory drugs such as Lenalidomide and monoclonal antibodies like Isatuximab SAR650984 are in clinical trials, showing potential in improving patient outcomes. • Regulatory support, including fast-track designations, is expediting the development and approval of novel T-Cell Lymphoma treatments. • CAR T-cell therapy, such as lisocabtagene maraleucel (Breyanzi), has been approved for certain lymphomas, offering new options for patients who have not responded to other treatments.

Fulcrum Therapeutics' Losmapimod Fails Phase 3 Trial in FSHD, Development Suspended

8 months ago

• Fulcrum Therapeutics' Phase 3 REACH trial of losmapimod in facioscapulohumeral muscular dystrophy (FSHD) did not meet its primary endpoint, showing no significant improvement in reachable workspace compared to placebo. • Secondary endpoints, including muscle fat infiltration and shoulder strength, also failed to achieve statistical significance in the losmapimod arm. • Fulcrum plans to suspend the losmapimod program and shift focus to pociredir for sickle cell disease and other early-stage programs. • The company's stock price plummeted following the announcement, raising concerns about its future prospects despite a solid cash position.