Researchers from Kyverna Therapeutics Inc. presented preclinical data for KYV-101, a first-in-class, fully human autologous anti-CD19 chimeric antigen receptor (CAR) T-cell therapy being developed for the treatment of patients with B-cell-driven neurologic autoimmune diseases, including multiple sclerosis and myasthenia gravis.
Papillon Therapeutics Inc.’s PPL-001 has been awarded orphan drug designation by the FDA for Friedreich’s ataxia. PPL-001 is an experimental gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy.
Capricor Therapeutics Inc. has expanded its commercialization and distribution deal with Nippon Shinyaku Co. Ltd. to include the EU and the U.K. for deramiocel, Capricor’s lead asset, in treating Duchenne muscular dystrophy (DMD). In the new agreement, Capricor will receive an up-front payment of $20 million. Capricor will handle development and manufacturing duties for deramiocel while Nippon Shinyaku will be responsible for the sales and distribution.
Somite Therapeutics Inc.’s lead program, SMT-M01, has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of Duchenne muscular dystrophy (DMD). The program leverages the company’s proprietary Alphastem artificial intelligence (AI) platform to develop a novel cell replacement therapy for DMD.
The newest data show increased survival times for heavily pretreated patients receiving Briacell Therapeutics Corp.’s breast cancer treatment. The company’s phase II results produced positive overall survival data using the cell-based immunotherapy Bria-IMT to treat late-stage metastatic breast cancer.
TC Biopharm (Holdings) plc has announced plans to begin proof-of-concept preclinical studies for its lead therapeutic, TCB-008, for the treatment of mpox.
Medigene AG has obtained FDA clearance of its IND application for lead program MDG-1015 for the treatment of advanced gastric cancer, ovarian cancer, myxoid/round cell liposarcoma and synovial sarcoma.
Medigene AG has provided a pipeline update in its half-year report for the first half of 2024. The company, which develops T cell receptor (TCR)-guided therapies for the treatment of cancer, confirmed for following positive EU and U.S. preliminary regulatory interactions, is on track for filing an IND in Q3 and a CTA in Q4 of 2024 for its lead candidate MDG-1015.
Precision Biosciences Inc. has announced that its experimental allogeneic T cell therapy azercabtagene zapreleucel (azer-cel) for the treatment of multiple sclerosis (MS) has received IND clearance from the FDA.
CSPC Pharmaceutical Group Ltd. has received clearance from China’s National Medical Products Administration (NMPA) to conduct clinical trials in China with its BCMA-targeting CAR T cell SYS-6020 for systemic lupus erythematosus (lupus) in China.
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