The FDA has awarded orphan drug designation to Papillon Therapeutics Inc.’s PPL-002, an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, for the treatment of Danon disease.
In the year’s fourth-largest deal, Prime Medicine Inc. will collaborate with Bristol Myers Squibb Co. in a research collaboration and license agreement totaling $3.61 billion. The two companies plan to develop reagents for ex vivo T-cell therapies. While the programs and targets have yet to be disclosed, BMS is expanding its CAR T development, begun more than five years ago, with this deal.
Cartherics Pty Ltd. has raised over its target of AU$15 million (US$10.3 million) in an oversubscribed private financing round. Funding will support a clinical trial for CTH-401, the company’s lead cell therapy for ovarian cancer, and expand its pipeline to include other diseases.
In the year’s fourth-largest deal, Prime Medicine Inc. will collaborate with Bristol Myers Squibb Co. in a research collaboration and license agreement totaling $3.61 billion. The two companies plan to develop reagents for ex vivo T-cell therapies. While the programs and targets have yet to be disclosed, BMS is expanding its CAR T development, begun more than five years ago, with this deal.
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.
RSS
