Gene and cell therapies will drive innovation for the next 10 years, Claus Zieler, the chief commercial officer at Astellas Pharma Inc., said in the newest episode of the BioWorld Insider podcast. Developers are on the cusp of breakthroughs because a gene can now be replaced “and that means we can potentially cure a disease rather than intervening in a disease.”
Dark genome miner Enara Bio Ltd. has closed a $32.5 million series B that will see the lead program targeting the first of a novel class of cancer antigens it has discovered through to the clinic. Enara calls these cancer antigens “dark antigens” (the name is trademarked). It says they can be found in solid tumors irrespective of the immune phenotype, and are often expressed at high prevalence across multiple different tumors.
Dark genome miner Enara Bio Ltd. has closed a $32.5 million series B that will see the lead program targeting the first of a novel class of cancer antigens it has discovered through to the clinic. Enara calls these cancer antigens “dark antigens” (the name is trademarked). It says they can be found in solid tumors irrespective of the immune phenotype, and are often expressed at high prevalence across multiple different tumors.
Resolution Therapeutics Ltd. is preparing for a phase I/II trial of its autologous engineered macrophage cell therapy, RTX-001, in the treatment of end-stage liver disease and has raised £63.5 million (US$83.3 million) to complete the study and to add further fibrotic and inflammatory disease programs to its portfolio. Recruitment to the study, to be conducted at 15 sites in Spain and the U.K., is due to start before the end of 2024, with the monocyte-derived patient macrophages being processed and modified at a facility in Edinburgh.
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.
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