Carmot Therapeutics Inc. has raised $160 million in series D financing to support a trio of early to midstage clinical programs focused on treating diabetes and obesity with peptide-based small-molecule incretin receptor modulators.
Verve Therapeutics Inc. has packed a lot into the past few weeks. The latest is a four-year research deal with Vertex Pharmaceuticals Inc. to find and develop an in vivo gene editing program for an undisclosed liver disease. Vertex will pick up the tab for program costs as Verve does the preclinical R&D. Verve is getting an up-front $60 million from Vertex, along with a $35 million equity investment.
The U.S. FDA has placed a clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating AT-845 following a serious adverse event of peripheral sensory neuropathy in one of the trial participants. AT-845 is an adeno-associated virus (AAV) gene replacement therapy being studied in adults with late-onset Pompe disease.
Investigators at Stanford University and Baylor College of Medicine have identified an exercise-induced appetite suppressant that led to weight loss when administered to obese mice. The molecule, Lac-Phe, has led to predictable excitement around the possibility of appetite-suppressing exercise in a pill.
New and updated preclinical and clinical data presented by biopharma firms at the Endocrine Society annual conference, including: Astellas, Bridgebio, Eiger, Horizon, Innovent, Neurocrine, Novo, Orphagen, Rhythm, Versanis.
The Cellular, Tissue and Gene Therapies Advisory Committee scrutinized Bluebird Bio Inc.’s gene therapy elivaldogene autotemcel (eli-cel) for early active cerebral adrenoleukodystrophy (CALD) in patients without a matched sibling donor.
Wall Street took in stride mixed FDA briefing documents with regard to the upcoming adcom review of Bluebird Bio Inc.’s two gene therapy prospects, and shares of the company (NASDAQ:BLUE) closed at $3.61, up 63 cents, or 21%.
Shares of Aeglea Biotherapeutics Inc. tumbled 51% June 2 on news that it received a refusal to file (RTF) letter from the U.S. FDA regarding the BLA for pegzilarginase for the treatment of arginase 1 deficiency, with the agency requesting additional efficacy data.
Abliva AB has gone above its market capitalization to raise the money it needs to start a phase II/III trial of KL-1333 for the treatment of primary mitochondrial disease.
Swanbio Therapeutics Inc. closed a $56 million series B round to take its lead gene therapy program, SBT-101, into clinical development later this year. The candidate, comprised of an adeno-associated virus type 9 vector encoding the ABCD1 peroxisomal ATP-binding cassette transporter, is in development for adrenomyeloneuropathy (AMN), an inherited disease that affects the central nervous system.
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