Ambulero Inc., a spinout from the University of Miami Miller School of Medicine, has received a positive response from a type B pre-IND meeting with the FDA on the development of AMB-301 as a treatment for the vascular disease Buerger's disease.
SBT-101 (Swanbio Therapeutics Inc.) is an adeno-associated virus serotype 9 (AAV9)-based gene therapy that delivers a functional copy of the ABCD1 gene and is under development for the treatment of adrenomyeloneuropathy (AMN).
Bluebird Bio Inc.’s elivaldogene autotemcel (eli-cel) gained U.S. approval late Sept. 16 for use in early active cerebral adrenoleukodystrophy (CALD), making it the firm’s second gene therapy to clear the FDA in as many months. Branded Skysona, eli-cel is expected to be available commercially by the end of 2022 and its launch will require only “incremental” company resources on top of those required for the ongoing launch of beta-thalassemia gene therapy Zynteglo (betibeglogene autotemcel), Bluebird said.
Ocular gene therapy firm Sparingvision SA raised €75 million (US$75 million) in a series B round to fund its transition to clinical development. The company is about to move its lead program, the mutation-agnostic gene therapy SPVN-06, into a phase I trial in retinitis pigmentosa (RP). “We are in the middle of the regulatory submission process,” CEO Stéphane Boissel told BioWorld.
Coave Therapeutics SA has entered into a collaboration with the Institute of Neurodegenerative Diseases (IMN), a joint research unit associating the University of Bordeaux and the French National Centre of Scientific Research (CNRS), to develop gene therapy programs targeting protein degradation in neurodegenerative disorders.
Akouos Inc. has received IND clearance from the FDA to initiate a first-in-human phase I/II pediatric trial of AK-OTOF, a gene therapy intended for the treatment of patients with otoferlin gene (OTOF)-mediated hearing loss.
The University of Sydney is investing AU$478 million (US$326.5 million) to build a leading biomedical precinct to fast-track research and shorten the time between discovery and development of transformative therapies.
The University of Sydney is investing AU$478 million (US$326.5 million) to build a leading biomedical precinct to fast-track research and shorten the time between discovery and development of transformative therapies.
Epigenic Therapeutics Co. Ltd., a company developing epigenomic-focused gene therapies in multiple indications, has raised $20 million across angel and pre-series A rounds to support preclinical studies, early stage research, and growth for its team.
Epigenic Therapeutics Co. Ltd., a company developing epigenomic-focused gene therapies in multiple indications, has raised $20 million across angel and pre-series A rounds to support preclinical studies, early stage research, and growth for its team.
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