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.
Bluebird Bio Inc. isn’t giving out much of the information on the margins for the cost of its newly approved cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia. The numbers will, the company said, come into better focus when another Bluebird drug is approved and launched.
The U.S. FDA has approved the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia requiring frequent red blood cell transfusions. The $2.8 million wholesale acquisition cost for the one-time I.V. infusion will make it one of the most expensive drugs in the U.S.
Vector Biopharma AG has secured a $30 million series A funding commitment from founding investor Versant Ventures to take forward a new gene delivery platform developed by Andreas Plückthun, of the University of Zurich, in Switzerland.
Frontera Therapeutics Inc. raised $160 million in a series B funding round to develop its lead gene therapy product candidate for retinal disease, FT-001, for which INDs have been approved by the U.S. FDA and China NMPA.
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