The National Institute of Neurological Disorders and Stroke (NINDS) has awarded a 3-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children’s Hospital to fund completion of preclinical studies, manufacturing and preparation of an IND application for a first-in-human trial to advance adeno-associated virus (AAV)-ATP7A gene therapy for the treatment of Menkes disease.

Coave Therapeutics SA’s CTx-TFEB program has received support through a grant from the ALS Association.

An Italian group of researchers has used zinc finger editing to silence the PCSK9 gene and improve blood cholesterol levels in mice by applying a single dose of their modifier. The epigenetic-based method could be an alternative to genome editing.

Pulsesight Therapeutics SAS has launched with seed financing, with the aim of developing disruptive non-viral gene therapies with minimally invasive delivery technology.

Voyager Therapeutics Inc. has announced the selection of a lead development candidate from its Friedreich’s ataxia program with collaborator Neurocrine Biosciences Inc.

Voyager Therapeutics Inc. has announced new data from two preclinical programs targeting pathological tau for the treatment of Alzheimer’s disease.

Parents Project Muscular Dystrophy (PPMD) has provided $500,000 in funding to Kinea Bio Inc. 

The Foundation for the National Institutes of Health (FNIH) has announced the online publication of the first playbook designed to help accelerate the development of adeno-associated virus (AAV) gene therapies for rare diseases.

Encoded Therapeutics Inc. is advancing its lead gene therapy candidate, ETX-101, for the treatment of SCN1A+ Dravet syndrome into the clinic.