Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)

This information pertains to a grant focused on advancing gene-based treatments for ultra-rare neurological and neuromuscular disorders. This grant is part of the Ultra-Rare Gene-Based Therapy (URGenT) network, which provides support for exploratory studies and planning activities that pave the way for the first wave of human clinical testing of gene-based therapies. These therapies include things like oligonucleotides and viral-based gene treatments.

The central aim of this funding opportunity is to speed up the development of a promising clinical candidate. This candidate should already have a strong biological rationale and demonstrated proof of concept data for their intended approach. This approach needs to be shown effective in a model system that is relevant to the specific patient population they are aiming to help. If successful, this candidate will be able to apply for an Investigational New Drug (IND) status and kick-start a clinical trial. Please note that engaging in an actual clinical trial is optional under this specific grant.