Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01, Clinical Trial Not Allowed)

Published by NIH

Gene TherapyUltra Rare Disease

The purpose of this FOA is to provide investigators with a mechanism to access contract research/medical organizations (CROs/CMOs) and subject matter experts (SMEs) within the NINDS Ultra-Rare Gene-based Therapy (URGenT) Network to support planning, manufacturing, and limited nonclinical therapeutic development efforts.

View this resource Bookmark this resource

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

Published by NIH

Gene TherapyUltra Rare Disease

The Ultra-Rare Gene-Based Therapy (URGenT) network supports Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) clinical testing of gene-based or transcript-directed therapeutics, such as oligonucleotides and viral-based gene therapies, for ultra-rare neurological or neuromuscular disorders. The goal of this funding opportunity announcement (FOA) is to accelerate the development of a promising clinical candidate with robust biological rationale and demonstrated proof of concept (POC) data for the intended approach in a model system relevant to a specified patient population towards an IND filing and the initiation of a clinical trial.

View this resource Bookmark this resource