The Neuromuscular Disease Foundation (also known as NDF) has garnered global attention by making notable advancements in their ambitious plan that was announced in late 2021, to develop the first gene replacement therapeutic to impact GNE Myopathy (GNEM).
The organization is rapidly working on a pre-IND (Investigational New Drug) package, a requisite step toward gaining authorization from the Food and Drug Administration (FDA) to conduct further studies, prior to their reaching the human dosing phase of the treatment. NDF’s team of researchers expects to submit the package to the FDA in 2023, with the intent to acquire the approvals needed for a subsequent IND submission, a step to the first clinical trial of the gene therapy on progressive disease GNEM.
The gene therapy being developed is the first-ever treatment designed to target the root genetic cause of the disease, with the potential to prevent muscle atrophy (or muscle wasting), that is a hallmark of the progressive condition.
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