Vesigen Therapeutics Awarded Grant from Friedreich’s Ataxia Research Alliance (FARA) to Develop a Targeted Genome Editing Therapeutic Strategy

Vesigen Therapeutics, a biotechnology company developing targeted therapies by engineering a distinct class of human extracellular vesicles called ARMMs (ARrestin-domain 1 Mediated Microvesicles), is pleased to announce receipt of a FARA (Friedreich’s Ataxia Research Alliance) General Research Grant. Vesigen will evaluate its proprietary technology to deliver CRISPR-Cas genome editing complexes as a non-viral disease-modifying strategy for patients diagnosed with the neurodegenerative disease Friedreich’s Ataxia (FA).

Under the terms of the grant, Vesigen Therapeutics will evaluate the use of ARMMs as a non-viral delivery vehicle for genome editing tools to excise the pathogenic repeat expansion in the Frataxin gene. A major focus of this work will require engineering of ARMMs to engage specific tissues and cell types most affected in FA.

“We are delighted to partner with FARA to harness the therapeutic potential of ARMMs as vehicles for targeted genome editing in proprioceptive neurons, which reside in the dorsal root ganglia. We believe that our technology is uniquely suited to enable cell type-specific correction of the underlying cause of a genetic disorder, such as FA,” said Joseph Nabhan, PhD, Chief Scientific Officer, Vesigen Therapeutics.

The FARA Grant Program funds competitive grants across the spectrum from basic research through drug development and clinical research programs. “The work being done by Vesigen Therapeutics has the potential to be transformative for FA patients,” says Jennifer Farmer, CEO of FARA. “We are grateful for Dr. Nabhan and his team for their work on this targeted genome editing strategy.”