FARA Awards Grant to Vesigen to Develop a Targeted Genome Editing Strategy for Friedreich’s Ataxia

Rare Daily Staff

The Friedreich’s Ataxia Research Alliance awarded a grant to Vesigen Therapeutics to 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.

Friedreich’s ataxia is an ultra-rare, inherited disorder that is typically diagnosed during adolescence. Patients with Friedreich’s ataxia experience progressive loss of coordination, muscle weakness, and fatigue, which commonly progresses to motor incapacitation and wheelchair reliance by their teens or early twenties, and eventually death.

Vesigen is developing targeted therapies by engineering a distinct class of human extracellular vesicles called ARMMs (ARrestin-domain 1 Mediated Microvesicles). ARMMs can be used to precisely deliver a wide range of payloads, including gene editing complexes, proteins, and RNAs to a unique set of tissue and cell types. Vesigen has demonstrated highly efficient in vitro and in vivo functional delivery of a range of payloads across multiple cell types.

The FARA Grant Program funds competitive grants across the spectrum from basic research through drug development and clinical research programs.

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 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, chief scientific officer of Vesigen Therapeutics.

Photo: Joseph Nabhan, chief scientific officer of Vesigen Therapeutics