The Friedreich’s Ataxia Research Alliance (FARA) has awarded Vesigen Therapeutics a general research grant to develop a targeted genome editing therapeutic strategy for Friedreich ataxia (FA).
Under the terms of the grant, Vesigen Therapeutics will evaluate the ability of its proprietary technology, called ARrestin-domain 1 Mediated Microvesicles (ARMMs), to deliver clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein (Cas) genome editing complexes to proprioceptive neurons.
The main goal is to use ARMMs as a nonviral delivery vehicle for genome editing tools to excise the pathogenic repeating GAA sequence in the frataxin gene of patients diagnosed with FA.
Read more about FA treatment
“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 at Vesigen Therapeutics.
To that end, Vesigen Therapeutics will work on engineering strategies for customizing ARMMs, which are a distinct class of human extracellular vesicles, to target the specific tissues and cell types most affected in FA.
“The work being done by Vesigen Therapeutics has the potential to be transformative for FA patients. We are grateful for Dr. Nabhan and his team for their work on this targeted genome editing strategy,” said Jennifer Farmer, CEO of FARA.
Read more about FA diagnosis
ARMMs were discovered and transformed into a drug delivery system at the Harvard School of Public Health in Boston, Massachusetts. They can be used to deliver a wide range of payloads (eg, gene editing tools, proteins, and RNAs) to target tissue and cell types.
FARA supports scientific research leading to treatments and a cure for FA. With its grant program, FARA funds competitive grants that cover basic research through drug development and clinical research programs.
Currently, there is no curative treatment for FA.
Reference
Vesigen Therapeutics awarded grant from Friedreich’s ataxia research alliance (FARA) to develop a targeted genome editing therapeutic strategy. News release. Vesigen Therapeutics, Inc.; March 14, 2023.
