CRISPR Therapeutics Receives FARA Grant to Develop Gene Editing Therapies for Friedreich’s Ataxia

CRISPR Therapeutics has received a grant from the Friedreich’s Ataxia Research Alliance to support its work on gene editing as a potential treatment for the disease.

The company will use the Kyle Bryant Translational Research Award on animal studies of CRISPR/Cas9 gene-editing strategies for FA. The size of the grant was not disclosed.

CRISPR Therapeutics will conduct the research in collaboration with Dr. Marek Napierala, an associate professor at the Stem Cell Institute of the University of Alabama at Birmingham.

“We believe that the combination of CRISPR’s pioneering gene-editing capabilities with Dr. Marek Napierala’s deep expertise in FA may accelerate the development of new therapies for FA,” Jennifer Farmer, FARA’s executive director, said in a press release.

“We are delighted to partner with Dr. Napierala and FARA on this journey to harness the potential of CRISPR/Cas9 gene editing for patients with FA,” said Dr. Tony Ho, head of research and development at CRISPR Therapeutics. “We believe that our gene editing technology is uniquely suited to target the underlying genetic causes of genetic disorders such as FA.”

CRISPR Therapeutics’ role in the collaboration will be to develop reagents that can regulate the genetics of a cell, with the potential to overcome the underlying genetic modifications that lead to FA.

Napierala’s team will evaluate the safety and effectiveness of the reagents in animal models that mimic human FA. The hope is that such studies will provide proof-of-concept information that can support clinical trials of the reagents. CRISPR Therapeutics will be responsible for the trials.

“With our growing understanding of the genetic and molecular underpinnings of FA in patients and animal disease models, the time is right to develop a CRISPR/Cas9-based therapeutic approach,” Napierala said. “I am excited to join CRISPR Therapeutics and FARA in attempting to develop a cure for this devastating disease.”

The award announcement coincided with FARA’s rideATAXIA fund-raising event in Philadelphia. RideATAXIA is a nationwide program that Kyle Bryant, who has Friedreich’s ataxia, started in 2009. His goals were to increase awareness of FA and raise funds for research aimed at developing treatments and finding a cure for the disease.

“FARA is thrilled to celebrate rideATAXIA Philly with the announcement of the Kyle Bryant Translational Research Award to CRISPR Therapeutics,” Farmer said. “We are grateful to all of the FA families that fundraise and the CureFA Foundation for providing the resources to advance research.”