MDA Awards $1M to AavantiBio for Gene Therapy Development in FA

The Muscular Dystrophy Association (MDA) has awarded AavantiBio $1,076,232 to advance the biotechnology company’s development of a gene replacement therapy for Friedreich’s ataxia (FA).

Awarded through the MDA Venture Philanthropy drug development program, the funds will help launch production of the clinical gene vector for AavantiBio’s therapy program. A Phase 2 clinical trial is expected to start this year.

“With the approval of several first-time gene-targeting therapies for neuromuscular diseases over the past several years, including the first-ever gene-replacement therapy for spinal muscular atrophy, our community should have much to hope for as more and more therapies continue to be developed,” Lynn O’Connor Vos, MDA’s president and CEO, said in a press release.

“MDA is thrilled to be a part of this quest to help further develop the first gene-targeting therapy for the treatment of Friedreich’s ataxia. By partnering with AavantiBio, together we can address the unmet need faced by patients who live with this genetic disease for which there are still no treatments and no cures,” Vos said.

The early-stage company AavantiBio was established in 2017 specifically to develop an effective therapy for Friedreich’s ataxia, a degenerative neuromuscular disease that damages the nervous system and causes loss of control of body movement. The disorder affects about 1 in 50,000 U.S. residents.

Because FA is caused by mutations in a single gene — FXN — the disease could potentially be treatable through a gene therapy approach. In such approaches, genetic material is delivered into the body’s cells, leading to a therapeutic effect by correcting an existing mutation or introducing a new gene to help fight a specific disease. The new genetic material can be delivered via viral and non-viral vectors.

In its effort to create the first effective treatment for FA, AavantiBio began working with the FA community five years ago. The company expects to include adult and pediatric patients in clinical trials.

“This is a great opportunity for AavantiBio, and we’re thankful to the MDA for their generous contribution,” said Manuela Corti, PhD, an assistant professor of pediatrics at the University of Florida (UF) and a company co-founder. “We hope to strengthen our collaboration as we work together on this project.”

Corti was previously awarded an MDA research grant to develop and assess a gene-replacement therapy in an FA mouse model. The latest funding will support a new university study — led by Sub Subramony, MD, professor of neurology — that will evaluate changes in neurological and cardiac function in FA patients treated with both intravenous (into the vein) and intrathecal (spinal) injections of the gene replacement therapy.

“We’re delighted to take the next step in fulfilling our commitment to patients and families living with FA based on this investment from the MDA,” said Barry Byrne, MD, PhD, AavantiBio co-founder and associate chair of pediatrics, and director of the Powell Gene Therapy Center at UF.

“We’re looking forward to initiating screening for the first clinical study and a pivotal study in FA before the end of the year. The endorsement and investment from the MDA will be key to our programmatic growth.”