Venture Philanthropy Funds Rare Neuro Disease Trial

Neurons (Laura Struzyna, University of Pennsylvania, NIH.gov)

13 Jan. 2020. A gene therapy start-up is receiving an award resembling venture financing to advance its treatment for a rare inherited neuromuscular disorder to clinical trials. Muscular Dystrophy Association in New York awarded AavantiBio in Gainesville, Florida some $1.1 million to support the company’s treatment for Friedreich’s ataxia, a rare progressive genetic disease that causes movement problems and impaired muscle coordination.

Friedreich’s ataxia is caused by a mutation in the FXN gene that codes for the protein frataxin. People inheriting two defective copies of the FXN gene, one from each parent, are likely to develop the disease, which allows nerve fibers in the spinal cord and peripheral nerves to degenerate and become thinner.

As nerve fibers degenerate, damage to the nerves increases, leading to awkward and unsteady movements, as well as impaired sensory functions. The disorder can also lead to damage to the cerebellum, the part of the brain coordinating balance and movement, as well as heart and spine.  Friedreich’s ataxia affects about 1 in 50,000 people and is usually diagnosed in childhood.

AavantiBio is a spin-off enterprise from University of Florida medical school in Gainesville, founded three years ago by pediatrics research professor Manuela Corti and Barry Byrne, director of the university’s gene therapy center. Corti and Byrne study therapies that deliver healthy FXN genes to the nervous system and heart using benign viruses, to replace the mutated genes causing the disease.

Corti received earlier funding from Muscular Dystrophy Association for preclinical research with lab mice testing a gene therapy for Friedreich’s ataxia, as well as from the Italian patient advocacy group for the disease known as GoFAR. In November 2019, GoFar also awarded AavantiBio a $600,000 grant to begin clinical trials of the company’s gene therapy.

The new Muscular Dystrophy Association award of $1,076,232 to AavantiBio is made from MDA’s venture philanthropy, or MVP fund. The MVP award supports the first clinical trials of AavantiBio’s gene therapy for Friedreich’s ataxia, or FA. “We’re looking forward to initiating screening for the first clinical study and a pivotal study in FA before the end of the year,” says Byrne in an MDA statement. “The endorsement and investment from the MDA will be key to our programmatic growth.”

The venture philanthropy fund supports promising higher-risk projects that would not otherwise advance without its support. MVP grants fund, for example, research and development hampered by smaller markets, new technology, or an unclear regulatory path. While not seeking an equity stake in the recipient, MVP awards resemble venture capital financing, in that payments are dependent on achieving milestones, and progress is subject to regular reviews and evaluation.

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