AavantiBio Launches with $107 Million Financing for Rare Disease Gene Therapies

Rare Daily Staff

AavantiBio launched with $107 million in a Series A financing round to develop gene therapies for rare genetic diseases. The private financing round includes a $15 million equity investment from Sarepta.

Investors in the financing include Perceptive Advisors, Bain Capital Life Sciences, and RA Capital Management together with Sarepta Therapeutics, which made a $15 million equity investment in the startup.

“Our equity participation in AavantiBio serves our strategy to build our gene therapy engine through targeted investment in potentially life-enhancing therapies as well as partnering with renowned genetic medicine pioneers such as Drs. Byrne and Corti,” said Doug Ingram, president and CEO, Sarepta Therapeutics.

Alexander “Bo” Cumbo, a seasoned biopharmaceutical executive, has been appointed AavantiBio’s chief executive officer and president, effective immediately. Cumbo joins AavantiBio after eight years at Sarepta, where he served as chief commercial officer and executive vice president.

GoFAR, an Italian patient advocacy group, and the Muscular Dystrophy Association Venture Philanthropy Fund provided AavantiBio’s initial seed funding.

AavantiBio’s vision is to harness the transformative science of gene transfer therapy and gene editing technologies to improve the lives of people living with fatal diseases. The company is advancing a pipeline of innovative gene transfer therapies in areas of high unmet medical need.

The company has strategic partnerships with the University of Florida’s Powell Gene Therapy Center and the MDA Care Center at UF Health where co-founders and renowned gene therapy researchers Barry Byrne and Manuela Corti maintain their research and clinical practices.

AavantiBio’s lead program builds on the foundational research efforts of Byrne and Corti in Friedreich’s Ataxia (FA), a rare inherited genetic disease that causes cardiac and central nervous system dysfunction.

FA causes movement problems and nervous system damage. Over time, the disease leads to degeneration in the spinal cord, peripheral nerves and cerebellum and causes impaired muscle coordination (ataxia) that gets worse over time. Poor coordination is one of the first noticeable features of FA. The neurological degeneration caused by the disease results in unsteady movements, impaired sensory function, and even the loss of speech. Affected individuals can also develop heart problems, diabetes, or curvature of the spine. However, the disorder does not affect cognitive ability. Though rare, FA affects 1 in every 40,000-50,000 people and is the most common form of hereditary ataxia in the United States.

“AavantiBio has a unique opportunity to change the lives of those living with FA and other rare diseases,” said Cumbo. “Central to our mission is building on the advancements in gene transfer therapies and harnessing these revolutionary technologies to realize their potential as life-altering medicines.”

The AavantiBio board of directors includes Cumbo, Byrne, Corti, and representatives of the investor group, and two independent directors. Louise Rodino-Klapac, senior vice president of gene therapy for Sarepta, will serve as a board observer.

Photo: Alexander “Bo” Cumbo, president and CEO of AavantiBio