RARE Daily

AavantiBio Partners with Catalent to Advance Gene Therapies for Rare Genetic Diseases

April 27, 2021

Rare Daily Staff

Recently launched startup AavantiBio has partnered with services provider Catalent to support the development and manufacturing of AavantiBio’s gene therapies, including its lead program in Friedreich’s ataxia.

Friedreich’s ataxia (FA) is a rare genetic disease caused by abnormally low levels of a protein called frataxin due to mutations in the FXN gene. Deficiency in frataxin leads to mitochondrial malfunction, impaired energy production, and progressive neuromuscular and cardiac damage. AavantiBio’s AAV-based therapy is designed to deliver a functional copy of the FXN gene to cells so that they can produce frataxin. FA affects one in every 40,000-50,000 people and is the most common form of hereditary ataxia in the United States.

Catalent will provide process development and CGMP manufacturing of AavantiBio’s adeno-associated viral (AAV) vector-based therapeutic candidate for use in clinical trials in the United States and Europe. Catalent will further support process optimization and look to reduce material timelines at its process and clinical development center in Baltimore, Maryland.

“This partnership highlights the importance of prioritizing chemistry, manufacturing, and controls as a foundational element of the company,” said Bo Cumbo, president and CEO of AavantiBio. “We believe this approach will position AavantiBio for long-term success as we begin to advance therapies to transform the lives of patients with FA and other rare diseases.”

Catalent has facilities for the production of plasmid DNA and cell and gene therapies in the United States and Belgium. These sites provide development and manufacturing services to support projects from clinical- to commercial-scale, and house multiple CGMP manufacturing suites, including fill/finish, testing laboratories, warehousing, supply chain capabilities, and other associated services.

Photo: Bo Cumbo, president and CEO of AavantiBio

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