Rare Daily Staff

Gene therapy company AavantiBio and biopharmaceutical manufacturer National Resilience said they have formed a strategic collaboration to support the development and manufacturing of AavantiBio’s pipeline of therapies, including its lead program in the rare neuromuscular disease Friedreich’s ataxia.

Resilience will provide process development and GMP manufacturing services including cell lines and viral banks for AavantiBio’s adeno-associated viral vector-based therapeutic candidate for Friedreich’s ataxia for use in both preclinical studies, and phase 1/2 clinical trials in the United States and Europe. Resilience will assist in the development and optimization of the manufacturing processes for GLP Tox and first-in-human material at its 183,000-square-foot, state-of-the-art facility in Alachua, Florida.

“This partnership supports our immediate and long-term objectives in developing and ultimately commercializing our diverse pipeline of gene therapies, beginning with our lead FA program,” said Bo Cumbo, president and CEO of AavantiBio. “With an emphasis on chemistry, manufacturing and controls for gene therapies, we are committed to ensuring the quality of manufacturing processes along with analytical development.”

Friedreich’s ataxia (FA) causes progressive nervous system damage and movement problems. The multisymptomatic disease usually begins in childhood and leads to degeneration in the spinal cord, peripheral nerves and cerebellum (the part of the brain that controls synchronization and balance) and causes impaired muscle coordination (ataxia) that worsens over time. 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. Though rare, FA affects one in every 40,000-50,000 people and is the most common form of hereditary ataxia in the United States.

Photo: Bo Cumbo, president and CEO of AavantiBio

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