AavantiBio is a clinical-stage biopharmaceutical company committed to developing and commercializing novel gene transfer and gene editing therapies for the treatment of rare and ultra-rare genetic diseases. The company is rapidly building a diverse portfolio of gene transfer and gene editing therapies to target debilitating genetic diseases for which the unmet medical need is high for which there are typically no approved therapies treating the underlying disease. AavantiBio is headquartered in Cambridge, MA with additional locations in Gainesville, FL.
As of now, only 5% of so-called rare diseases (orphan diseases) have an FDA-approved treatment available to patients. Rare diseases are defined as those affecting fewer than 1 in 200.000 people with almost 80% of all rare diseases being of genetic nature. With recent progress in gene-editing technologies, including breakthroughs …
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