BioCentury
ARTICLE | Emerging Company Profile

Actio seeks rare toeholds into common diseases with $55M A round

Led by geneticist David Goldstein, the San Diego company thinks its ‘one-to-many’ strategy builds the economic case for going after very rare diseases

September 12, 2023 6:22 PM UTC

Actio aims to leverage the target biology revealed by the strong genetic perturbations that drive rare diseases to treat a wider range of pathologies with shared phenotypic signatures.

“One of the strongest pieces of evidence we have for what these targets do are mutations that cause severe disease, for example, mutations that dramatically activate a channel,” co-founder and CEO David Goldstein told BioCentury. “What are the phenotypic consequences of that activation?”...