july 2025
Build backwards: A framework for rare disease gene therapy development
Rare disease gene therapies don’t fail due to poor science, they fail because of a flawed strategy. Our latest article challenges the conventional linear model of therapeutic development and presents a more viable approach for rare conditions.
Why building backwards leads to better clinical, regulatory, and commercial outcomes
How ViroCell’s modular lentiviral platform supports scalability across rare indications
What it means to design gene therapies that are built to reach patients
Read the complete article, co-authored by Nicholas Ostrout, Ph.D., VP of Corporate Development & Strategy and CSO Farzin Farzaneh, Ph.D., ViroCell Biologics; and Hilary Schultz, M.S., CEO, Persephoni Biosciences LLC.