The 2022 edition of Science for Health, co-hosted by MEDVIA on 21 November in Brussels, focuses on gene therapy. Dr Federico Mingozzi, CSO of Spark Therapeutics, will speak on the technical challenges hampering progress in the area

Federico Mingozzi is the CSO of Spark Therapeutics, the company behind Luxturna – the world’s first FDA-approved gene therapy for a genetic disease. Though the gene therapy community likes to focus on success stories like Luxturna, the reality is that gene therapy is a new modality, and the field is still developing.

BioVox spoke to Dr Mingozzi about some of the technical challenges currently hampering progress in gene therapy, and how people are working together to overcome these hurdles.

Q: Gene therapy faces a lot of technical challenges, starting with the delivery itself. Could you tell us a bit about this?

Federico Mingozzi: Despite the successes in the clinic, some challenges with gene therapy remain. Many of these – particularly regarding efficiency and toxicity associated with high vector doses – are due to the fact that some of our tools aren’t fully optimized for targeting specific tissues.

For example, in a disease like Duchenne muscular dystrophy, where you’re trying to target muscle tissue, vectors used to deliver the DNA payload may end up off-target in the liver. This can then trigger liver toxicity, which is something we’ve seen in clinical trials, particularly where very large doses of AAV were used systemically.

Similarly, if you’re trying to target the central nervous system, current tools aren’t very efficient at crossing the blood-brain barrier, meaning that high-doses are required, and the vector ends up in other tissues across the body.

See the rest of this interview at BioVox

Science for Health takes place online and in Brussels on 21 November. See the full program and sign up here