Grace Science warns FDA bottleneck threatens ultra-rare gene therapy progress

Grace Science, a biotech focused on ultra-rare diseases, has been developing a gene-therapy candidate that appeared close to regulatory approval.

The company, co-founded by Nobel laureate Carolyn Bertozzi and backed by $72 million in funding, has attracted attention for its potential to treat conditions with few options.

A recent FDA request has created a bottleneck, raising concerns that the therapy's timeline could be delayed, as illustrated by a father who built a gene therapy for his daughter and now faces the regulatory hold-up.