BOLD - Why Audentes' rAAV Gene Therapy Can Be A Game Changer
Company Thesis
Audentes Therapeutics' (BOLD) flagship candidate AT-132 has shown very good risk-reward profile in the treatment of newborns with X-linked Myotubular Myopathy (XLMTM). The therapy has amazing efficacy; immunogenicity profile, genotoxicity profile, and demonstrated satisfactory safety data in its ASPIRO interim Phase I/II data release. This rare disorder has an addressable market value of $550 million in peak annual revenues and, currently, has no treatment option available aside from ventilator support. In all, the candidate has an estimated 55% chance the therapy will make it from Phase I/II to the end of