VC Enthusiasm For Gene Therapy Is Going Viral
Audentes is the latest gene therapy start-up to land a large Series A round, taking $30 million to explore treatments for two rare muscle diseases. It’s another sign that VCs believe the risks around gene therapy are continuing to abate – and that pharmas aren’t far behind them.
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Shares of uniQure, Spark Therapeutics and bluebird bio all opened lower April 27 after Celladon announced its heart failure gene therapy Mydicar failed to show a benefit versus placebo in a mid-stage trial.
As gene therapy moves closer toward becoming an established therapeutic mode, developers have focused mainly on ophthalmology and monogenic rare diseases, but improvements in vector technology are pointing the field toward targets in the central nervous system. The latest entrant is Voyager Therapeutics Inc., which launched in February with a $45 million Series A commitment from Third Rock Ventures and a lead program in Parkinson's disease.
uniQure Grants Chiesi Select Commercial Rights For The EU’s First Approved Gene Therapy Glybera And Hemo B Gene Therapy
uniQure has licensed commercial rights covering Europe and select emerging markets to Italy’s Chiesi for first-to-market gene therapy Glybera and a developmental hemophilia B product. The Dutch biopharma is writing the book on pricing and commercialization, regulatory approval, and market access strategy for gene therapy.