Mark Curtis & Richard Philipson
Providing a critical overview of the sector’s commercial developments – M&As, licensing agreements & collaborations, financial results, IPOs and clinical/regulatory updates, with commentary from our Expert Contributors.
Allogeneic cell therapies were in the spotlight this past month with Rubius Therapeutics raising an additional $100M to fund its red blood cell platform, and Sigilon Therapeutics securing an Advanced Therapy Medicinical Product Designation from the FDA for its encapsulation technology for transplant. Both companies were birthed in recent years by Flagship Pioneering. On the autologous front JW Therapeutics, a subsidiary of Juno Therapeutics, raised $90M to fund development of CAR-T therapies in Asia, and Bluebird bio and Celgene decided to share the development work to bring their anti-BCMA CAR-T to market in the United States.
This month sees several companies achieve notable regulatory “endorsements”, with MeiraGTx receiving EMA PRIME designation for its gene therapy candidate for achromatopsia, AveXis receiving Sakigake designation for its SMA Type 1 gene therapy in Japan and Abeona Therapeutics receiving Rare Pediatric Disease Designation for its gene therapy program in CLN1 disease. Whilst not providing any guarantee of future approval, these designations nevertheless indicate good quality programs underpinned by solid scientific data. Avexis is one of the few companies to seek Sakigake designation in Japan to complement its existing European PRIME and US Breakthrough Therapy designations, indicating its interest in early engagement with all three key regulatory authorities to discuss its clinical plans and data.
Citation: Cell Gene Therapy Insights 2018; 4(3), 241-254.