Nightstar Therapeutics plc
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Latest From Nightstar Therapeutics plc
AGTC hopes to gain the first approval in X-linked retinitis pigmentosa for its gene therapy AGTC-501, now in a Phase II/III study. It also is advancing development in achromatopsia, another unmet medical need.
BIIB112, the gene therapy candidate Biogen picked up in $877m acquisition of Nightstar, missed its primary endpoint in X-linked retinitis pigmentosa. Phase III data for BIIB111, also from Nightstar, are expected soon.
Biogen will pay Denali $560m up front plus a $465m equity investment in exchange for co-development and co-commercialization rights to clinical-stage LRRK2 inhibitors for Parkinson's disease.
Second quarter performance was better than expected, but commercial weaknesses show the need for R&D wins and M&A deals as Tecfidera generics and aducanumab uncertainty weigh on the company.
- Other Names / Subsidiaries
- Nightstar Therapeutics Limited
- NightstaRx Ltd