Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
Номер патента: EP4150051A1
Опубликовано: 22-03-2023
Автор(ы): James M. Wilson, Ralf Schmid
Принадлежит: University of Pennsylvania Penn
Опубликовано: 22-03-2023
Автор(ы): James M. Wilson, Ralf Schmid
Принадлежит: University of Pennsylvania Penn
Реферат: Provided is a recombinant adeno-associated virus (rAAV) having an AAV capsid and a vector genome which comprises a nucleic acid sequence encoding a functional CDKL5 (hCDKLK5). Also provided are a production system useful for producing the rAAV, a pharmaceutical composition comprising the rAAV, and a method of treating a subject having CDD, or ameliorating symptoms of CDD, or delaying progression of CDD via administrating an effective amount of the rAAV to a subject in need thereof.
Gene therapy for treating cdkl5 deficiency disorder
Номер патента: US20230054144A1. Автор: Stewart Craig,Sean Christopher DAUGHERTY,Sharyl Lynne Fyffe-Maricich,Matthew Scott Fuller,Margaret Caroline Wright,Lorelei Ioana Stoica. Владелец: Ultragenyx Pharmaceutical Inc. Дата публикации: 2023-02-23.