Abstract: The present invention provides a polynucleotide and recombinant AAV encoding human alpha galactosidase A. The present invention also provides a method of treating Fabry disease comprising administering the recombinant AAV to a subject in need thereof.
Abstract: The present invention relates to a modified adeno-associated virus (AAV) capsid polypeptide and a novel recombinant adeno-associated virus (rAAV) comprising the modified AAV capsid polypeptide for delivering the gene product for the therapy. The present invention also relates to a pharmaceutical composition comprising the rAAV of the present invention, and a method of treating a retinal disease comprising the administration of the rAAV or the pharmaceutical composition of the present invention to an eye of a subject in need thereof.
Abstract: The present invention provides a polynucleotide and vector, in particular AAV vector, encoding a nanobody against VEGF. The present invention also provides a method of treating a disease associated with VEGF, such as the overexpression of VEGF, e.g., Wet-AMD and DME comprising administering the vector by, e.g., intravitreal injection to provide stable expression of the nanobody in eyes.
Type:
Application
Filed:
March 15, 2024
Publication date:
August 1, 2024
Applicant:
Skyline Therapeutics Limited
Inventors:
Jinzhao HOU, Yanqun SHU, Qiongqiong REN, Heping LI