ADENO ASSOCIATED VIRAL (AAV) VECTORS FOR TREATMENT OF PROPIONIC ACIDEMIA (PA) CAUSED BY MUTATIONS IN PROPIONYL-COA CARBOXYLASE BETA (PCCB)
US Application 63/706,441
Filed on 2024-10-11
Filed on 2024-10-11
CIRCULATING HEPATIC BIOMARKERS ASSOCIATED WITH METHYLMALONIC ACIDEMIA AND METHODS OF THEIR USE
US Application 63/631,025
Filed on 2024-04-08
Filed on 2024-04-08
SYNTHETIC GBA1 GENES AND AAV VECTORS TO TREAT GAUCHER DISEASE
US Application 63/627,542
Filed on 2024-01-31
Filed on 2024-01-31
CODON-OPTIMIZED HUMAN NPC1 GENES FOR THE TREATMENT OF NIEMANN-PICK TYPE C1 DEFICIENCY AND RELATED CONDITIONS
US Application 18/816,498
Filed on 2024-08-27
Filed on 2024-08-27
SYNTHETIC MMAB GENES AND AAV VECTORS TO TREAT COBALAMIN B DEFICIENCY
US Application 63/499,013
Filed on 2023-04-28
Filed on 2023-04-28
IMPROVED GENE THERAPY CONSTRUCTS FOR THE TREATMENT OF PROPIONIC ACIDEMIA CAUSED BY MUTATIONS IN PROPIONYL-COA CARBOXYLASE ALPHA
US Application 63/294,511
Filed on 2021-12-29
Filed on 2021-12-29
Novel Vectors and Uses Thereof
US Application 62/907,718
Filed on 2019-09-29
Filed on 2019-09-29