by Peter Ciszewski | Feb 14, 2019
The Food and Drug Administration’s (FDA’s) Division of Neurology has accepted its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory action date of August 19, 2019. Golodirsen is a phosphordiamidate...
by Peter Ciszewski | Feb 14, 2019
Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses his company’s first approved product, Luxturna — a one-time gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal...
by Peter Ciszewski | Feb 12, 2019
Pushkal Garg, MD, Chief Medical Officer at Alnylam discusses acute hepatic porphyria (AHP) and givosiran, an investigational, subcutaneously administered RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of AHP....
by Peter Ciszewski | Feb 11, 2019
Mohamad Mohty, MD, PhD, Professor of Hematology at Sorbonne University and Head of the Hematology and Cellular Therapy Department at the Saint Antoine Hospital in Paris discusses some of the exciting presentations at this year’s American Society of...
by Peter Ciszewski | Feb 8, 2019
Manuel Litchman, MD, President, Chief Executive Officer at Mustang Bio discusses his company’s X-linked severe combined immunodeficiency (SCID) program. X-linked SCID is an inherited disorder of the immune system that occurs almost exclusively in males....
