January 2023 Recap: Drug Pipeline Updates
The table provided is a review of notable updates that occurred in January 2023 for investigational products in development.
The table provided is a review of notable updates that occurred in January 2023 for investigational products in development.
Olezarsen is an investigational LICA antisense medication designed to inhibit the production of apoC-III for patients at risk of disease due to elevated triglyceride levels.
AOC 1020 is designed to reduce the expression of DUX4 mRNA and DUX4 protein in muscles.
The table provided is a review of notable updates that occurred in December 2022 for investigational products in development.
Vamorolone is a first-in-class dissociative steroidal drug that retains the anti-inflammatory activity of corticosteroids but with a novel mechanism of action.
The application was approved through the FDA’s 505(b)(2) regulatory pathway.
Bitopertin is an investigational oral, selective inhibitor of glycine transporter 1 designed to modulate heme biosynthesis by limiting glycine uptake.
Pegunigalsidase alfa is a long-acting recombinant, PEGylated, cross-linked α-galactosidase-A investigational product candidate.
SRP-9001 is an investigational gene transfer therapy designed to deliver a shortened, functional component of dystrophin to muscle tissue.
The table provided is a review of notable updates that occurred in October 2022 for investigational products in development.