rare disease
In Brief This Week: Lantern Pharma, AstraZeneca, Genesupport, UniQure, PanGIA Biotech
News items for the week of Dec. 9, 2024.
SpliceBio to Kick off Phase I/II Trial of Stargardt Disease Gene Therapy in 2025
SpliceBio aims to begin a study testing its protein splicing gene therapy in the first half of 2025, now that the FDA has cleared its IND.
Researchers at the meeting reported on long-term activity of commercialized gene therapies and preliminary data on investigational candidates.
Parents are suing the defendants for negligent genetic counseling and testing that they allege precluded their ability to avoid having children with Duchenne.
Krystal Biotech Expects CHMP Opinion on Skin Condition Gene Therapy in Early 2025
The firm is on track to launch the gene therapy for dystrophic epidermolysis bullosa, called Vyjuvek in the US, in Germany in Q2 2025.
Oct 24, 2024