Business, Policy & Funding
Breaking news on precision medicine business, policy & funding.
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.
UCLA, University College London Advancing Immune Disorder Gene Therapy Toward FDA Submission
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Researchers founded Rarity PBC as a public benefit corporation that will license the IP and develop operations to potentially market the gene therapy for ADA-SCID.
Medigene Selects KRAS G12V Candidate Within WuXi TCR-Guided T-Cell Engager Collaboration
The firms partnered earlier this year to leverage each other's technologies and develop multiple T-cell receptor-guided T-cell engagers.
BioArctic Doses First Parkinson's Patient in Trial of Alpha-Synuclein-Targeting Drug
Exidavnemab is a monoclonal antibody designed to eliminate aggregated clumps of the protein biomarker and slow progression of Parkinson's disease.
In particular, patients who had fewer than 10 annual vaso-occlusive events before the gene therapy were more likely to have none after treatment.