DelveInsight's Pancreatic Cancer Market Insights report includes a comprehensive understanding of current treatment practices, ...
The 13 partners hope their research into establishing a universal test can help save time and money for therapy developers.
The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...
ROME, Italy, Dec. 10, 2025 /PRNewswire/ -- Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex ...
EpilepsyGTx previously raised $10 million in pre-seed and seed funding led by the UCL Technology Fund, managed by AlbionVC in collaboration with UCL Business, the commercialisation company of UCL with ...
Overall response rate (ORR) of 55% and median progression-free survival of 14.5 months in patients without TP53 gene mutationPhase 2 results ...
Based on initial positive results from HOPE-B, etranacogene dezaparvovec became the first gene therapy approved for adults ...
Waskyra, developed by Fondazione Telethon, is the first gene therapy that the FDA has approved from a non-profit applicant.
Friedreich’s ataxia is a genetic disorder that causes progressive damage to the nervous system and brain, leading to a loss ...
The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...
The three-hit model, developed from more than a decade of systems biology research, proposes that autism develops when three ...
FDA approves 1st gene therapy for Wiskott-Aldrich syndrome, offering a new treatment for patients lacking matched stem cell donors.
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