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Latest From Jaguar Gene Therapy
Applied Therapeutics’ Pivotal Galactosemia Product Stalls, Confirming Analyst Concerns
Analysts’ fears have been confirmed as a US FDA demand for clinical outcomes data has forced Applied Therapeutics to delay a planned filing for its lead product candidate, AT-007, in the rare disease galactosemia based on biomarker data alone.
All Over For Servier's Autism Ambitions
The diuretic bumetanide had shown potential to improve the core symptoms of children and adolescents with autism in small studies but has failed to demonstrate any sign of effectiveness in two late-stage trials.
Tech Transfer Roundup: BridgeBio’s R&D Efforts Span New Seven New Agreements
Taysha gains control of GAN candidate and program; BI licenses COMT inhibitors from Lieber Institute; plus three new gene therapy-driven tie-ups between biopharma and academia.
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