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- ARPA-H allocates up to $125 million to accelerate personalized RNA therapy manufacturingARPA-H announced a new program, called GIVE, that will provide up to $125 million in research funding to develop faster, decentralized manufacturing methods for individualized RNA‑based genetic med…
- Ultragenyx gene therapy for Angelman syndrome fails Phase 3 trial, company to cut expensesAn experimental gene therapy targeting Angelman syndrome did not meet its primary endpoint in a Phase 3 study, according to Ultragenyx. The trial showed no difference between the therapy, GTX-102,…