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- Agios drops next-gen sickle cell drug after Phase 2 data show no advantage over Novo’s candidateAgios Pharmaceuticals said it is halting development of its next‑generation pyruvate kinase activator, tebapivat, for sickle cell disease after a mid‑stage trial failed to demonstrate a benefit ove…
- Broad Institute, Boston Children’s Hospital and Jackson Laboratory launch nonprofit to advance affordable gene therapies for rare diseasesThe Broad Institute, Boston Children’s Hospital and Maine’s Jackson Laboratory announced a new collaboration to develop gene‑based treatments for rare genetic disorders. The three institutions will…STAT · 23d agoDealPositive