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genetic diseases
Biotech
Denali ph. 1 Pompe plans hit with clinical hold
Denali does not expect the hold to delay its plans, and still intends to submit a clinical trial application in Europe in the first half of 2026.
Darren Incorvaia
Dec 4, 2025 11:52am
Pharvaris clocks a pivotal win in race for rapid HAE relief
Dec 3, 2025 9:10am
Genetic medicines biotech halves team
Nov 20, 2025 10:15am
Harmony halts genetic disease program after phase 3 failure
Nov 4, 2025 12:45pm
BridgeBio's calcium deficiency candidate scores phase 3 win
Oct 29, 2025 7:00am
Chiesi fertilizes Arbor's gene therapy with $2B biobucks deal
Oct 6, 2025 7:00am
