Venglustat, a glucosylceramide synthase inhibitor (GCSi), works by reducing the abnormal buildup of sugar-and-fat molecules in cells and organs
The approval is grounded in data from a Phase III randomized, double-blind, placebo-controlled, crossover study in NPC patients
Contera Pharma will receive an upfront payment and full research funding for each target under investigation
Skyhawk Therapeutics enters into an option agreement to grant Merck KGaA, Darmstadt, Germany, exclusive global rights to drug candidates pursued under the collaboration upon option exercise
AbCellera will receive an upfront payment and is eligible to receive additional milestone payments
The global VR market to grow at a compound annual growth rate of 13% from $7 billion in 2018 to $28 billion in 2030.
Agreement builds on previous Novartis success using emerging technologies to develop first-in-class gene therapies for neurological disorders
Entos will receive an initial payment of US $50 million, which includes an equity investment by Lilly
HD causes cognitive, behavioral, and physical challenges, including involuntary movements known as HD chorea, which can disrupt daily life
The Phase 1 single and multiple ascending dose study is set to begin dosing in early Q1 2026
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