Drug Profile

Research programme: AAV gene therapies - Novarits

Latest Information Update: 28 Apr 2026

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

28 Apr 2026 No recent reports of development identified for research development in CNS-disorders in USA (Parenteral)
02 Jan 2024 Voyager Therapeutics expands its capsid license agreement with Novartis to advance gene therapies for Huntington’s disease and Spinal muscular atrophy
02 Jan 2024 Preclinical trials in Huntington's disease in USA (Parenteral)

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