Drug Profile

Research programme: NAV AAV9 vector based gene therapies - Pfizer

Alternative Names: NAV Technology-based gene therapies - Pfizer

Latest Information Update: 28 Jan 2025

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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 No

28 Jan 2025 No recent reports of development identified for preclinical development in Friedreich's ataxia in USA (Parenteral)
31 Dec 2020 Preclinical trials in Friedreich's ataxia in USA (Parenteral)
31 Jul 2019 Pfizer in-licenses NAV AAV9 vector based technology from REGENXBIO for treatment of Friedreich's Ataxia

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