Research programme: Friedreich's ataxia gene therapies - Adverum Biotechnologies

Drug Profile

Research programme: Friedreich's ataxia gene therapies - Adverum Biotechnologies

Alternative Names: AAVrh 10

Latest Information Update: 02 May 2018

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At a glance

  • Originator Annapurna Therapeutics; Institut de Genetique et de Biologie Moleculaire et Cellulaire
  • Developer AAVLife; Institut de Genetique et de Biologie Moleculaire et Cellulaire
  • Class Gene therapies
  • Mechanism of Action Frataxin expression stimulants; Gene transference
  • 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

Highest Development Phases

  • Preclinical Friedreich's ataxia

Most Recent Events

  • 01 Apr 2018 Adverum Biotechnologies terminates an exclusive license agreement with REGENXBIO for Friedreich's ataxia using NAV®rAAVrh10 vector delivery
  • 29 Apr 2014 AAVLife and ReGenX Biosciences enters into an exclusive license agreement for Friedreich's ataxia using NAV®rAAVrh10 vector delivery
  • 06 Apr 2014 Preclinical trials in Friedreich's ataxia in France (Intravenous)
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