Research programme: ophthalmologic gene therapy - Adverum/Regeneron

Drug Profile

Research programme: ophthalmologic gene therapy - Adverum/Regeneron

Alternative Names: AVA 311

Latest Information Update: 13 Jul 2016

Price : $50

At a glance

  • Originator Avalanche Biotechnologies; Regeneron Pharmaceuticals
  • Developer Adverum Biotechnologies; Regeneron Pharmaceuticals
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Retinoschisis

Most Recent Events

  • 24 Nov 2015 ERS Genomics has patent protection for CRISPR-Cas9 patent portfolio in US
  • 24 Nov 2015 CRISPR-Cas9 genome-editing patents licensed to Regeneron Pharmaceuticals
  • 24 Aug 2015 Preclinical trials in Retinoschisis in USA (Intraocular) (Avalanche 10-K, August 2015)
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