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Drug Profile


Alternative Names: AAV-hUPF1; AAV-UPF1

Latest Information Update: 23 Apr 2024

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At a glance

  • Originator Brandeis University
  • 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 Amyotrophic lateral sclerosis

Most Recent Events

  • 23 Apr 2024 Preclinical development in Amyotrophic-lateral-sclerosis is ongoing in USA (Intrathecal, Injection) (MeiraGTx pipeline, April 2024)
  • 31 Dec 2023 MeiraGTx has patent protection for ALS gene therapy program and the AAV-UPF1 in USA, Austria, Australia, Belgium, Denmark, France, Germany, Hong Kong, Ireland, Italy, Netherlands, Norway, Spain, Sweden, Switzerland/Liechtenstein and United Kingdom before December 2023
  • 31 Dec 2023 MeiraGTx has patents pending for ALS gene therapy program and the AAV-UPF1 in USA, Canada, Europe, Hong Kong before December 2023
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