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Amyotrophic lateral sclerosis gene therapy - Specific Biologics

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Drug Profile

Amyotrophic lateral sclerosis gene therapy - Specific Biologics

Latest Information Update: 26 Apr 2026

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At a glance

  • Originator Specific Biologics
  • Class Gene therapies
  • Mechanism of Action C9orf72 protein replacements; 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 No

Highest Development Phases

  • Preclinical Amyotrophic lateral sclerosis

Most Recent Events

  • 01 Apr 2026 Preclinical trials in Amyotrophic lateral sclerosis in USA (Parenteral)

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