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Research programme: small molecules therapeutics - GABAeron

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Drug Profile

Research programme: small molecules therapeutics - GABAeron

Latest Information Update: 28 Mar 2026

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At a glance

  • Originator GABAeron
  • Class Small molecules
  • Mechanism of Action Sterol modulators
  • 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

  • No development reported Smith-Lemli-Opitz syndrome

Most Recent Events

  • 28 Mar 2026 No recent reports of development identified for preclinical development in Smith-Lemli-Opitz-syndrome in USA
  • 01 Feb 2022 Preclinical trials in Smith-Lemli-Opitz syndrome in USA before February 2022 (GABAeron pipeline, February 2022)

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