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ABO 202

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Drug Profile

ABO 202

Alternative Names: AAV-CLN1 - Abeona Therapeutics; ABO-202; scAAV-CLN1; TSHA-118

Latest Information Update: 02 Apr 2024

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At a glance

  • Originator University of Edinburgh; University of North Carolina at Chapel Hill
  • Developer Abeona Therapeutics; Taysha Gene Therapies; University of North Carolina at Chapel Hill
  • Class Gene therapies
  • Mechanism of Action Gene transference; Palmitoyl-protein thioesterase replacements
  • Orphan Drug Status

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

    Yes - Neuronal ceroid lipofuscinosis
  • New Molecular Entity No

Highest Development Phases

  • Suspended Neuronal ceroid lipofuscinosis

Most Recent Events

  • 18 Mar 2024 Abeona Therapeutics has patents pending for 'optimized CLN1 genes and expression cassettes for use in treating CLN1 disease' and 'treating CLN1 disease using a combination of intrathecal and intravenous administrations' in the US, Europe and other geographical regions
  • 10 May 2023 Suspended - Phase-I/II for Neuronal ceroid lipofuscinosis in Canada (IV) due to limited financial and management resources
  • 10 May 2023 Suspended - Phase-I/II for Neuronal ceroid lipofuscinosis in USA (IV) due to limited financial and management resources

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