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ACTUS 101

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Drug Profile

ACTUS 101

Alternative Names: AAV2/8-LSPhGAA; ACTUS-101; Pompe Disease rAAV Gene Therapy - Bayer

Latest Information Update: 05 Nov 2023

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At a glance

  • Originator Duke University
  • Developer Actus Therapeutics; Duke University; National Institute of Arthritis and Musculoskeletal and Skin Diseases
  • Class Gene therapies
  • Mechanism of Action Alpha-glucosidase 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

  • Phase I/II Glycogen storage disease type II

Most Recent Events

  • 17 Mar 2021 Phase-I/II trials is still ongoing for Glycogen storage disease type II in USA (IV) (NCT03533673)
  • 01 Nov 2018 Phase-I/II clinical trials in Glycogen storage disease type II in USA (IV) (NCT03533673)
  • 14 Dec 2017 Phase-I clinical trials in Glycogen storage disease type II in USA (IV) (NCT03533673)

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