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ASC 618

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Drug Profile

ASC 618

Alternative Names: Adeno-associated viral vector haemophilia A gene therapy - ASC Therapeutics; ASC-618; CRISPR/cas9 edited human factor VIII - ASC Therapeutics

Latest Information Update: 16 Dec 2024

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At a glance

  • Originator ASC Therapeutics
  • Class Antihaemorrhagics; Blood coagulation factors; Gene therapies
  • Mechanism of Action Factor VIII replacements; Gene transference
  • Orphan Drug Status

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

    Yes - Haemophilia A
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Haemophilia A

Most Recent Events

  • 11 Dec 2024 Adverse events and efficacy data from a phase I/II trial Haemophilia A released by Expression Therapeutics
  • 31 Aug 2022 Phase-I/II clinical trials in Haemophilia A in USA (Parenteral) (NCT04676048)
  • 16 May 2022 ASC 618 receives Orphan Drug status for Haemophilia A in Europe prior to May 2022

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