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Research programme: CRISPR/Cas9-based stem cell therapies - Intellia Therapeutics/Novartis

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Drug Profile

Research programme: CRISPR/Cas9-based stem cell therapies - Intellia Therapeutics/Novartis

Alternative Names: ADPT 03 programme

Latest Information Update: 28 Mar 2023

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At a glance

  • Originator Intellia Therapeutics
  • Class Antianaemics; Gene therapies; Stem cell therapies
  • Mechanism of Action Cell replacements; Fetal haemoglobin expression stimulants
  • 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

  • Discontinued Sickle cell anaemia

Most Recent Events

  • 23 Feb 2023 Discontinued - Preclinical for Sickle cell anaemia in USA (IV)
  • 19 Jan 2023 Novartis plans to file regulatory application for ADPT 03 in Sickle cell anaemia in or after 2026 (Novartis pipeline, January 2023)
  • 28 Feb 2022 The US Patent and Trademark Office issues decision in favor of Broad Institute for interference claim for patent related to CRISPR-Cas9 genome editing technology in USA

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