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Leber congenital amaurosis- gene therapy - IVERIC bio

Drug Profile

Leber congenital amaurosis- gene therapy - IVERIC bio

Alternative Names: mini-CEP290; mini-CEP290 Iveric; mini-CEP290: LCA10

Latest Information Update: 01 Dec 2023

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At a glance

  • Originator University of Massachusetts Medical School
  • Developer IVERIC bio
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Cep290 protein modulators; Gene transference
  • Orphan Drug Status

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

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Leber congenital amaurosis

Most Recent Events

  • 28 Aug 2023 Preclinical studies in Leber congenital amaurosis in USA (unspecified route) (Iveric Bio pipeline, August 2023)
  • 11 Jul 2023 IVERIC bio has been acquired by Astellas Pharma
  • 24 Mar 2023 IVERIC bio has patents pending for eye disorder gene therapies in USA, the EPO Countries, China, Japan and certain other jurisdictions, prior to December 2022
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