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X-linked severe combined immunodeficiency gene therapy program - Mustang Bio

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Drug Profile

X-linked severe combined immunodeficiency gene therapy program - Mustang Bio

Alternative Names: CL20-4i-EF1alpha- HgammaC-OPT; CL20-i4-EF1α-hγc-OPT; Ex vivo gene therapy - Mustang Bio; Gene-modified CD34+ hematopoietic stem cells; IND-14570; Lentiviral genetherapy; MB 207; MB-107; MB-107 XSCID gene therapy; MB-117; MB-217; VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT; X-linked severe combined immunodeficiency gene therapy - Mustang Bio; XSCID gene therapy

Latest Information Update: 07 Feb 2024

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At a glance

  • Originator St. Jude Childrens Research Hospital
  • Developer Mustang Bio; National Heart, Lung and Blood Institute; National Institute of Allergy and Infectious Diseases; National Institutes of Health Clinical Center; Seattle Children's Hospital; St. Jude Childrens Research Hospital; UCSF Benioff Childrens Hospital
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    Yes - Severe combined immunodeficiency
  • New Molecular Entity No

Highest Development Phases

  • Phase II Severe combined immunodeficiency

Most Recent Events

  • 30 Jan 2024 National Institute of Allergy and Infectious Diseases in collaboration with National Institutes of Health Clinical Center reinitiates the enrollment in a phase I/II clinical trial for Severe combined immunodeficiency (In children, In adolescents, In adults, Treatment-experienced) in USA (IV) (NCT01306019)
  • 19 Apr 2023 St. Jude Children's Research Hospital suspends phase-I/II trial in Severe combined immunodeficiency (In neonates, In infants, Newly diagnosed) in USA (IV) (NCT01512888)
  • 26 May 2022 National Institute of Allergy and Infectious Diseases in collaboration with National Institutes of Health Clinical Center suspends a phase I/II clinical trial in Severe combined immunodeficiency (In children, In adolescents, In adults, Treatment-experienced) in USA (IV) (NCT01306019)

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