Gene therapy digest - 2021

Gene therapy digest - 2021

PRIME to boost Mustang Bio’s MB-107 before pivotal trial in XSCID

MB-107 (Lentiviral gene therapy; Phase 2) – Mustang Bio/ Fortress Biotech

  • EMA granted Priority Medicines designation to MB-107 for the treatment of X-linked severe combined immunodeficiency (“XSCID”) in newly diagnosed infants
  • Other designations

Regulatory body

Designation

Year

EMA

Advanced therapy medicinal product (ATMP)

Apr 2020

ODD

Nov 2020

FDA

ODD, Rare pediatric disease

Sep 2020

RMAT

Aug 2019

  • Lentiviral vector based MB-107 is currently being assessed in a Phase 1/2 clinical trial for XSCID in newly diagnosed infants and in a Phase 1/2 clinical trial for XSCID patients previously treated with HSCT and for whom re-treatment is indicated (For full story click here)

Mustang Bio plans to enroll first patient in MB-107 pivotal trial in the Q2 2021

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CI Scientists Commentary:

  • After ODD and Rare pediatric disease designation for MB-107, Mustang Bio was all set for initiation of pivotal trial for XSCID in newly diagnosed infants
  • But FDA imposed a clinical hold in Jun 2020 citing need for data on CMC which delayed the timelines for this therapy
  • The clinical hold was withdrawn in Feb 2021. This was followed by a PRIME designation in EU. Mustang bio with this stroke of good luck will accelerate its Phase 2 pivotal trial
  • MB-107 was sourced by Fortress Biotech and is currently being developed by its partner, Mustang Bio

– Dr. Kowndinya, CI Scientists

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