iPS cell derivedmyogenic progenitor cells (MYOPAX) for the treatment of muscular dystrophies

Rita Perlingeiro, University of Minnesota

29th April, 2017

This is a proposal from Dr. Rita Perlingeiro and colleagues at the University of Minnesota to develop allogeneic HLA-matched iPSC cell-based therapy (MYOPAX) for Duchenne muscular dystrophy (DMD).  The proposal describes IND-enabling studies and plans for a first-in-human Phase 1 safety/dose escalation trial of pluripotent-derived skeletal myogenic progenitors for DMD.

In vivo validation studies utilizing the optimized preclinical product will be conducted in the mdx mouse.  An open label, dose escalation, phase 1 trial to determine the safety, feasibility and MTD of intramuscular injection of MYOPAX in adults with DMD is planned.

TACT welcomed the fact that this approach has the potential to treat DMD of all genotypes as well as many related disorders. The committee felt that the program progresses through well-defined milestones with clear go-no go criteria. The report makes recommendations on further pre-clinical studies to be performed as well as confirmation of some pre-clinical data by an independent laboratory. TACT also makes recommendations on the inclusion criteria, study protocol and consultation with additional experts including physiotherapists.

To request a full copy of the report please contact the applicant Rita Perlingeiro:

12 Oct 2017