Targeting Calpain Inhibitors in Neuromuscular Diseases
Award last edited on: 5/24/2023

Sponsored Program
Awarding Agency
Total Award Amount
Award Phase
Solicitation Topic Code
Principal Investigator
Alfred Stracher

Company Information

Abeona Therapeutics Inc (AKA: PlasmaTech Biopharmaceutcials Inc~Access Pharmaceuticals Inc~Chemex Corporation~Chemex Pharmaceuticals Inc)

1330 Avenue of the Americas 33rd Floor
New York, NY 20029
   (214) 905-5100

Research Institution

State University of New York

Phase I

Contract Number: 1R41NS039222-01
Start Date: 9/26/2000    Completed: 9/25/2002
Phase I year
Phase I Amount
Based on previous studies using the calpain inhibitor leupeptin (acetyl-leucyl-leucyl-arginal) to inhibit muscle wasting in animal models of muscular dystrophy and denervation atrophy, the investigators have devised a unique method by which the active portion of the leupeptin molecule can be directly targeted to the affected tissue by chemically linking it to an appropriate carrier. Molecules which are normally concentrated in a tissue by active transport 20-100 fold their plasma levels such as carnitine in muscle and taurine in nerve cells can act as such carriers. They have shown, in preliminary studies, that the chemical entity carnityl-leucyl-argininal is at least 13 fold more effective in inhibiting calpain intracellularly in skeletal muscle than is leupeptin alone. The purpose of these studies will be to expand on these initial observations towards the development of such compounds as therapeutic agents in the treatment of a variety of neuromuscular and neurodegenerative disorders.

Thesaurus Terms:
calpain, drug delivery system, drug design /synthesis /production, enzyme inhibitor, neuromuscular disorder carnitine, muscle necrosis, taurine laboratory mouse, laboratory rat

Phase II

Contract Number: ----------
Start Date: 00/00/00    Completed: 00/00/00
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