Yissum - Research Development Company of the Hebrew University

Novel Treatment of Mitochondrial Disorders using Enzyme Replacement Therapy

Posted by Yissum - Research Development Company of the Hebrew UniversityResponsive · Innovative Products and Technologies · Israel

Summary of the technology

novel chimeric proteins comprising a membrane transferring moiety and an enzymatic moiety. These chimeric proteines may be used for treating mitochondrial diseases by using enzyme replacement therapy (ERT)
OLD: Enzyme Replacement Therapy for Mitochondrial Disorders
Project ID : 27-2006-382

Description of the technology


DNA mutations in mitochondrial proteins/enzymes (nuclear encoded) that may lead to disease are present in approximately 1 in 8,000 individuals.

Any treatment for mitochondrial diseases must be capable of targeting and crossing the cellular and the two mitochondrial membranes, as well as crossing the blood-brain barrier (BBB).

Our Innovation

  • Novel concept for treating mitochondrial diseases through enzyme replacement therapy (ERT) using a fusion protein made up of a protein transduction domain fused to a functional component of a mitochondrial enzyme. The innovation enable to replaces the mutated endogenous enzyme and restores the activity of the essential mitochondrial multi-component enzymatic complex (pyruvate dehydrogenase complex (PDHC)) to near normal enzymatic function.


  • Major opportunities in the many different mitochondrial diseases and other metabolic diseases
  • Many other diseases including Alzheimer’s, dementia, Parkinson’s, diabetes, hypertension, heart disease that are only just being understood to involve damaged mitochondrial enzymes


Novel fusion proteins enable ERT for the first time:

  • Novel approach to treating mitochondrial disease involves replacing mutated enzymes within the mitochondria in general, and in the context of replacing one mutated component to restore the activity of an immense multi-component enzymatic complex
  • No current known treatments or cures for mitochondrial disease.
  • The Lipoamide dehydrogenase (LAD) subunit was fused with the transactivator of transcription (TAT) peptide, which can rapidly cross the membranes. There are results also for Friedrich Ataxia as well as additional indications.
  • For the first time, demonstration of both introduction and functioning of a normal mitochondrial enzyme to replace a damaged enzyme, as well as replacement of one mutated component to restore the activity of an essential mitochondrial multicomponent enzymatic complex

Key Features

  • TAT capable of rapid crossing of biological membranes, enabling the TAT-LAD fusion protein to be delivered into cells and their mitochondria.
  • Concept proven in LAD deficiency and in Friedrich Ataxia; already applied to other mitochondrial and metabolic disorders
  • Development Milestones
  • Seeking investment and cooperation for production of the protein in large quantities, toxicity studies and clinical trails. Enlarge the scope of molecules for additional mitochondrial diseases and other metabolic disorders

Project manager

Keren-Or Amar
VP, Business Development, Healthcare

Project researchers

Haya Galski- Lorberboum
HUJI, School of Medicine - IMRIC
Biochemistry and Molecular Biology

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About Yissum - Research Development Company of the Hebrew University

Technology Transfer Office from Israel

Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Founded in 1964 to protect and commercialize the Hebrew University’s intellectual property. Ranked among the top technology transfer companies, Yissum has registered over 8,900 patents covering 2,500 inventions; has licensed out 800 technologies and has spun-off 90 companies. Products that are based on Hebrew University technologies and were commercialized by Yissum generate today over $2 Billion in annual sales.

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