IP protection



Please provide a short description of the state-of-the-art and/or current trends in the field? How does the result fit into it?

There’s currently no cure for any form of muscular dystrophy. Research into gene therapy may eventually provide treatment to stop the progression of some types of muscular dystrophy. Current treatment is designed to help prevent or reduce deformities in the joints and the spine and to allow people with muscular dystrophy to remain mobile as long as possible.

Corticosteroids, such as prednisone, may help improve muscle strength and delay the progression of certain types of muscular dystrophy. But prolonged use of these types of drugs can weaken bones and increase fracture risk. Several different types of therapy and assistive devices can improve quality and sometimes length of life in people who have muscular dystrophy, including range-of-motion exercises, mobility aids, breathing assistance and surgery.

A drug called AVI-4658 allows to the cellular machinery to “skip” over the genetic region containing the mutation, allowing a shortened but functional version of the dystrophin protein to be produced in patients with Duchenne muscular dystrophy.

To our knowledge, our proposal is the first aiming to cure muscular dystrophy by inhibiting proteolysis of the muscular structural proteins. This initial step in turn produce substrates of main proteolytic pathways and lead to muscle fiber degeneration.



What is the problem/need/knowledge gap that the research result is responding to?  How was it addressed before?

Muscular dystrophies are devasting conditions leading often to full dependence and premature death. Present treatment aims to improve the quality of patient’s life but direct  curing is unknown. Therefore there is a pressing need for the elaboration a knowledge-based therapy.

Present therapies are basically clinical, pharmacologic therapy is confined to a single condition, Duchenne muscular dystrophy. ICD-10 G71.0 consists of several dystrophic disorders as follows: autosomal recessive, childhood type, resembling Duchenne or Becker; benign (Becker); benign scapuloperoneal with early contractures (Emery-Dreifuss); distal Facioscapulohumeral; limb-girdle; ocular; oculopharyngeal; scapuloperoneal; severe (Duchenne) as clinically distinct disorders. G71.1 describes further (myotonic) disorders: Dystrophia myotonica (Steinert); Myotonia chondrodystrophic, drug-induced, symptomatic; dominant (Thomsen); recessive (Becker); neuromyotonia (Isaacs); paramyotonia congenital; pseudomyotonia. All these conditions are characterized by muscle fiber degeneration as basic mechanism of muscle wasting.

Our proposal aims a radically new approach by inhibiting the initial step of muscle degeneration-wasting.



What is the potential for further research?
We aim to address basic pathognomic conditions of a devasting disorder, muscular dystrophy. Our results will definitely disseminate muscular dystrophy research and open new horizons on the field.



What is the proposed method of IPR-protection? (patent, license, trademark etc.)

For IPR-protection we suggest patenting the results at national/international levels. The phase PCT 1 has to be announced in the year 2012. When the product development is completed and the market possibilities are investigated as well as potential partnerships the project owner has to decide the further steps of patenting.




What are the steps that need to be taken in order to secure the IPR-protection? What is the cost of IPR-protection?
The cost of the novelty survey and achieving the PCT 1 phase in Europe is € 4000 



What is you overall assessment of the scientific maturity of the research result?
Our research provided a well-established hypothesis that progression of muscular dystrophy relies on initial proteolytic cleavage of bulk muscle proteins. These results are based on solid genetic, molecular, cell biology and biochemical basis. Inhibiting of the initial proteolytic cleavage hampers all subsequent steps leading to muscle fiber degeneration and wasting by degradation protein fragments proteolytically.




Please put X as appropriate. 1 2 3 4 5
Scientific maturity X
Synergies X
State-of-the-art/innovation X
IPR-potential X


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