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A French technology transfer office offers new treatment against spinal muscular atrophy

Country of Origin: France
Reference Number: TOFR20180131001
Publication Date: 29 March 2018

Summary

A French technology transfer office (TTO) offers new treatment against spinal muscular atrophy (SMA), a rare disease without any available treatment.  This innovative treatment uses flunarizine for the relocation of the survival motor neuron (SMN) protein into an accessory body in neurons named Cajal body.
The TTO is looking for pharmaceutical corporates or biotech companies, either for a licensing agreement or a technical cooperation agreement in case of further development.

Description

A French technology transfer office (TTO) is acting on behalf of an established public laboratory of a university of the Paris region.

* Market challenges :
Spinal muscular atrophy (SMA) is a neurodegenerative disorder leading to progressive muscle weakness, atrophy, and in severe types, premature death. It is classified as a rare disease affecting 1 in 10,000 births to 1 in 6,000 births worldwide.

SMA is caused by the mutation or deletion of the survival motor neuron-1 (SMN1) gene, which encodes the survival motor neuron (SMN) protein, necessary for the survival of motoneurons.

* State of the art :
Some drug candidates aim at motoneuron protection or SMN protein levels increase. However, until now, no treatment is available against SMA.

Studies have shown that SMN protein is found in an accessory body in neurons named Cajal body but its presence is affected in SMA patients.

* Proposed technology :
The present offer relates to a new treatment based on relocation of SMN Protein into Cajal bodies to restore their function and improve the SMA phenotype.

Through an in vitro screening on SMA patients’ cells using many drugs whose therapeutic potential is under investigation, flunarizine has been identified as a the only hit candidate to relocate and restore SMN function. It is a drug classified as a calcium antagonist which is prescribed for various uses like migraine.

In vivo studies in mice show relocation of SMN into Cajal bodies as well as a strong improvement of survival and mobility. More recent studies show effects on both muscles and neurons (results available under non-disclosure agreement) and ongoing research will elucidate the details of the mechanism of action.

The TTO acting on behalf of this university is looking for corporates, medium-sized companies or startups operating in drug design or biotechnology development. These companies are focused in order to set up a licensing agreement, or a technical cooperation agreement in case of further development.

Key words :
Spinal muscular atrophy, motor neuronopathy, neurodegenerative disorder, SMA, SMN, flunarizine, motor neuron, Cajal body

Advantages and Innovations

* Innovation :
Flunarizine has been identified as the only hit candidate against spinal muscular atrophy (SMA) with exclusive capacities of relocating and restoring survival motor neurones (SMN) function.

* Advantages :
- Flunarizine is a well known compound and a drug classified as a calcium antagonist which is prescribed for various uses like migraine,
- flunarizine can be used in combination with treatment aiming to increase SMN levels.

Stage Of Development

Available for demonstration

Stage Of Development Comment

In vivo evidence of survival motor neurones (SMN) relocation into Cajal bodies following flunarizine treatment has been shown.

In vivo evidence of muscular default correction following flunarizine treatment has also been shown.

Besides, the neuroprotective effect is under ongoing analysis, including studies of both molecule and mechanism.

Requested partner

The partner sought could be a large corporate, medium-sized company or startup operating in pharmaceutical, drug design or biotechnology development.

These companies are focused in order to set up a licensing agreement, or a technical cooperation agreement in case of further needed development to meet industrial and clinical challenges.

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