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Oficina francesa de transferencia de tecnología ofrece un nuevo tratamiento contra la atrofia muscular espinal

Resumen

Tipo:
Oferta Tecnológica
Referencia:
TOFR20180131001
Publicado:
10/04/2018
Caducidad:
11/04/2019
Resumen:
Una oficina francesa de transferencia de tecnología ofrece un nuevo tratamiento contra la atrofia muscular espinal (SMA), una enfermedad rara para la que no existe tratamiento. Este nuevo tratamiento utiliza flunarizina y se basa en la relocalización de la proteína de supervivencia de neuronas motoras (SMN) en cuerpos de Cajal para la restauración de su función y mejora del fenotipo de la atrofia muscular espinal. La oficina de transferencia de tecnología busca compañías farmacéuticas o biotecnológicas con el fin de establecer acuerdos de licencia o cooperación técnica para continuar el desarrollo.

Details

Tittle:
A French technology transfer office offers new treatment against spinal muscular atrophy
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
IPs:
Patents granted
CommeR Statunts Regarding IPR Status:
International patent application (Europe, US and Canada).
Publication date: March 2014.

Partner sought

Type and Role of Partner Sought:
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.

Client

Type and Size of Client:
Industry SME 11-49
Already Engaged in Trans-National Cooperation:
Si
Languages Spoken:
English
French

Keywords

Technology Keywords:
06003002 Expresión genética, investigación proteómica
06002004 Ingeniería de proteínas
06001012 Investigaciones médicas
06001014 Neurología, investigación cerebral
06001015 Productos farmacéuticos / medicamentos