A French technology transfer office offers new treatment against spinal muscular atrophy
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.
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.
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.
Available for demonstration
Intellectual Property Rights (IPR)
Register your interest
How it works
- Tell us about yourself
- We’ll discuss with you
- We put the right partners in touch
EEN help you find the right partner, rather than you going it alone.
Our role is to review and collate the most suitable submissions, and then send them to the client who posted the opportunity. We consult with you, and the client, to make the process professional and easy.
These are live opportunities. Your registration of interest on the site is just like a professional approach to a business at a networking event. To stand the best chance of success, make your submission really sing. Sell why the client who posted the opportunity should work with you. Excite them. Ask questions. Try and avoid copy and pasting words from elsewhere.
Once the client has chosen their partner, we'll introduce them over email and keep in touch with both parties to see how it's going. Sometimes things progress quickly. Sometimes because of changing priorities for either party, things progress slowly, but you never know - your next big business break could start right here.
First we need to check you’re human.
Thank you for verifying your email. We have sent you a confirmation email containing a 6 digit verification code to unlock the form below