Partnering opportunity

Curative drug for genetic diseases due to nonsense mutation to be transfered by a French Technology Transfer Office


A French Technology Transfer Office (TTO) offers an new treatment dealing with the primary cause of genetic diseases due to nonsense mutation. It turns the protein with such a mutation into a complete functional protein, which allows to consider a full recovery. The French TTO is looking for pharmaceutical corporates or biotech companies interested by a licensing or a technical cooperation agreement. Investors operating in biotechnology are also targeted in the context of a financial agreement.

Partner sought

The partner sought could be a pharmaceutical corporate, biotech company or startup operating in the drug design for genetic diseases due to nonsense mutation (cystic fibrosis or others), which may be interested by a licence agreement. These companies may also be interested by a technical cooperation agreement in case of further development or co-development, such as in vivo trials dealing with other genetic disease different from cystic fibrosis. Moreover, investors operating in biotechnology could be interested by a financial agreement in the context of creating a new startup company.


A Technology Transfer Office (TTO) is acting on behalf some major life sciences research labs operating in the Paris region. One of them has demonstrated the mechanism of action of a new orally-administered molecule as a curative treatment for genetic disorders due to nonsense mutations. The proof of concept has been performed on cystic fibrosis, and is under investigation for other nonsense mutations. *Definitions : - The Messenger Ribonucleic Acid (mRNA) is a temporary and unfinished copy of the Desoxyribonucleic Acid (DNA) which lies in nucleus cells and contains all genetic properties of a whole living subject, - the ribosom is the cell part which transcripts the mRNA and produces a full-length protein according to the transcripted information, - a nonsense mutation is the resulting point from a failing DNA where genetic information is missing and so-replaced by a premature stop signal in the mRNA. *Market challenges : Nonsense mutations are responsible for 10% of all genetic diseases, mainly for cystic fibrosis whose symptoms are acute respiratory failure and digestive capacity impairment. The number of patients with this severe chronic disease is about 30,000 in United States and 40,000 in Europe (7,000 in France). Until now, there is no curative treatment but only symptomatic relief procedures which needs a strong coordination between several medical specialties such as physiotherapy, nutrition and clinical psychology. * State of the art : Among all genetic diseases due to nonsense mutation, Duchenne Muscular Dystrophy (DMD) is the only one to benefit from an existing curative treatment using ataluren drug. Nevertheless, clinical investigation using ataluren hasn't shown any significant impact on other nonsense mutation diseases. Indeed, the biological target of ataluren is not known, and its mechanism of action is uncertain : it is thought to make ribosomes less sensitive to premature stop signals but it hasn't been proved until now. * Proposed technology : A new ingredient has been extracted from a specific fungus, and used as the active principle of an innovative drug. Applied to cystic fibrosis, it has been proved to enable the ribosom to correctly transcript the mRNA without any disruption due to nonsense mutation. Doing so, it allows the production of a full-length and functional protein. The French TTO is looking for pharmaceutical corporates, biotech companies and startups interested by a licensing agreement. A technical cooperation agreement is also open to be discussed in case of further investigation on cystic fibrosis or other genetic disesases due to nonsense mutation. Investors operating in biotechnology are also sought in case they are interested in a startup creation. *Keywords : genetic disorder, nonsense mutation, exon skipping, cystic fibrosis.

Advantages and innovations

The innovative drug enables the ribosom to correctly transcript the mRNA without any disruption due to nonsense mutation. Doing so, it can be used as a treatment for genetic diseases due to nonsense mutation such as cystic fibrosis. *Advantages : - Compared to ataluren, the new drug is 2.5 times more efficient when used as an indication for cystic fibrosis, leading to a 80% production ratio of full-length and functional proteins (see figure), - the new drug delivery process is much patient-friendly as compared to ataluren (oral pill versus needle injection), - the active principle is easily available, since the fungus extract as well as the molecule have been clearly identified, - the mechanism of action could be transfered to other genetic diseases due to nonsense mutation.

Development stage

Under development/lab tested

Intellectual Property Rights (IPR)

Patent(s) applied for but not yet granted

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.

?What value does EEN add?
This is your pitch: remember to include your unique selling points (USP) and why someone would want to do business with you
Tell us why you are a good fit for this opportunity, and why you think you're the right people for this partnership
If there's anything additional, or commercially sensitive you'd like to know about this opportunity, please let us know

When you entered your email address above, we emailed you a code to verify you're human and have access to that account.

?What's this?

What next?

To finish up, we just need some extra details about you and your company