French company developing new targeting of enzyme therapies for lysosomal disease is seeking partners with complementary approaches
A French start-up created in 2012 develops new therapeutic approach based on membrane receptor targeting and nanomaterials. The technology allows the targeting of drugs to lysosomes for therapies of lysosomal storage diseases or cancer. The company would like to establish new partnerships to develop lysosomal disease treatments combining enzyme replacement therapy with other therapeutics (gene therapy, chaperone,...) in order to make a joint proposal for EJP RD JTC2020 call for project.
The company has developed new targeted therapeutics to treat Lysosomal Storage Disorders. Thanks to the company's proprietary synthetic derivatives, they provide a compound with high affinity for the mannose 6-phosphate receptor and resistant to phosphatases. These compounds can be easily grafted on the glucidic moiety of the proteins and allow the production of recombinant enzymes with a homogenous glycosylation for a best lysosomal targeting via the mannose-6-phosphate receptor. Up to date, the company obtained extended results on Pompe disease but they are looking for partners to apply their technology for the improvement of other therapeutic enzymes. The company is looking for partners willing to apply to the 2nd EJP RD Joint Transnational Call for Rare Diseases Research Project (JTC 2020) wich focuses on Pre-clinical Research to Develop Effective Therapies for Rare Diseases.
Different approaches like gene therapy and enzyme therapies can be complementary for better treatment. Partners developing gene therapies (or chaperone therapies) could associate enzyme therapy developed by the company.
Official deadline for the call : 2020/02/18 Deadline for the EoI : 2020/02/11 Anticipated duration of the project : 3 years