Date: 2016-02-02
Type of information: R&D agreement
Compound: small molecule therapies
Company: Anagenesis Biotechnologies (France) Ksilink (Germany)
Therapeutic area: Neuromuscular diseases - Regenerative medicine
Type agreement: R&D
Action mechanism:
Disease: Duchenne muscular dystrophy
Details: * On February 2nd , 2016, Anagenesis Biotechnologies and Ksilink announced the signature of a collaborative research agreement. Each partner will apply its expertise to the discovery and development of small molecule therapies for Duchenne muscular dystrophy thanks to unique stem-cell derived disease models. Anagenesis Biotechnologies develops a proprietary technology that allows for the efficient, reproducible and chemically defined differentiation of pluripotent cells into mature skeletal muscle. The company has shown that differentiated embryonic stem (ES) cells derived from mdx mice exhibit a striking branched phenotype resembling that described in Duchenne muscular dystrophy patients, thus providing an attractive model to study the origin of the pathological defects associated with the disease. This work therefore opens the possibility to develop in vitro models to study the pathology of muscular dystrophies as well as high throughput assays based on myogenic cells for drug screening. Ksilink is a public private partnership that spans an integrated therapy development process from “bed to the bench, and back to the bed”. Having access to leading clinical and life science expertise in France and Germany, Ksilink sources the most effective strategies towards patient needs. It uses cutting edge technologies to de-risk the science underlying the development path, specializing on the use of patient induced pluripotent stem cells (iPSC)-based disease models. Ksilink also functions as a program-specific investment fund enabling partners to carry innovation to the patient. With this collaborative research agreement, both partners will take advantage of Anagenesis Biotechnologies’ proprietary skeletal muscle system and of Ksilink’s expertise and equipment to identify the phenotypic differences between Duchenne muscular dystrophy and wild type (WT) cells differentiated in vitro, and screen for compounds capable of “rescuing” the Duchenne muscular dystrophy cells back to a WT phenotype. Once identified, the hits will go through selection and optimization stages, with the aim of initiating clinical trials within 5 years with the compound showing most promise as a drug candidate against Duchenne muscular dystrophy.
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