Date: 2016-07-07
Type of information: Collaboration agreement
Compound: MeCP2 protein
Company: Armagen (USA - CA) Rett Syndrome Research Trust (USA - CT)
Therapeutic area: Rare diseases - Genetic diseases - Neurological diseases
Type agreement: collaboration
Action mechanism: protein. Rett Syndrome is caused by mutations in MeCP2 (methyl CpG binding protein 2) that is critical for maintaining the health of brain cells. ArmaGen will develop a form of the MeCP2 protein that can cross the blood-brain barrier (BBB) to compensate for the mutated protein. The ArmaGen technology of delivering proteins into the brain takes advantage of naturally occurring receptors on the surface of the BBB that shuttle complex biologics into the brain. By fusing the MeCP2 protein together with an antibody to the receptor, the “Rett fusion protein” essentially piggybacks its way into the brain.
Disease: Rett syndrome
Details: * On July 7, 2016, ArmaGen announced a collaboration with the Rett Syndrome Research Trust (RSRT) to develop a novel therapeutic for the treatment of Rett Syndrome, a rare genetic postnatal neurological disorder that affects girls almost exclusively. The disease is caused by mutations in MeCP2 (methyl CpG binding protein 2) that is critical for maintaining the health of brain cells. Under the terms of the research collaboration, ArmaGen will utilize its expertise and technology to develop a form of the MeCP2 protein that can cross the blood-brain barrier (BBB) in an effort to compensate for the mutated protein. RSRT, a non-profit organization dedicated to advancing a cure for Rett Syndrome, will fund the development of this novel fusion construct as well as lend technical support to the experiments. ArmaGen will be responsible for any future development activities.
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