One million dollars (US$) are committed to fund the best research proposal/s which support enhancing gene therapy delivery for the treatment of Duchenne Muscular Dystrophy
Duchenne UK (DUK), a UK-based patient organisation, and Parent Project Muscular Dystrophy (PPMD), a USA-based patient organisation, are pleased to announce the launch of their joint 2020 Research Grant Call and wish to invite research proposals for submission and review.
Specifically, and solely, we are interested in receiving proposals relating to:
Minimising the immunological issues of gene therapy in the treatment of Duchenne Muscular Dystrophy
It is likely (but not certain) that funding will be awarded to a single project.
Over the last few years we have seen huge progress in the treatment of the root cause of Duchenne muscular dystrophy through the delivery of replacement (micro) dystrophin using gene therapy. The delivery to muscle of the genetic material for these microdystrophins has been achieved through the use of viral capsids, predominantly AAV-derived. Although we are still in the early stages of this research, we do have evidence that we can detect the presence of microdystrophin protein in treated boys. However, there are some major drawbacks relating to the immune responses to such delivery vehicles. A significant number of boys have a pre-existing resistance to the capsids (and so are ineligible for treatment), but even those that have no such innate resistance will develop it after the first administration, making second dosing (if required) very difficult.
More is needed to advance our understanding of the best ways we can safely, effectively and repeatedly deliver gene therapies to the muscles where they are needed.
Deadline: July 21, 2020