2020 Joint Research Grant Call
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, they 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
Why have we chosen to focus on this area?
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.
What sort of projects are we looking to fund?
Primarily we are inviting proposals which are investigating ways to mitigate the immune response both in terms of pre-existing resistance and resistance induced by receiving a gene therapy dose. These may focus on the ‘damping down’ or ‘circumventing’ the immune system itself or may be investigating alternative delivery methods that promote a minimal immune response. It is important to note that if the latter is the focus of a proposal, we will only consider methods that can deliver full-length dystrophin constructs or enable robust transfection of satellite cells.
In addition, this call has a relatively narrow remit in terms of the development stage. Submissions ready for, or in, early clinical trials will be favoured although we will consider proposals that are in late preclinical stage which show great promise.
Who can apply?
Proposals may be submitted from academic groups or from small commercial concerns. In either case, contracting discussions will include shared ROI.