ZF Partnership
The chemical genomics technology company Summit plc and the non-profit Action Duchenne a Duchenne Muscular Dystrophy (DMD) Charity have joined forces to pursue a project involving the development of new medicines to treat the severe muscle wasting disease DMD. The project is called the ZF Partnership Project.
DMD is a genetic condition that is x linked and so predominantly affects boys. Over 40,000 boys in the world are affected by a disease that will leave them in wheelchairs by 10years and without treatment they will die in late teens. There is no cure for DMD and the current gold standard treatment relies on the use of corticosteroids.
The Project will utilise the unique dystrophin deficient zebrafish mutant on a drug screening platform developed by Summit plc in Oxford, England to identify new compounds that might become new drugs in the battle to treat DMD. This Project will build upon the experience of drug development and clinical trial infrastructure already in place at Summit plc. The Charity will bring an invaluable set of skills together with its DMD Registry, a patient database that could assist in the process of fast tracking any drug candidates to human clinical trial. This Project will be a new fundraising focus for the Charity.
ZF Partnership – Medicines for Duchenne Muscular Dystrophy
Vision
Summit plc is a chemical genomics technology company that discovers and develops proprietary and novel drugs and provides services to the pharmaceutical industry. The company's most advanced drug development programme is focused on developing a new treatment for DMD based on the up-regulation of utrophin.
ZF stands for zebrafish. Summit plc has developed a unique infrastructure to screen thousands of drugs on dystrophin deficient zebrafish embryos. This has the advantage of screening in vivo (i.e. live animals) for drug like molecules which reduce the muscular dystrophy, and hence potentially saves time and increases the chances of success of candidate drugs selected for DMD clinical trials.
Currently Summit plc is building a research and discovery infrastructure to seamlessly take drug like compounds from primary screens through to DMD patients at a cost of several million pounds. Summit plc wants to augment its current utrophin upregulation programme with a new screen in zebrafish embryos which would leverage this infrastructure (i.e. medicinal chemistry expertise, drug development capability, clinician networks etc). Compounds identified from these additional screens would be complementary to any output from the current screen.
Mission
Summit plc wants to be able to develop three lines of new drugs with new funding from the Charity:
1. The first line of new drugs will be derived from a screen to up-regulate Utrophin by increasing RNA levels - one fisherman!! This is the current screen running within Summit plc.
2.The second will be new utrophin upregulators from the Zebrafish screen.
3. The most novel screen will be to target drugs that modulate any mechanism that regulates muscle growth or muscle development which will result in a reduction of skeletal MD - the trawler!!
ZF Partnership
Action Duchenne and Summit leading the field of Duchenne Drug discovery
Pasrtners include:
Summit Plc
Action Duchenne
Charleys Fund
Gavriel Meir Trust