Development Of AD214 A Novel Anti-fibrotic Treatment For Advanced Age Related Macular Degeneration
Funder
National Health and Medical Research Council
Funding Amount
$687,994.00
Summary
Age related macular degeneration remains the leading cause of blindness in this and other industrialised countries. Although treatment is available for an advanced form of the disease, many do not respond, or lose significant amounts of vision with long term treatment. This proposal will provide critical data for commericalizing a novel therapy called an i-body (AD214), that reduces vision loss in this disease by blocking the formation of scarring with reduced need for injections.
Huntington’s disease is a devastating neurological disorder, with no drugs currently available to cure or treat the underlying cause. Our recent laboratory work on a drug called PBT2 was the foundation of a encouraging clinical trail for this disease. Here, we propose to investigate a drug called Zn(DTSM) for this disease, which has similar properties to PBT2, but we expect will have a greater effect.
Development Of Novel Gene Therapy Vectors For Thalassaemia
Funder
National Health and Medical Research Council
Funding Amount
$287,307.00
Summary
Thalassaemia, is a common inherited disorder affecting haemoglobin synthesis. Synthesis of ?/?-globin chain is balanced during normal red blood cell production. Any disruption in the ratio of ?/?-globin chain results in anaemia. In this study, we will explore gene therapy strategies to restore balanced ?:? globin expression and ultimately improve the severely anaemic phenotype in ?-thalassaemia patients.
Development Of Therapeutic Copper Delivery Agents For Menkes Disease
Funder
National Health and Medical Research Council
Funding Amount
$651,467.00
Summary
Menkes disease does not currently have an effective treatment. The disease is caused by genetic defects that reduce copper transport into the brain and cause mental retardation and death. We have developed drugs that deliver copper into the brain and should cure Menkes disease. We aim to demonstrate that our drugs are effective in mice that have the same genetic defect as patients. Successful results will allow us to begin treating Menkes disease patients to determine if we can cure the disease.