Epilepsy is one of the most common chronic neurological disorders; it affects 1% of the world’s population, yet about 1 in 3 patients fail to achieve seizure control with current drugs. We will improve the properties of small molecules (drugs) that specifically target the GTPase activity of the enzyme dynamin, to reduce seizure effect in the brain by a novel mechanism. We will optimize and pre-clinically test these future chemical entities as potential anti-epileptic drugs.
We aim to develop a new class of cholesterol-lowering drugs by blocking the interaction between a protein in the blood called PCSK9 and its receptor, which is implicated in cholesterol absorption. We will do this by designing small stable peptides (mini proteins) that mimic part of the receptor and have the potential to interfere with the normal PCSK9 binding process. These drugs should be less expensive and potentially less immunogenic than competing therapies based on antibodies.
Further Development Of The Clinical Potential Of H2 Relaxin
Funder
National Health and Medical Research Council
Funding Amount
$651,768.00
Summary
The hormone relaxin mediates cardiovascular and kidney changes during pregnancy. These important functions have led to its current use in clinical trials for the treatment of acute heart failure, a condition affecting millions of patients worldwide. However, there is an urgent need for a longer lasting form of relaxin for prolonged treatment of patients. Our studies will focus on understanding the blood breakdown of the peptide to lead to the design of longer lasting relaxin analogues.
Peptide Conjugates Of Splice-correcting Oligonucleotides For Enhanced In Vitro And In Vivo Delivery For Neuromuscular Disease Therapy.
Funder
National Health and Medical Research Council
Funding Amount
$332,347.00
Summary
Currently, there is no known cure for certain neuromuscular genetic disorders. However, recently identified synthetic DNA-type biomolecules have shown promising results in reversing such diseases in mice. These biomolecules cannot easily enter the cells in high enough quantity to elicit their beneficial effects. Therefore, this project will aim at identifying novel vecotrs that, when coupled to these biomolecules, are capable of delivering them into specific cell types as well as into the brain.
Development Of Next Generation Drugs For Chronic Myeloid Leukaemia
Funder
National Health and Medical Research Council
Funding Amount
$632,726.00
Summary
Chronic myeloid leukaemia (CML) is one of the four most common types of leukaemia. With current therapies, 15–20% of patients newly diagnosed for CML will die in the next five years, and it is therefore vitally important to discover new treatments. The aim of this project is to develop a new generation of drugs to treat CML based on new approaches (i.e., different type of molecules and different binding site) that can combat the resistance acquired to the current treatments.