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Discovering New Medicines
for Unmet Clinical Needs
Our Portfolio
Developing Novel Medicines for unmet medical needs
We are developing a diverse pipeline of small molecules with the potential to improve the lives of millions of people around the world.
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Indication | Hit Identification | Lead Optimisation | Preclinical Toxicology | Phase 1 | Phase 2 |
|---|---|---|---|---|---|
Cognition in Schizophrenia | |||||
Anxiety Disorders | |||||
Major Depressive Disorder | |||||
Postpartum Depression | |||||
Schizophrenia | |||||
Chronic Pain | |||||
Krabbe Disease | |||||
Depression | |||||
Motor Neuron Disease | |||||
Undisclosed Lysosomal Target | |||||
Acute Myeloid Leukaemia | |||||
Colon Cancer |
Our Programs
Synaptic Health
Restoring connectivity between brain cells
Neurotransmitters are the body’s chemical messengers. They are molecules used by the nervous system to transmit messages between neurons, or from neurons to muscles and play a crucial role in regulating various psychological functions, including mood, emotion, stress response, and cognition.
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Communication between two neurons happens in the synaptic cleft (the small gap between the synapses of neurons). When an electrical signal reaches the end of a neuron, it triggers the release of neurotransmitter molecules into this synaptic cleft. These neurotransmitters then bind to receptor molecules on the next neuron, which triggers a response. There are many types of neurotransmitters, including dopamine, serotonin, dopamine and gamma-aminobutyric acid (GABA), each playing a crucial role in the body’s function - from triggering muscle contractions, to regulating mood and sleep.
Understanding neurotransmitters and their functions is fundamental to neuroscience and is key to developing treatments for neurological disorders
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Through its understanding of the complex interplay of these neurotransmitters and their receptors, the Medicines Discovery Institute is developing novel treatment for disorders such as cognition impairment in schizophrenia, anxiety disorder, major depressive disorders and post partum depression.
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"Our understanding of the selectivity of GABAA receptor sub-units and its effects on neurological functions provides us with valuable insights into the development of treatments for various neurological disorders"
Dr Oana Popa
Research Fellow, Neuroscience
Lyososomal Storage in Neurodegeneration and Aging
Repairing cell recycling
Lysosomal storage disorders (LSDs) are inherited metabolic disorders that are characterised by an accumulation of substrates and macromolecules in cells due to a functional defect of the lysosomes.
There are approximately 70 different LSDs, including Gaucher disease, Fabry disease and Niemann-Pick disease. Although individually these are rare disorders, collectively they can affect around 1 in 5000 people.
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The pathophysiology of these diseases is complex; they can affect several organ systems and almost all have neurological involvement. Some of the most common LSD symptoms include neurological manifestations, spleen and liver enlargement, and skeletal malformations. Almost all LSDs affect children, and taken together they are the most common cause of neurodegeneration in children.
Current therapies for some LSDs include enzyme replacement therapy, substrate reduction therapy and gene therapy, however the cost of these treatments is high, and their success varies from one individual to another.
There is a pressing need to find better therapies and the Medicines Discovery Institute is developing new treatments to restore defects in lysosomal function
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"Lysosomal research is an emerging area owing to the development of new tools capable of measuring lysosomal function at the acidic pH found within these compartments.
We are particularly interested in applying our research on understanding the events that unfold when these proteins become dysfunctional in lysosomal diseases"
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Dr Emyr Lloyd-Evans
Deputy Director, Biology
Oncology
Making discoveries that save lives
The incidence of cancer in Wales has surged by 16% since 2002, with a notable rise in diagnosed cases, reaching 20,058 in 2019. In response to this escalating challenge, the Medicines Discovery Institute collaborates with Cardiff University Medical School and the University Hospital of Wales under the Wales Cancer Research Centre umbrella. Together, they aim to pioneer novel drug treatments for challenging-to-treat cancers.
"Our commitment to advancing drug discovery is an ongoing efforts to investigate new drug targets. Our collaborations with biomedical researchers across Cardiff University form a key part of the fostering environment conducive to pioneering breakthroughs in cancer treatment"
Dr Darren LeGrand
Senior Research Fellow, Medicinal Chemistry