Screening for novel drugs is one of the costliest steps of drug development. We use a cost-effective Drosophila model for screening small molecules, mRNA therapy and anti-sense oligos (ASOs) against less explored diseases and Rare Genetic Disorders. Drosophila shows more than 80% conservation with human genes and pathways. Over last hundred years, Drosophila researchers have developed excellent genetic tools and behavioural assays which will help us test the effect of drugs tested and evaluate the impact of the drugs on key pathways, behaviours and lifespan. After the primary screen in flies, the positive candidates will be tested in cell cultures and animal models. The use of flies will substantially reduce the cost of screening for novel drugs. This will make it possible to develop low cost and effective treatments for rare genetic disorders.
Vertical Lead: Vasanth Thamodaran
Spinal Muscular Atrophy (SMA) is a recessive genetic disorder affecting 1 in 7000 live births. SMA also causes significant fatality in children below 2 years. This neurodegenerative disorder is caused due to the lack of a functional protein called Survival Motor Neuron (SMN). There is progressive loss of motor neurons and muscles leading to altered mobility, eating and breathing problems, scoliosis and death. The current therapeutic treatments available include a single drug, protein or gene replacement which fall under the most expensive treatments in biomedicine. One of the main cost factors in drug development is the screening of novel effective drugs. We are developing a Drosophila model that mimics the SMA disorder as seen in humans. This model is used to test reversal of a few or many of the disorder symptoms using different therapeutic interventions like small molecules, anti-sense oligos, mRNA replacement.