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Duchenne Muscular Dystrophy (DMD) is a rare muscle disorder affecting children that results in loss of the ability to walk. It’s rapidly progressive — average life expectancy is about 20 years — and caused by genetic mutations on the X chromosome that regulates the production of dystrophin, a protein thought to play a role in maintaining muscle cell membranes.

For DMD sufferers and other patients with rare degenerative diseases, there’s now hope on the horizon. Insilico Medicine and A2A Pharmaceuticals today launched Consortium.AI, a new venture founded with the goal of applying advances in artificial intelligence (AI) to cutting-edge drug discovery.

Through Consortium.AI, the two companies will collaboratively develop therapeutic treatments for DMD and other severe genetic disorders and use machine learning to validate the most promising candidates.

Baltimore-based Insilico Medicine pioneered the use of generative adversarial networks (GANs) — a class of algorithms well-suited to unsupervised learning — in molecular structure creation for diseases with a known ligand (a complex biomolecule) but no target (a protein associated with a disease process). Its team of more than 50 researchers is actively working on drug discovery programs in cancer, dermatological diseases, fibrosis, Parkinson’s, Alzheimer’s, ALS, diabetes, sarcopenia, and aging.


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“We are pleased to partner with Insilico Medicine, combining our strengths and complementary technologies to accelerate the advancement of better therapeutics into the clinic for the patients that need them,” said Dr. Elena Diez Cecilia, head of business product development at A2A Pharmaceuticals. “Muscular Dystrophy is a debilitating and terminal degenerative condition that causes muscle inflammation and wasting, and there is a huge need for more effective therapies.”

Much in the same vein, New York-based A2A Pharmaceuticals uses computational tools to design therapies for difficult-to-treat disease targets like protein-protein interactions.

“A2A Pharmaceuticals has a team of highly talented drug hunters with a proven track record in discovery, development, and licensing of the drug candidates,” Insilico Medicine founder and CEO Alex Zhavoronkov said in a statement. “We are very happy to partner with their team to address the unmet medical needs of patients who are desperately waiting for solutions. This is a fantastic application for AI.”

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