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First AI-Designed Drug Candidate To Reach Human Trials

The AI-designed drug will be used to treat chronic lung disease idiopathic pulmonary fibrosis, or IPF.
Insilico Medicine

Artificial intelligence (AI) backed drug discovery company Insilico Medicine announced last week that it was dosing the first healthy volunteer in a microdose trial of ISM 001-005. Designed with the help of AI, the drug is a small-molecule inhibitor of a biological target that was discovered by Pharma.AI. The trial is being conducted in Australia. 

The AI-designed drug will be used to treat chronic lung disease idiopathic pulmonary fibrosis, or IPF. IPF usually leads to progressive and irreversible lung-function decline and affects 20 people out of over 100,000 globally. 

Chief Scientific Officer of Insilico, Freng Ren, said in a press release that this drug discovery and trial marks a significant milestone in the AI-drug discovery space. This is because the said candidate is the first-ever AI-discovered novel molecule based on an AI-discovered target. 

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AI-driven pharma tech company Insilico Medicine was founded in 2014 and is headquartered in Hong Kong. In June this year, the company raised $255 million in order to improve its clinical trials and launch newer research programmes. 

According to Ren, the team has leveraged an end-to-end AI-powered drug discovery platform with the use of biology and generative chemistry in order to discover the biological targets and generate novel molecules with drug-like properties. The team has developed the drug using machine learning model Insilico’s Generative Tensorial Reinforcement Learning, or GENTRL. The process cost $2.6 million and took less than 18-months to reach its preclinical stage. 


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This drug is the first of its kind ever to enter the clinic, and Ren is hopeful that it will be the first of many. 

According to the company, the ISM 001-005 drug has shown promising results in several preclinical studies. This includes vitro biological, pharmacokinetic and safety studies. In fact, Insilico also stated that the compound has shown significant improvement during myofibroblast activation that contributed to the development of fibrosis. The drug is potentially relevant for a large range of fibrotic indications. 

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Debolina Biswas
After diving deep into the Indian startup ecosystem, Debolina is now a Technology Journalist. When not writing, she is found reading or playing with paint brushes and palette knives. She can be reached at debolina.biswas@analyticsindiamag.com

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