In the intricate battle against diseases, finding effective solutions has always been a complex endeavor. The key lies in identifying the disrupted biological processes, locating the precise targets, and introducing molecules that can rectify the issue without causing harm.
Today, scientists have a game-changing tool at their disposal: artificial intelligence (AI). With its ability to process and analyze vast amounts of data, AI empowers researchers not only to comprehend the intricate connections between biological processes, aging, and diseases but also to design molecules with the essential qualities for successful clinical trials and patient treatments. This revolutionary approach is transforming the field of drug discovery, outpacing traditional methods in terms of speed and efficacy.
A New Era in Biotech
For biotech companies that spearheaded early research in generative biology and chemistry, the advent of AI-designed drugs marks a pivotal moment.
Among these companies is Insilico Medicine, whose scientists were among the pioneers in publishing a groundbreaking method using deep adversarial autoencoders to develop new molecules with desired properties, which can be used in cancer treatment. They now have several cancer-fighting assets in their pipeline, which were designed using their proprietary AI platform Pharma.AI. The platform is able to process massive quantities of biological, chemical, and published data to identify promising targets and design entirely novel molecules.
Insilico Medicine recently made a significant announcement, as their AI-designed oncology drug targeting USP1, a protein-coding gene involved in DNA damage response and repair, received FDA (the U.S. Food and Drug Administration) approval as an Investigational New Drug (IND). This groundbreaking compound will enter clinical trials in the United States in July, followed by trials in China. It is one of many cancer and immuno-oncology drugs in the company’s pipeline, and one of four AI-designed molecules presented at the recent American Association for Cancer Research (AACR) annual meeting.
Unleashing the Potential: AI-Designed Molecules
“We are developing and evaluating ISM3091, a potent and highly selective small molecule inhibitor of USP1, as a target anticancer agent,” said Sujata Rao, MD, SVP, Head of Global Clinical Development at Insilico Medicine. “PARP inhibitors (PARPi) are the first clinically approved drugs designed to exploit synthetic lethality, which have demonstrated positive clinical treatment effects. However, not all patients respond to PARPi, and those who do benefit often develop resistance. ISM3091 promises to be a next-generation synthetic lethal therapy with the potential to bring innovative solutions to patients with solid tumors.”
The USP1 inhibitor is one of a number of small molecules specially designed with novel structures using Insilico’s generative AI platform in order to capitalize on new ways to fight cancer.
AI’s Ongoing Evolution and Insilico’s Vision
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Insilico Medicine’s remarkable progress extends beyond cancer research. Their AI-powered platform has enabled the design of 13th PCC (Preclinical Candidate Compounds) since January 2021, targeting various diseases and disorders. These encompass programs focused on fibrosis, central nervous system disorders, inflammatory bowel disease, chronic kidney disease, and breast cancer.
Notably, Insilico’s lead program, which targets idiopathic pulmonary fibrosis, has received Orphan Drug Designation from the FDA and is rapidly approaching Phase 2 studies, bringing hope to patients suffering from this debilitating condition.
As AI technology continues to evolve, Insilico Medicine remains at the forefront of innovation. The race to help patients with AI-designed drugs is accelerating, and Insilico’s robust pipeline is a testament to just how the company continually adapts to advancements.