• HOME
  • NEWS
  • EXPLORE
    • CAREER
      • Companies
      • Jobs
    • EVENTS
    • iGEM
      • News
      • Team
    • PHOTOS
    • VIDEO
    • WIKI
  • BLOG
  • COMMUNITY
    • FACEBOOK
    • INSTAGRAM
    • TWITTER
Wednesday, October 8, 2025
BIOENGINEER.ORG
No Result
View All Result
  • Login
  • HOME
  • NEWS
  • EXPLORE
    • CAREER
      • Companies
      • Jobs
        • Lecturer
        • PhD Studentship
        • Postdoc
        • Research Assistant
    • EVENTS
    • iGEM
      • News
      • Team
    • PHOTOS
    • VIDEO
    • WIKI
  • BLOG
  • COMMUNITY
    • FACEBOOK
    • INSTAGRAM
    • TWITTER
  • HOME
  • NEWS
  • EXPLORE
    • CAREER
      • Companies
      • Jobs
        • Lecturer
        • PhD Studentship
        • Postdoc
        • Research Assistant
    • EVENTS
    • iGEM
      • News
      • Team
    • PHOTOS
    • VIDEO
    • WIKI
  • BLOG
  • COMMUNITY
    • FACEBOOK
    • INSTAGRAM
    • TWITTER
No Result
View All Result
Bioengineer.org
No Result
View All Result
Home NEWS Science News Biology

Insilico Medicine receives IND approval for novel AI-designed USP1 inhibitor for cancer

Bioengineer by Bioengineer
May 25, 2023
in Biology
Reading Time: 3 mins read
0
Novel AI-Designed USP1 Inhibitor Receives IND Approval
Share on FacebookShare on TwitterShare on LinkedinShare on RedditShare on Telegram

Insilico Medicine (“Insilico”), a generative artificial intelligence (AI)-driven clinical stage drug discovery company, today announced that the U.S. Food and Drug Administration (FDA) recently approved the initial investigational new drug (IND) application for ISM3091 for the treatment of patients with solid tumors. This is Insilico’s first oncology program to advance to the clinical validation stage.

Novel AI-Designed USP1 Inhibitor Receives IND Approval

Credit: Insilico Medicine

Insilico Medicine (“Insilico”), a generative artificial intelligence (AI)-driven clinical stage drug discovery company, today announced that the U.S. Food and Drug Administration (FDA) recently approved the initial investigational new drug (IND) application for ISM3091 for the treatment of patients with solid tumors. This is Insilico’s first oncology program to advance to the clinical validation stage.

The open-label, multicenter Phase I clinical trial of ISM3091 will be conducted simultaneously in the U.S. and China to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of ISM3091 in patients with advanced solid tumors, and through dose escalation and dose optimization to identify the recommended dose level for Phase 2 monotherapy. Insilico has filed the IND submission to the U.S. FDA and the NMPA and expects to first initiate the study in the U.S. center in July 2023.

“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.”

ISM3091 is a potentially best-in-class, orally available and highly selective small molecule inhibitor of USP1, which has emerged as a synthetic lethal target for cancer treatment that plays a crucial role in DNA damage response and repair. In preclinical experiments, ISM3091 was found to be potently efficacious against multiple tumor cell lines and in vivo models with BRCAm, as well as in HRR-proficient models. It is also well tolerated in different species with a high margin of safety. Insilico disclosed select data during a poster presentation at the American Association for Cancer Research (AACR) Annual Meeting in April 2023. 

“ISM3091 is Insilico’s third internal program to advance to the clinical stage. We are pleased to see that it has demonstrated promising safety profiles and efficacy features in preclinical,” said Feng Ren, PhD, co-CEO and Chief Scientific Officer of Insilico Medicine. “The published patent of ISM3091 shows that the molecule designed with the support of Chemistry42 is distinctly novel, which once again shows the ability of AI to generate compounds from scratch. We are committed to taking advantage of AI-driven drug discovery to rapidly develop innovative therapeutics for unmet medical needs.”

The novel structure of ISM3091 was designed and optimized using Insilico’s proprietary AI platform, Chemistry42, which is built on years of modeling and pre-training of large biological, chemical, and textual datasets and includes 42 generative engines and more than 500 predictive engines for scoring that allows researchers to generate molecules with desired properties through structure-based drug design (SBDD) and ligand-based drug design (LBDD). Relying on the synergy between its powerful AI platform and strong internal drug discovery expertise, Insilico nominated the preclinical candidate targeting USP1 in Apr 2022, only 9 months after the program was initiated. 

“Since 2021, Insilico has nominated 12 preclinical candidate compounds, including ISM3091, the small molecule USP1 inhibitor, and our first oncology program to reach the clinical stage,” said Alex Zhavoronkov, PhD, Founder and CEO of Insilico Medicine. “We hope to further validate our candidate compound in the multicenter trial, as well as the capabilities of our AI-driven drug discovery platform.”

Powered by Pharma.AI, Insilico’s proprietary drug discovery platform driven by generative AI, the company has developed an innovative portfolio comprising over 30 pipelines, with three leading programs in the clinical stage. Namely, ISM3091, a potential best-in-class USP1 inhibitor granted FDA IND approval; ISM3312, an orally available 3CLpro inhibitor with broad anti-coronavirus activity in Phase I trials; and ISM018_055, the first AI-discovered drug candidate for the treatment of Idiopathic Pulmonary Fibrosis (IPF) already validated in Phase I and granted Orphan Drug Designation (ODD) by the FDA.

 

About Insilico Medicine

Insilico Medicine, a clinical stage end-to-end generative artificial intelligence (AI)-driven drug discovery company, is connecting biology, chemistry, and clinical trials analysis using next-generation AI systems. The company has developed AI platforms that utilize deep generative models, reinforcement learning, transformers, and other modern machine learning techniques for novel target discovery and the generation of novel molecular structures with desired properties. Insilico Medicine is developing breakthrough solutions to discover and develop innovative drugs for cancer, fibrosis, immunity, central nervous system diseases, infectious diseases, autoimmune diseases, and aging-related diseases. 

Website: www.insilico.com 



Share12Tweet8Share2ShareShareShare2

Related Posts

blank

New Insights into Evolution Revealed Through Lizard Genetics

October 8, 2025
blank

Cell-Free DNA Reflects Tumor Transcription Factor Activity

October 8, 2025

New Method to Monitor Wild Reindeer Populations Could Boost Conservation Efforts

October 8, 2025

New Molecular Method Detects Varroa Destructor in Nigeria

October 8, 2025

POPULAR NEWS

  • Sperm MicroRNAs: Crucial Mediators of Paternal Exercise Capacity Transmission

    1053 shares
    Share 421 Tweet 263
  • New Study Reveals the Science Behind Exercise and Weight Loss

    99 shares
    Share 40 Tweet 25
  • New Study Indicates Children’s Risk of Long COVID Could Double Following a Second Infection – The Lancet Infectious Diseases

    95 shares
    Share 38 Tweet 24
  • Ohio State Study Reveals Protein Quality Control Breakdown as Key Factor in Cancer Immunotherapy Failure

    78 shares
    Share 31 Tweet 20

About

We bring you the latest biotechnology news from best research centers and universities around the world. Check our website.

Follow us

Recent News

Analyzing Methadone Levels in Post-Mortem Cases

New Vaccine Demonstrates Potential Against Typhoid and Invasive Salmonella in Initial Human Trial

Innovative 3D Printing Technique ‘Grows’ Ultra-Strong Materials

Subscribe to Blog via Email

Success! An email was just sent to confirm your subscription. Please find the email now and click 'Confirm' to start subscribing.

Join 63 other subscribers
  • Contact Us

Bioengineer.org © Copyright 2023 All Rights Reserved.

Welcome Back!

Login to your account below

Forgotten Password?

Retrieve your password

Please enter your username or email address to reset your password.

Log In
No Result
View All Result
  • Homepages
    • Home Page 1
    • Home Page 2
  • News
  • National
  • Business
  • Health
  • Lifestyle
  • Science

Bioengineer.org © Copyright 2023 All Rights Reserved.