Advanced Science: Insilico Medicine utilized AI technology for novel target discovery and drug repurposing to treat endometriosis

Insilico Medicine (“Insilico”), a clinical stage generative artificial intelligence (AI)-driven biotechnology company has recently announced the breakthrough study achieved in collaboration with a well-known university in Hong Kong. Targeting endometriosis, a condition that affects more than 190 million women worldwide, the researchers used Insilico’s PandaOmics, the artificial intelligence-driven platform, to identify two novel therapeutic targets and a FDA approved drug as a potential repurposed treatment option. The findings were published in December 2024 in Advanced Science（IF=15.1）.

Endometriosis, a common gynecological condition in women, happens when tissue similar to the lining of the uterus grows outside the uterus, often leading to dysmenorrhea, infertility, and chronic pain, which significantly impacts patients' quality of life. Currently, there is no cure for endometriosis since the cause is unknown. Painkillers and hormone therapies are conventional treatment options offering symptomatic relief, while severe cases may need surgery to remove endometriotic lesions. Furthermore, studies have shown that endometriosis is a systemic disease that may increase the risk of developing other chronic conditions, such as adenomyosis and ovarian cancer.

In the study, the research team utilizes PandaOmics the AI-driven biological engine to identify novel targets and potential non-hormonal drug repurposing strategies for endometriosis treatment. PandaOmics, a generative biology platform self-developed by Insilico Medicine, is based on over 20 biological models and proprietary databases. The platform not only supports professional target screening and indication exploration but also evaluates and ranks candidate targets based on novelty, credibility, commercial viability, durability, safety, and other key filters.

The research team used PandaOmics the AI platform to perform a meta-analysis of an endometriosis-associated dataset, searching for new therapeutic targets. They revealed for the first time that guanylate-binding protein 2 (GBP2), a protein that regulates immune and inflammatory processes, and hematopoietic cell kinase (HCK), which is involved in cell proliferation and survival signaling, are therapeutic protein targets in endometriosis. In the target validation studies, the results showed reducing the levels of GBP2 and HCK in mouse models of endometriosis led to smaller and lighter endometriotic lesions, less cell growth and more cell death. Notably, these targets also show upregulation in human endometriosis tissue samples, highlighting their relevance to the disease.

Moreover, the study identified integrin beta 2 (ITGB2) as a potential target for endometriosis, which is also the target of Lifitegrast, an FDA-approved small molecule integrin antagonist currently used in dry eye treatment. Results showed the drug effectively suppressed lesion growth when administered in an endometriosis mouse model, indicating that it could be a viable treatment option for endometriosis in clinical settings in future. Notably, the use of AI for target discovery helps shorten the time it takes to develop novel drugs. 

Professor Ronald Wang Chi-chiu, corresponding author of the paper, said, “Identifying GBP2 and HCK as novel therapeutic targets helps us understand the immune factors involved in endometriosis, paving the way for more targeted and effective treatment strategies. These proteins were found to be more active in human samples of endometriosis, highlighting their relevance to the disease mechanism. Our experiment in endometriosis mouse models showed targeting these proteins could help reduce the growth and development of endometriotic lesions.” 

Frank Pun, PhD, Head of the Hong Kong R&D Center of Insilico Medicine co-authored the paper, he said, “The research further highlights the revolutionary impact of artificial intelligence in the acceleration of target discovery and drug repurposing. AI has simplified target identification and improved our capacity to analyze biomedical data, offering a more rapid and cost-efficient approach compared to conventional methods. The study showcases AI's potential to enhance our disease understanding and validates its efficacy in identifying therapeutic targets and repurposing drug candidates.”

 

References

[1] Utilizing AI for the Identification and Validation of Novel Therapeutic Targets and Repurposed Drugs for Endometriosis
https://doi.org/10.1002/advs.202406565

[2] CUHK finds dry eye medicine as repurposed drug for endometriosis | The Standard
https://www.thestandard.com.hk/breaking-news/section/4/224991/CUHK-finds-dry-eye-medicine-as-repurposed-drug-for-endometriosis

[3] CUHK unveils novel therapeutic targets and drug repurposing opportunities for endometriosis https://med.cuhk.edu.hk/press-releases/cuhk-unveils-novel-therapeutic-targets-and-drug-repurposing-opportunities-for-endometriosis

 

About Insilico Medicine

Insilico Medicine, a global clinical stage biotechnology company powered by generative AI, 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. 

www.insilico.com

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