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HitGen and the PACS1 Syndrome Research Foundation announce an agreement to discover ligands that address issues related to the mutated PACS1 gene product Release Time:2022-04-19


CHENGDU, China, April 19, 2022 – HitGen Pharmaceuticals Inc. and the PACS1 Syndrome Research Foundation announce an agreement focused on the application of HitGen’s DNA-encoded library (DEL) technology to identify ligands that could selectively bind to the mutated PACS1 Protein and therefore serve as a treatment or a cure for patients with PACS1 Syndrome.


HitGen’s technology platform, centered around the design, synthesis, and interrogation of over one trillion component libraries of DNA encoded small molecules, will be deployed to discover novel chemical leads for therapeutic targets identified by the PACS1 Syndrome Research Foundation.


“While over half of FDA’s 2021 novel drug approvals were to treat rare or “orphan” diseases, PACS1 syndrome with currently less than 200 cases known world-wide has not been addressed by the biotechnology sector. Consequently, we are privileged to collaborate with the PACS1 Syndrome Research Foundation and their academic colleagues in these discovery efforts and we acknowledge and applaud their vision to identify potential cures for this challenging disease of children.” said Dr. Jin Li, Chairman of the Board and Chief Executive Officer of HitGen. He added that “HitGen continues to grow as a world-class drug discovery organization. In addition to our collaborations with industry partners, we are committed to engaging with philanthropic foundations and academic institutions to provide solutions for patients with unmet medical needs. Together we advance the notion that the unprecedented scope of DNA-encoded chemistry to access chemical space and identify liganding crannies in mutated gene products is a viable approach to address toxic outcomes related to the mutation.” 


“The prospect of identifying small molecules that bind selectively to the mutant PACS1 protein and might prevent its toxicity is extremely exciting. The extraordinary rich libraries of encoded small molecules that have been assembled by HitGen are a highly promising source of potential drugs that could help patients overcome the deleterious effects of the mutation. We are privileged to benefit from HitGen’s expertise and involvement,” said Taruna Reddy, President of the PACS1 Syndrome research foundation.




About the PACS1 Syndrome Research Foundation

The PACS1 Syndrome Research Foundation is focused on funding cutting-edge scientific research towards finding a cure for PACS1 Syndrome. PACS1 syndrome is caused by a single-point mutation in the PACS1 gene. Patients with PACS1 Syndrome have severe neurological abnormalities, and will be dependent life-long on caregivers. They have intellectual disabilities, global developmental delays, epilepsy, autism, and significant language impairments. 

For Further information please contact:

Taruna Reddy, President and Founder, taruna@pacs1foundation.org


About HitGen Inc.

HitGen Inc. is a biotech company headquartered in Chengdu, China, with subsidiaries in Cambridge, UK and Houston, USA. It became a publicly listed company in Shanghai Stock Exchange in April 2020 (ticker code 688222.SH). HitGen has established a drug discovery research platform for small molecules and nucleic acid drug centered on the design, synthesis and screening of DNA encoded chemical libraries (DELs), fragment-based drug discovery (FBDD) and structure-based drug design (SBDD) technologies. HitGen's DELs currently contains more than 1 trillion novel, diverse, drug-like small molecules and macrocyclic compounds. These compounds are members of DELs synthesized from many thousands of distinct chemical scaffolds, designed with tractable chemistry, and have yielded proven results for the discovery of small molecule leads against precedented and unprecedented classes of biological targets, in collaborations with industry and academia in the Americas, Asia, Europe, Africa, and Australia.

For further information, please contact

Dr. Jin Li, Chairman & CEO, HitGen Inc. Tel: +86 028 85197385

Dr. Barry Morgan, Chief Scientific Officer, HitGen Pharmaceuticals Inc. Tel: +1 508 840 9646


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