Polaryx Therapeutics, Inc., a developer of drug candidates for lysosomal storage disorders, has recently gained IND (Investigational New Drug Application) approval from the U.S. FDA. This approval is related to the study of PLX-200 treatment among patients suffering from JNCL or CLN3 (Juvenile Neuronal Ceroid Lipofuscinosis).

Juvenile Neuronal Ceroid Lipofuscinosis is a genetic lysosomal storage disorder that is caused by the mutation of the CLN3 gene. This kind of rare and fatal NCL disease is most prevalent and occurs among 3 out of 100,000 births. Patients with CLN3 suffer from several impairments including blindness, progressive neurological deterioration, seizures, severe motor & cognitive decline, eventually leading to death in the 2^nd decade of life. No drugs are currently available to treat or delay the progress of this disease.

According to Dr. Hahn-Jun Lee, M.Sc., Ph.D., Chief Executive Officer and President of Polaryx Therapeutics, the company is excited at gaining approval from the FDA to carry out the clinical trials of the CLN3 with PLX-200. In addition to the latest FDA approval, it has also recently gained CLN2 IND approval with the PLX-200 drug. Its team of experts has been working continuously to accelerate the preparation of clinical trials and rapidly initiate the clinical studies.

As per the statement made by Alex Yang, J.D., LLM, Chief Executive Officer and President of Mstone Partners in Hong Kong & Polaryx Therapeutics’ Chair of the Board, the recent FDA approvals for CLN3 clinical trials with PLX-200 will enable the company to advance forward in conducting the clinical studies for human efficacy on a wide range of the Batten disease indications. The company is also planning to enter potential partnerships to expand its reach in other areas of lysosomal storage disorder and effectively discover potential treatment of this rare disease and cater to the unmet needs in the healthcare setting.

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