AceLink Therapeutics Receives Orphan Drug Designation for its Novel GCS inhibitor AL01211 for the
Treatment of Fabry Disease

AceLink Therapeutics, Inc. (AceLink), an innovative biopharmaceutical company developing transformative therapies for genetic diseases, today announced it has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for AL01211 as a treatment for Fabry Disease. “Receiving an FDA Orphan Drug Designation further validates AL01211 as a novel, once-daily orally administered GCS inhibitor as a potential treatment to improve the quality of life of patients suffering from Fabry disease,” said Marvin Garovoy, M.D., Chief Medical Officer of AceLink Therapeutics.  “AL01211 was selected based on its unique properties, including high potency and inability to cross the blood-brain barrier, to more effectively treat the peripheral organs affected by Fabry Disease.”  

In an effort to expand future access to AL01211, AceLink Therapeutics plans to conduct Phase II clinical studies involving Fabry disease patients who are either naive to enzyme replacement therapy or are seeking an alternative to enzyme replacement therapy. “Our objective is to rapidly advance AL01211, so we have initiated discussions with potential commercial partners that are equally committed to ensuring that Fabry Disease patients around the world have access to a novel, once-a-day oral therapy with the potential to dramatically improve their quality of life, said Jerry Shen, Ph.D., Chief Executive Officer of AceLink Therapeutics. 

About AL01211 

AL01211 is a proprietary GCS inhibitor with excellent potency (single-digit nanomolar IC50), great selectivity, and other favorable drug properties that support once-daily oral administration.  AL01211 offers a much-needed oral small molecule therapy as an alternative to current therapies for Fabry disease that require frequent intravenous infusions.  Phase II clinical studies of AL01211 in patients with Fabry disease are planned to start in 2023.  

About GCS inhibitor 

GCS (glucosylceramide synthase) catalyzes the first step in the synthesis of glycosphingolipids, a group of bioactive molecules that play important roles in various cellular processes and diseases.  GCS inhibitors reduce the production of glycosphingolipids, thereby exerting beneficial effects to diseases such as Fabry Disease and Gaucher disease. 

About AceLink Therapeutics, Inc. 

Founded in 2018, AceLink Therapeutics is an innovative biopharma startup focusing on developing safe and effective medicines to address genetic diseases with high unmet needs. The company’s initial focus is to develop novel therapeutics for Fabry disease. For more information, please visit

Contact Information: 

Jerry Shen, Ph.D. 

CEO and President 

Phone: (925) 262-3145 

Email: [email protected] 

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