(BUSINESS WIRE)–AceLink Therapeutics, Inc. (AceLink), an innovative biopharmaceutical company developing transformative therapies for genetic diseases, today announced that the company will present two abstracts featuring the development of AL01211 to treat Fabry disease and AL00804 to treat Gaucher disease and other neuronopathic glycosphingolipid storage diseases at the 19th Annual World Symposium Conference, being held in Orlando, Florida, from February 22-26, 2023.
Details of the abstracts are as follows:
Abstract Title: Development of AL00804, a novel brain penetrant glucosylceramide synthase inhibitor, to treat Gaucher disease and other neuronopathic glycosphingolipid storage diseases
Authors: Michael Babcock, Ying Feng, Benjamin Liou, Venette Fannin, Yi Lin, Jerry Shen, Ying Sun
Session Title: Basic Science – Poster Session III
Session Date & Time: Thursday, February 23, 3:00 PM – 4:00 PM ET
Abstract Number: LB-07
Location: Hilton Orlando, Kiosk 26-B
Abstract Title: Development of AL01211, an oral, non-brain penetrant glucosylceramide synthase inhibitor (GCSi), to treat Fabry disease
Authors: Michael Babcock, Jianhong Zheng, Li Li, Jessica Gail Shurr, Marvin Garovoy, Jerry Shen
Session Title: Translational Research – Poster Session IV
Session Date & Time: Thursday, February 23, 4:00 PM – 5:00 PM ET
Abstract Number: LB-08
Location: Hilton Orlando, Kiosk 8-A
AL01211 is a proprietary, non-brain penetrant 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.
AL00804 is a highly brain penetrant GCS inhibitor being developed for the treatment of neuropathic glycolipid storage diseases including Gaucher disease, GM2 gangliosidosis (Tay-Sach and Sandhoff disease), GM1 gangliosidosis, and possibly certain genetic forms of Parkinson’s disease.
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, which are caused by the accumulation of these lipids.
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 www.acelinktherapeutics.com.