AceLink Presents Preclinical Data on AL00804 at WORLD Symposium

– AL00804, a highly brain penetrant GCS inhibitor, efficiently reduced GL1 accumulation in the brain of preclinical models –

– In a head-to-head comparison in a neuronopathic model for Gaucher disease, AL00804 exhibited higher potency and greater brain penetration compared to other GCS inhibitors currently in development –

– AL00804 is IND-ready and aims to be the best-in-class treatment of neuronopathic Gaucher disease, GM2 and GM1 gangliosidosis –

(BUSINESS WIRE)–AceLink Therapeutics, Inc. (AceLink), an innovative biopharmaceutical company developing transformative therapies for genetic diseases, today presented data on the development of AL00804, a novel brain penetrant glucosylceramide synthase inhibitor, to treat Gaucher disease and other neuronopathic glycosphingolipid (GSL) storage diseases. AL00804 is a novel, oral, highly brain penetrant glucosylceramide synthase inhibitor (GCSi).

The presented work was done in collaboration with Dr. Ying Sun’s team at Cincinnati Children’s Hospital Medical Center and will be a poster presentation during the 2023 WORLD Symposium, being held in Orlando, Florida, from February 22- 26, 2023.

“We are pleased with the efficacy of AL00804 in reducing symptoms of CNS disease in several preclinical models,” said Jerry Shen, PhD, Chief Executive Officer and Founder of AceLink. “AL00804 was selected based on its unique properties, including high potency and ability to cross the blood-brain barrier, to more effectively treat patients with neuronopathic diseases. We have completed IND-enabling work in support of AL00804 and are excited for its further development in clinical settings.”

Data Highlights:

  • Pharmacokinetics studies support once daily, oral administration of AL00804.
  • AL00804 is highly brain penetrant that efficiently reduced brain GL1 and lyso-GL1 accumulation in preclinical disease models.
  • AL00804 reduced CNS disease symptoms, significantly delayed motor function decline and extended survival.
  • When tested head-to-head in a neuronopathic model of Gaucher disease, AL000804 performed better than Venglustat.
  • The increased potency and superior brain penetration compared to Venglustat make AL00804 a potentially safer and more efficacious molecule for treating neuronopathic GSL storage disorders.

Details on the AceLink WORLD Symposium Presentation

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 from 3:00 – 4:00 PM ET
Abstract Number: LB-07
Location: Hilton Orlando, Kiosk 26-B

About AL00804

AL00804 is a proprietary, oral, highly brain penetrant GCS inhibitor being developed for the treatment of neuronopathic glycosphingolipid 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

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