AceLink Therapeutics, Inc., a pioneering biopharmaceutical company dedicated to creating life-changing treatments for genetic diseases, has unveiled the promising early results of AL00804, an oral brain-penetrant glucosylceramide synthase inhibitor. AL00804 has the potential to treat Gaucher disease and other neuronopathic glycosphingolipid (GSL) storage diseases. This novel and innovative therapy could bring hope to countless people suffering from these conditions.
We are excited to announce that our collaborative work with Dr. Ying Sun’s team at Cincinnati Children’s Hospital Medical Center will be presented as a poster at the 2023 WORLD Symposium in Orlando, Florida from February 22- 26, 2023. Join us there to learn more about our cutting-edge research and its implications for the future of healthcare.
AceLink is thrilled to announce the success of AL00804 in reducing symptoms of CNS disease in several preclinical models. With its unique properties of high potency and the ability to cross the blood-brain barrier, AL00804 is a promising candidate for the treatment of neuronopathic diseases. Jerry Shen, PhD, Chief Executive Officer and Founder of AceLink, is proud of the team’s IND-enabling work and looks forward to advancing AL00804 into clinical settings.
AL00804 is the newest breakthrough in treating neuronopathic GSL storage disorders, with its superior brain penetration and increased potency compared to Venglustat. Pharmacokinetics studies have confirmed its effectiveness with once daily, oral administration. In preclinical disease models, AL00804 was found to reduce brain GL1 and lyso-GL1 accumulation, and its efficacy was proven with significant delay in motor function decline and extended survival. Even head-to-head, AL00804 came out on top in a neuronopathic model of Gaucher disease, making it a potentially safer and more effective treatment option.
AL00804 is an exciting oral drug, uniquely capable of crossing the blood-brain barrier, that is being developed to treat a variety of neuronopathic glycosphingolipid storage diseases. This includes conditions such as Gaucher disease, GM2 gangliosidosis (Tay-Sach and Sandhoff disease), GM1 gangliosidosis, and even some genetic forms of Parkinson’s disease. This revolutionary treatment has the potential to drastically improve the lives of countless individuals.
About GCS inhibitor
GCS (glucosylceramide synthase) is a key enzyme in the synthesis of glycosphingolipids, bioactive molecules that are essential for various cellular processes and diseases. Inhibiting GCS activity has been effective in treating diseases such as Fabry disease and Gaucher disease, which arise from an accumulation of these lipids. By reducing their production, these GCS inhibitors can bring relief to those suffering from these conditions.