9 May 2023 - - US-based biopharmaceutical company AceLink Therapeutics, Inc has appointed Pedro Huertas, M.D., Ph.D., as Chief Medical Officer, the company said.

Dr. Huertas is a distinguished expert in rare diseases and will serve as an important resource for AceLink as the company advances its development pipeline.

Dr. Pedro Huertas has 30 years of pharmaceutical industry experience in research and development, medical affairs, and regulatory affairs in therapeutics for rare genetic diseases.

Prior to joining AceLink, Dr. Huertas was Chief Medical officer for Eloxx Pharmaceuticals, Sentien Biotechnologies, Inozyme Pharma, Novazyme Pharmaceuticals, and Kriya Therapeutics.

His prior experience includes positions at Pfizer, Shire, Genzyme, Amicus Therapeutics, Novazyme, Advanced Cell Technology and StemCells, Inc.

Dr. Huertas is a graduate of Stanford University (M.S., Biochemistry), Harvard University (Ph.D., Cell and Developmental Biology), Harvard Medical School, and MIT Sloan School of Management (M.S., Management).

He also trained in Internal Medicine, Rheumatology and Palliative Care at Massachusetts General Hospital.

AL01211 is a non-brain penetrating oral inhibitor of GCS, or glucosylceramide synthase, which is an enzyme that facilitates the first step in the production of glycosphingolipids, a diverse group of biologically active fatty molecules.

Fabry disease is caused by genetic mutations that result in an abnormal buildup of certain glycosphingolipids, particularly globotriaosylceramide, inside cells.

By blocking the activity of GCS, AL01211 is designed to reduce this toxic buildup, decrease inflammation and improve organ function, ultimately slowing disease progression.

According to AceLink, the investigational therapy has high potency against GCS and other pharmacological properties which will allow once-daily oral dosing.

Of note, the therapy is unable to cross the blood-brain barrier, meaning it will not get into the brain or spinal cord if administered systemically.

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.

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.

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.