BioArctic develops new treatment for Gaucher's disease

STOCKHOLM, Nov. 8, 2022 /PRNewswire/ -- BioArctic AB (publ) (NASDAQ Stockholm: BIOA B) today announced that the company has initiated a new project focused on enzyme replacement therapy for Gaucher's disease in combination with the company's Brain Transporter technology. Today there is no treatment available for neuropathic Gaucher's disease and BioArctic's aim is to develop a therapy which also affects the neuropathic manifestation of the disease.

Gaucher's disease is a rare genetic disorder in which a reduced function of the enzyme glucocerebrosidase leads to accumulation of glycolipids in certain organs. Symptoms range from mild to severe and can appear at any time, from infancy to old age, and include anemia, fatigue, easy bruising, and a tendency to bleed as well as an enlarged spleen and liver. In rare neuropathic Gaucher disease, neurological symptoms also occur which include difficulties controlling eye movements, unsteadiness, seizures and cognitive impairment.^[1] These patients usually experience the first symptoms in childhood and rarely survive 40 years of age due to the effects of the disease on the brain.

The standard of care today includes enzyme replacement therapy. These replacement enzymes do not however, treat the neuropathic disease since the enzymes do not cross the blood brain barrier and therefore do not reach the brain. By linking an enzyme replacement therapy to BioArctic's Brain Transporter platform, the company hopes to develop a treatment which address the neuropathic as well as the peripheral manifestations of the disease.

"People with neuropathic Gaucher's disease today have very limited treatment options. Using our expertise in neurological disorders, joint with our Brain Transporter technology platform, we hope to be able to change that and help these patients," said BioArctic's CEO Gunilla Osswald.

The information was released for public disclosure, through the agency of the contact persons above, on November 8, 2022, at 08:00 a.m. CET.

For further information, please contact: 
Gunilla Osswald, CEO                                                                        
E-mail: gunilla.osswald@bioarctic.se                                               
Phone: +46 8 695 69 30                                                                    

Oskar Bosson, VP Communications and IR
E-mail:  oskar.bosson@bioarctic.se 
Phone: +46 70 410 71 80 

About BioArctic AB

BioArctic AB (publ) is a Swedish research-based biopharma company focusing on disease-modifying treatments for neurodegenerative diseases, such as Alzheimer's disease, Parkinson's disease and ALS. BioArctic focuses on innovative treatments in areas with high unmet medical needs. The company was founded in 2003 based on innovative research from Uppsala University, Sweden. Collaborations with universities are of great importance to the company together with its strategically important global partner Eisai in Alzheimer disease. The project portfolio is a combination of fully funded projects run in partnership with global pharmaceutical companies and innovative in-house projects with significant market and out-licensing potential. BioArctic's Class B share is listed on Nasdaq Stockholm Mid Cap (ticker: BIOA B). For more information about BioArctic, please visit www.bioarctic.com.

^[1] https://gaucheralliance.org/what-is-gaucher-disease-2/

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