JCR has developed a technology to pass the blood-brain barrier (J Brain Cargo”, based on anti-transferrin receptor antibody). The objective of the forthcoming clinical trials is the treatment of Hunter’s disease, a deficiency of iduronic acid 2-sulfatase (IDS) which leads to a lysosomal diseases, mucopolysaccharidosis-2. In IDS knock-out mice, the resulting disease could be treated by administering a genetically modified IDS (fusion protein of IDS with the above antibody) preparation to the brain.”
JCR Pharma press release, November 2, 2016
JCR Pharma will start clinical tests on enzyme therapy in brain using their brain cargo” technology”