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