JCR Pharma has developed a blood-brain-barrier (BBB) passage technology under the brand name of „J-Brain Cargo“. After announcing to work on a treatment of Hunter’s disease (JR-141) which is based on passage of the BBB, JCR now has announced to develop a genetically modified acidic α-glucosidase (JR-162) which is transported to the brain via J-Brain Cargo and to skeletal muscle and is investigated in mice and monkeys as a treatment for Pombe disease. Pombe disease (acid maltase deficiency) is a lysosomal disease, where glycogen accumulates due to deficiencies of lysosomal acidic α-glucosidase which degrades glycogen It affects the heart and skeletal muscles and progresses with gait disturbance, orthostatic disorder, and breathing difficulties due to respiratory muscle dysfunction.

JCR Pharma news release, February 23, 2017