Nippon Shinyaku Co Ltd of Japan is to acquire development and commercialisation rights to gene therapies for two rare metabolic disorders under a new agreement with Regenxbio Inc of the US. The deal relates to the development of therapies for mucopolysaccharidosis 1, also known as Hurler syndrome, and mucopolysaccharidosis 2, also known as Hunter syndrome. Both diseases are caused by the absence or malfunctioning of lysosomal enzymes which are needed to break down long chains of sugar carbohydrates.
