According to a publication from Biospace, British clinical drug developer Orchard Therapeutics has secured global intellectual property rights to research, manufacture, and market a gene therapy program for the treatment of mucopolysaccharidosis Type I (MPS-I).

The gene therapy, originally developed by the San Raffaele-Telethon Institute for Gene Therapy in Milan, Italy, is based on ex vivo autologous hematopoietic stem cells.

About Mucopolysaccharidosis

Mucopolysaccharidosis (MPS) is a group of genetically inherited disorders characterized by the body’s inability to effectively break down long sugar molecules called mucopolysaccharides. If the body can’t effectively break the sugars down, they can accumulate in cells, blood, and connective tissue.

Cases of MPS are further categorized into seven different types, numbered I-IX. Types V and VIII, though once accepted in medicine, were over time discovered to be non-distinct conditions. “Type V” MPS was found to be a subtype of MPS-I, and “Type VIII” was misdiagnosed entirely (it is not recognized as a valid medical condition).

The symptoms of MPS, and their severity, are significantly dependent on the specific type encountered.

Mucopolysaccharidosis Type I is caused by a mutation in the gene IDUA, which is normally responsible for coding the production of an enzyme called IDUA. IDUA is responsible for assisting the breakdown of glycosaminoglycans (GAGs – long sugar molecules) in lysosomes, cell organelles that play key roles in digestion and waste removal. Mutations to the IDUA gene can result in a lack of IDUA throughout the body, causing GAGs to build up in lysosomes.

The result can be swelling of different organs and tissues throughout the body. The current standard of care is stem cell and enzyme replacement therapies.

Orchard Therapeutics Acquires Gene Therapy Rights

Orchard Therapeutics acquired the program from the San Raffaele-Telethon Institute for Gene Therapy.

The program seems especially promising at treating Hurler syndrome, a severe form of MPS-I that typically presents symptoms in the first years of life, based on preliminary data. The deal involved an upfront cash payment of an undisclosed amount, along with contingent payments upon reaching major development milestones, and royalties upon market authorization.

Participants are currently being enrolled in an ongoing proof-of-concept study.

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