Betting on modified vs. natural

Morquio syndrome only affects about 3,000 people in the developed world but Vivendy Therapeutics Ltd. and BioMarin Pharmaceutical Inc. believe there’s enough opportunity to compete for the market. Vivendy’s investors agree, as the company last week completed the second tranche of a CHF32 million ($30 million) series A round.

Also known as mucopolysaccharidosis (MPS) IVA, Morquio is an autosomal recessive lysosomal storage disease that occurs due to a lack of N-acetylgalactosamine 6-sulfate sulfatase (GALNS). The enzyme is involved in the breakdown of keratin sulfate, which is primarily found in bones and connective tissue...