ION356 is an investigational antisense oligonucleotide (ASO) medicine designed to decrease the production of proteolipid protein-1 (PLP1) for the potential treatment of male children with a diagnosis of Pelizaeus-Merzbacher disease (PMD).

About Pelizaeus-Merzbacher Disease (PMD)

PMD is one of a group of disorders known as leukodystrophies, which impact the growth of the myelin sheath – a protective covering that forms around nerve fibers in the brain and is vital in the sending of electrical impulses. PMD is a rare X-linked recessive leukodystrophy that is caused by duplications, mutations, or deletions in the gene that controls the production of a myelin protein called proteolipid protein-1 (PLP1). These changes can affect coordination, ability to move independently, and cognitive function. Symptoms may be present from birth, but onset and presentation can vary from person to person. There are currently no disease-modifying medicines for PMD on the market.

Safety and efficacy have not been evaluated by any regulatory authorities for the indication described.

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