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March 22, 2022 7:00 PDT
Adge Pharmaceuticals secures license to evaluate two new programs in rare pediatric diseases.

MOUNTAIN VIEW, CA – March 22, 2022 - Adge Pharmaceuticals Inc. has secured a global license to RO269228, a clinical stage oral small molecule originally developed by Roche for osteoporosis. Adge is now evaluating the molecule for the treatment of Rett syndrome and Osteogenesis imperfecta and potentially other indications with unmet medical needs.

Adge has complemented the already existing data package with relevant preclinical data for Rett Syndrome and Osteogenesis imperfecta and is in communications with the US FDA to move this asset expeditiously to the clinic.

Rett syndrome is a neurodevelopmental disorder that is caused by a mutation in the MECP2 gene and almost exclusively affects girls. It is characterized by normal early growth and development followed by a slowdown in development, slowed brain and head growth, loss of purposeful use of the hands, distinctive hand movements, problems with walking, seizures, autistic behavior and intellectual disability. Rett syndrome is the second most prevalent neurodevelopmental disorder in girls after Down syndrome affecting an estimated 1 in 9,000 to 10,000 females.

Osteogenesis imperfecta, also known as brittle bone disease, is a group of genetic diseases that primarily affect the bones. People with this condition have bones that fracture easily, often from mild trauma or with no apparent cause. Multiple fractures are common, and in severe cases, can occur even before birth. Osteogenesis imperfecta affects approximately 1 in 10,000 to 20,000 people worldwide. An estimated 25,000 to 50,000 people in the United States have the condition.

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