|Report Code: PP10144||Report Type: Indication Pipeline Reports||Available format:|
Mucopolysaccharidosis type I (MPS I) is a condition that affects many parts of the body. Earlier, MPS I disorder was divided into three separate syndromes; Hurler syndrome (MPS I-H), Hurler-Scheie syndrome (MPS I-H/S), and Scheie syndrome (MPS I-S), listed from most to least severe. Later on, these were clubbed as one disease, due to overlap between each of these three syndromes. Children with MPS I often have no signs or symptoms of the condition at birth, although some may have a soft out-pouching around the belly-button (umbilical hernia) or lower abdomen (inguinal hernia). People with severe MPS I generally begin to show other signs and symptoms of the disorder within the first year of life, while those with the attenuated form have milder symptoms that develop later in childhood. Common symptoms include, large head (macrocephaly), build-up of fluid in brain (hydrocephalus), heart valve abnormalities, distinctive-looking facial features, an enlarged liver and spleen (hepatosplenomegaly), and a large tongue (macroglossia).
The drug candidates of MPS I pipeline include, but are not limited to, JOT102, SB-318 and CRISPR/Cas9. Some of the companies having drugs in the MPS I pipeline are Jupiter Orphan Therapeutics Inc., Sangamo Therapeutics Inc., and CRISPR Therapeutics Ltd.