A Critical Unmet Need Wilson's disease is a rare genetic disorder that affects approximately 1 in 30,000 individuals worldwide. The condition is caused by mutations in the ATP7B gene, which impair ...

is a gene therapy for Wilson’s disease being developed with Pfizer. The AAV vector gene therapy targets the ATP7B gene, responsible for causing the condition. It is currently being investigated ...

Splicing of pre-mRNA into mature mRNA is an essential step for gene ... Wilson’s disease is a rare disease caused by aberrant splicing of the ATPase copper transporting β (ATP7B) mRNA, which ...

Prime Medicine is also focused on advancing its Wilson’s Disease program, which targets prevalent mutations in the ATP7B gene. The company expects to initiate IND-enabling activities for this ...

Adapted from Schilsky ML, Tavil AS. Wilson disease. In: Schiff's Diseases of the Liver. Philadelphia: Lippincott Williams and Wilkins; 2006:1023–1040. Most practitioners prefer start with trientine.