Topic: gene therapy

GSK inks money-back deal on $665K gene therapy Strimvelis

GlaxoSmithKline has struck a money-back guarantee on its gene therapy Strimvelis. The once-and-done treatment for “bubble boy” disease, only the second of its kind, raised questions about pricing and payment in the up-and-coming field. GSK’s arrangement offers some potential answers.
DNA

Gene transfer shows success in neurodegenerative disorders

Researchers have uncovered a new gene therapy that can replace a deficient enzyme in patients suffering from the neurodegenerative disorders Tay-Sachs and Sandhoff. In a pair of separate studies, they were able to show the feasibility and efficacy of gene transfer in preclinical models.