Search results
3 days ago · This method involves the delivery of transgenes that can alter proteins involved in the pathogenesis or protection against AD. The primary objective of gene therapy in AD is to modify the disease process by enhancing neuroprotection, reducing pathological proteins, and modulating function.
5 days ago · Spur is advancing a breakthrough gene therapy candidate for Gaucher disease and a potential first-in-class gene therapy candidate for adrenomyeloneuropathy, as well as a research strategy to move gene therapy into more prevalent diseases, including forms of Parkinson’s, dementia, and cardiovascular disease.
5 days ago · The FDA’s approval in December of a new gene editing treatment for sickle cell disease not only represents a potential cure for a painful illness that afflicts millions of people globally...
2 days ago · One month ago, the FDA approved two gene therapies for sickle cell disease, including the first CRISPR drug to get the agency's nod. At the Informa Biotech Showcase on the outskirts of the JP...
3 days ago · This drug is being developed for the treatment of Hereditary Angioedema (HAE), a rare genetic disorder, and is expected to enter Phase 3 trials by the end of the year for both on-demand and...
2 days ago · Axovant attempted to reinvent itself as a gene therapy company, but dissolved in 2023. In 2017, Roivant partnered with the private equity arm of the Chinese state-owned CITIC Group to form Sinovant. In 2017, Ramaswamy struck a deal with Masayoshi Son in which SoftBank invested $1.1 billion in Roivant.
5 days ago · Twelve gene therapies have been approved by the FDA to date to treat a variety of diseases, including the first ever gene edited therapeutic. The CMC package required to effectively...
