Inherited Disease
News on Mendelian diseases, hereditary cancer, variant classification, cystic fibrosis testing and more.
In Brief This Week: Qiagen, Care Access, Immix Biopharma, Atsena Therapeutics, Aspen Neuroscience
News items for the week of Jan. 6, 2025.
Vertex Gains Rights to Orna Therapeutics' Drug Delivery Tech in $1.07B Deal
Vertex aims to further bolster its gene-editing therapy pipeline by using Orna's lipid nanoparticle delivery technologies during the three-year collaboration.
With new treatments in neurology, and more in development in cardiology and autoimmune diseases, precision medicine is moving beyond cancer and rare disease.
CBO Outlines Budgetary Considerations for Policies Focused on Sickle Cell Disease Gene Therapy
The Congressional Budget Office said policies to increase treatment uptake could affect Medicaid, Social Security, and other government programs.
Sangamo Regains Rights to Hemophilia A Gene Therapy After Pfizer Ends Partnership
Sangamo is seeking new partners to continue advancing giroctocogene fitelparvovec, which reported positive Phase III results this year.
Dec 11, 2024
Dec 2, 2024
Nov 27, 2024
In Brief This Week: Hemogenyx, Intellia, Care Access
Nov 22, 2024