rare disease
Intellia Cuts AATD Gene Insertion Program, 27 Percent of Workforce in Strategic Reorganization
These moves will enable the gene-editing company to focus resources on late-stage programs as it aims to launch a commercial product by 2027, it said.
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.
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.
FDA Approves New 'Vanza Triple' Cystic Fibrosis Drug From Vertex, Expands Label for Trikafta
Alyftrek is approved as a new treatment for cystic fibrosis, while the FDA expanded the label for Trikafta to include additional genetic mutations.
Ultragenyx Submits BLA for Sanfilippo Syndrome Type A Gene Therapy, Seeks Accelerated Approval
The company separately said it has started treating patients within a Phase III trial testing an ASO drug for Angelman syndrome.
Dec 11, 2024