Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

Non-profit organisations play a crucial role in supporting rare disease therapy development and rescuing shelved programmes, ...

Progress of in vivo gene therapy for use in malignancies, monogenic disorders, and degeneration disease. AAV, adeno-associated virus; TK, thymidine kinase; ADA-SCID, adenosine deaminase-deficient ...

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission. A paper published in Science ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

Investing.com -- UniQure NV (NASDAQ:QURE) shares tumbled 36% after the U.S. Food and Drug Administration informed the company that data from its early- to mid-stage trial of AMT-130, a gene therapy ...

Health systems learn why cell and gene therapy success depends on workflows, staffing, and finances—not just science—and how to build them.