Eli Lilly and Regeneron are leading the push to treat congenital deafness with gene therapies, seeking a piece of a potential ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying gene cargoes because of their superior gene segmentation flexibility and ...

Researchers developed a DNA barcoding system to track the behavior of gene therapy nanoparticles within cells. The method ...

All six participants tolerated surgical and intra-cochlear SENS-501 administration well, with no serious adverse events reported.

Scientists at the University of Science and Technology of China have identified a signaling chain that starts with gut ...

The cell and gene therapy industry has achieved breakthroughs once thought impossible, translating novel concepts into real therapeutic options for patients. The FDA has now approved 43 cell and gene ...

Strategies Shaping Industry Growth. EINPresswire/ -- The Cell And Gene Therapy market is dominated by a mix of global biopharmaceutical leaders and specialized biotechnology innovators, with companies ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...