GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

Astellas Gene Therapies remains intent on treating X-linked Myotubular Myopathy (XLMTM) through a gene therapy—but instead of AAV-based resamirigene bilparvovec (AT132), Astellas is now partnering ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

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, ...

VectorBuilder pioneers cutting-edge innovation for AAV gene delivery, with a one-stop solution supporting programs from research to early discovery to clinical development. Its proprietary technology ...

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a ...

Partnership accelerates AGC Biologics' leadership in the AAV market, applying its BravoAAV™ platform to complex gene therapies for inherited blindness MILAN--(BUSINESS WIRE)-- On the heels of ...

The cell and gene therapy sector has never lacked attention, but all too often, the narrative swings from one extreme to ...

Ocugen has reported preliminary phase 2 data on its eye disease gene therapy, providing more evidence that the candidate may ...

Sarepta Therapeutics Inc. made known a third gene therapy death, this time with SRP-9004 for limb-girdle muscular dystrophy. The patient was a late-stage, non-ambulatory 51-year-old man participating ...