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 ...

Safe and reproducible delivery of gene therapy vector into the subretinal space is essential for successful targeting of the retinal pigment epithelium (RPE) and photoreceptors. The success of surgery ...

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 ...

Researchers have developed a new and improved viral vector -- a virus-based vehicle that delivers therapeutic genes -- for use in gene therapy for sickle cell disease. In advanced lab tests using ...

An optimized and newly engineered form of the adeno-associated vector 9 (AAV9) vector used to deliver the galactosylceramidase gene to a mouse model of the inherited neurogenerative and rapidly fatal ...

Gene therapy developers targeting diseases requiring high dose therapies face a challenge. Current vector production cell lines and genetic manipulation techniques are too costly for large-scale ...

GAITHERSBURG, Md., June 24, 2025 /PRNewswire/ -- Vector BioMed, a purpose-driven contract vector development and manufacturing organization (CVDMO) focused on advancing access to cell and gene ...

Return to see the world's first patient treated with a new 'dual vector' gene therapy for a rare genetic disease that also affects the retina, Usher syndrome type 1B. The innovative procedure was ...

For biopharmaceutical companies, gene therapies are an economic challenge. They are expensive to make yet the diseases the treat are rare, so manufacturers tend to charge a lot for them to generate ...

Epilepsy affects more than 50 million people worldwide, making it one of the most common neurological disorders. Although ...

Gene therapy has provided treatment options for diseases that are beyond the reach of traditional approaches. Since 2016, between the European Medicines Agency (EMA) and the U.S. Food and Drug ...