gene-therapy

An excellent overview and analysis by Patrick Mineault on the exciting news that 7 chemogenetics clinical trials have begun in China! I very much hope that these trials show enough promise that they stimulate further work in this area. Spotlight on DREADDs and chemogenetics.

The collaboration brings together Synastra’s expertise in genomic engineering, AAV vector design, rare-disease gene therapy, and translational development with NewBiologix’s proprietary cell engineering and rAAV manufacturing technologies. The post NewBiologix and Synastra Join Forces to Advance rAAV Manufacturing for Duchenne Gene Therapy appeared first on GEN - Genetic Engineering and Biotechno…

The birth of Louise Brown in 1978, the world's first baby conceived through assisted reproductive technology (ART), was a major breakthrough that reshaped the field of reproductive medicine.

Furthermore, the researchers observed that in over half of the participants, the tumors stopped growing or reduced in size for as long as three years post-treatment—including one participant whose cancer has remained to this day in complete remission. B7-H3-targeting CAR-T cell therapy could bring new hope to patients living with treatment-resistant recurrent glioblastomas who currently […]

Nature, Published online: 01 September 2026; doi:10.1038/d41586-026-02740-w Small trial targeted liver proteins that play a part in the regulation of lipid levels.

A Rutgers-led international team co-authored a study, which was published in Nature Neuroscience, revealing 36 genes significantly increasing the risk for obsessive-compulsive disorder (OCD) and chronic tic disorders. This research offers the most comprehensive biological insight into the development of these conditions and potential future treatments.

Longwei Liu is working to create cures that remotely trigger cell and gene therapies for conditions including cancer and a genetic disorder that can rob infants of their vision. The post USC researcher uses ultrasound, genetic engineering to develop targeted treatments for blindness and other diseases appeared first on USC .

Inherited retinal dystrophies (IRDs) represent heterogeneous genetic eye disorders frequently linked to RPE65 mutations. Voretigene neparvovec (VN, Luxturna), an AAV2-mediated gene therapy, remains the sole approved drug for biallelic RPE65-associated IRDs. This study presents 3-, 6- and 22-month follow-up findings of the first mainland Chinese patient with compound heterozygous RPE65-related Leb…

Duke researchers developed an injectable scaffold that helped stroke-damaged brains grow new blood vessels, support nerve regrowth, and recover movement in mice. The treatment appears to work partly by recruiting the body’s own immune cells, including neutrophils that may switch from damaging to helpful under the right conditions.

In January 2026, Tim Andrews, who had received a genetically edited pig kidney in January 2025, became the recipient of a human kidney.

Tim Andrews was the first patient to receive a genetically modified pig kidney as a “bridge” to a human organ transplant.

Two recipients of gene-edited pig kidneys have successfully received human organs, a milestone in the wider effort to advance xenografts.

Scientists from Nanyang Technological University, Singapore (NTU Singapore) have developed microscopic peptide droplets that deliver large amounts of gene-silencing molecules into cancer and immune cells, reducing the growth of hard-to-treat colorectal tumours by about 67 per cent in mice.

This eBook brings together expert perspectives on several of the most pressing analytical topics facing today’s cell and gene therapy developers. The post The Analytical Blueprint for Cell & Gene Therapy Success appeared first on GEN - Genetic Engineering and Biotechnology News .

BCL6, a driver of lymphoma, suppresses genes involved in cell death. A new molecule designed at Stanford Medicine blocks BCL6 activity and activates downstream genes that trigger cell death. A protein that helps keep lymphoma cells growing can be turned against the cancer itself. Stanford Medicine researchers designed a molecule that redirects one of the [...]

A new program from ARPA-H has devoted funding to make individualized RNA-based genetic medicine more efficient to produce and improve accessibility.

Massachusetts General Hospital is among the teams selected by the Advanced Research Projects Agency for Health (ARPA-H) to help make personalized genetic medicine manufacturing possible. The initiative is aimed at accelerating the development of individualized genetic therapies by improving how these treatments are designed, produced, and delivered. The multidisciplinary team includes MGBs Gene C…