CARsgen Therapeutics and Jaguar Gene Therapy had received incentives in 2021 to hire 200 workers each at new facilities.
Thanks to advances in imaging and diagnostic technologies, clinicians can now detect many genetic disorders in the womb, ...
Still, broad uptake has long been an uphill battle for the gene therapy as it competes with a variety of treatments including ...
RNA therapeutics target translation rather than DNA, aiming to correct shared protein production errors. By enabling cells to ...
After a rocky 2025, Sarepta Therapeutics’ executives admit they have work to do to bring patients back into the fold as sales ...
CARsgen Therapeutics and Jaguar Gene Therapy received incentives in 2021 to create around 200 jobs each at new facilities.
If you are wondering whether uniQure's current share price reflects its true worth, you are not alone. This article walks ...
AMT-130 Huntington’s gene therapy data, FDA Phase III sham-trial push, cash runway, and 2026 catalysts—read now.
Hunter Syndrome is a rare genetic condition that can cause damage to the body and the brain. It often causes children to pass away in their teens, but a UNC Health ...
Recent transactions make clear that large pharmaceutical companies now view rare disease as a core area for sustained growth, ...
When Ingram became Sarepta Therapeutics’ CEO in 2017, he didn’t have a connection to muscular dystrophy, but he has developed ...
How innovative technologies like AI and gene editing are advancing rare disease drug discovery and development.
Some results have been hidden because they may be inaccessible to you
Show inaccessible results