As Eli Lilly awaits an FDA approval decision for its oral GLP-1 orforglipron, the pharma has discarded three clinical-stage drugs, including a gene therapy from its $1 billion acquisition of Prevail ...
Life Biosciences is pushing cellular rejuvenation into the clinic with ER-100, an experimental gene ...
Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...
Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...
The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...
A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...
A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.
Studies of Hunter and Hurler syndromes on hold ...