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 ...

Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...

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

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 ...

The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...

(Yicai) Jan. 28 -- Chinese medical researchers have made a breakthrough in gene therapy that overcomes the limitations of ...

The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...

Studies of Hunter and Hurler syndromes on hold ...

Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of avigbagene parvec (FLT201) ...