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 ...
Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...
Life Biosciences is pushing cellular rejuvenation into the clinic with ER-100, an experimental gene ...
Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
ZipBio, a biotechnology company pioneering AI-driven protein therapeutics, announced an exclusive license agreement with MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage ...
Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...
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 five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.
The EMA’s positive opinion on Kygevvi marks a turning point for patients with TK2d, an ultra-rare genetic muscle disease previously managed only with supportive care.
Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of avigbagene parvec (FLT201) ...