Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...
Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...
GlobalData on MSN
Genespire to take rare disease gene therapy to clinic in 2026
GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.
The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of avigbagene parvec (FLT201) ...
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
Stocktwits on MSN
Rare stock slips 3% today — what’s up with Ultragenyx’s gene therapy for Sanfilippo syndrome?
Ultragenyx now expects a 6-month review period for the therapy and a likely decision from the FDA regarding potential ...
Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic defect.
MedPage Today on MSN
FDA Halts Two Gene Therapy Trials After Child Develops Brain Tumor
Studies of Hunter and Hurler syndromes on hold ...
U.S. drugmaker Eli Lilly and Company signed an agreement worth up to $1.12 billion with Seamless Therapeutics, the ...
The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...
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