The University of Colorado Anschutz Gates Institute has received Investigational New Drug clearance from the U.S. Food and Drug Administration, marking the first chimeric antigen receptor (CAR) T-cell ...
A key cancer-fighting gene in leukemia is switched off—not broken—and scientists from The Jackson Laboratory (JAX) have found ...
The authors describe the scientific foundations of a study of gene therapy for hearing loss caused by damaging variants in the gene OTOF. Department of Otorhinolaryngology and Head and Neck Surgery, ...
UniQure NV’s shares plunged after US regulators said the company should conduct another study of its Huntington’s disease gene therapy before it’s approved. The US Food and Drug Administration ...
As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated in the United States and Europe remains financially out of reach for most ...
Growing revenues from Casgevy will help CRISPR Therapeutics fund the development of its pipeline therapies. The gene-editing biotech isn't profitable yet. CRISPR Therapeutics has a deep pipeline of ...
Hemophilia usually is an inherited bleeding disorder in which the blood does not clot properly. Patients with severe hemophilia A may have to manage their condition with regular at-home injections or ...
Cystinosis is a multisystemic lysosomal storage disorder caused by pathogenic variants in CTNS, the gene encoding cystinosin, a lysosomal transmembrane cystine transporter. In patients with cystinosis ...
Kyoto, Japan -- Cell and gene therapies, or CGT, have come a long way since they were first introduced. In the last few decades, both cell therapy -- the transplantation of living cells -- and gene ...
A first-of-its-kind inhalable gene therapy for lung cancer that genetically modifies people’s lung cells has been fast-tracked towards potential approval after promising clinical trial results. “Very ...
When Harith Rajagopalan considers the millions of patients who have taken a GLP-1 drug to treat diabetes or obesity, he sees a revolution that is failing to realize its promise. “We are literally ...
Ocugen has reported preliminary phase 2 data on its eye disease gene therapy, providing more evidence that the candidate may outperform existing treatments from Apellis Pharmaceuticals and Astellas ...
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