AVALA Hospital, in partnership with Retina Associates New Orleans, has successfully performed Louisiana’s first ENCELTO procedure, a cell-based gene therapy for adults with macular telangiectasia type ...
The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...
Subjecting participants to invasive procedures without the prospect of therapeutic benefit is unjustifiable,” says the CEO of ...
A senior FDC official said the product is a “failed therapy” and confirmed that regulators have asked uniQure to run a new trial to prove the therapy actually helps Huntington’s patients. The ...
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QURE jumps 20% – analysts upgrade stock on Huntington’s disease therapy update, retail eyes $150
UniQure’s AMT-130 is a gene therapy to reduce levels of the Huntingtin protein in the brain. ・The FDA recently said that data from uniQure’s Phase I/II trials were not sufficient and recommended a new ...
In a strikingly blunt briefing, a senior FDA official accused uniQure of pushing “distorted” data to mask a “failed” therapy ...
Regeneron has posted updated clinical data on DB-OTO, providing more evidence of the gene therapy’s potential to treat a rare genetic form of deafness as it plans for a potential FDA filing later this ...
The U.S. Food and Drug Administration (FDA) has chosen Dr. Vinay Prasad, a professor at the University of California-San Francisco, to lead its Center for Biologics Evaluation and Research. An ...
Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...
A Spanish study shows that patients with genetic dilated cardiomyopathy who experience severe arrhythmias are at an elevated risk of developing advanced heart failure and requiring a heart transplant.
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