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The FDA just approved the first gene editing therapy for sickle cell anemia, but it'll cost $2.2 million per person
The FDA approved the first gene therapy treatments for sickle cell disease on Friday, but it will cost patients millions of dollars. One of the approved therapies, developed by Vertex ...
Hosted on MSN11mon
FDA approves first human gene editing therapy for sickle cell disease
It's the first approved human gene editing therapy. The FDA said Casgevy and Lyfgenia are the two milestone cell-based gene therapies approved for sickle cell treatment. Sickle cell disease ...
pharmaphorum1y
FDA approves Adstiladrin as first gene therapy for NMIBC
The application was granted Priority Review, Breakthrough Therapy, and Fast Track designations. The FDA approval makes Adstiladrin the first gene therapy to be approved for adult patients with ...
pharmaphorum1d
FDA clears Vyjuvek as first topical gene therapy
The FDA has approved Krystal Biotech’s gene therapy Vyjuvek for dystrophic epidermolysis bullosa (DEB), a rare genetic disease characterised by fragile skin that can split and blister even with ...
The Business Journals29d
Nanoscope Therapeutics to seek FDA approval for lead product
Nanoscope said it will now begin the application process for approval to sell its gene therapy, MCO-10. "We are pleased with the positive interactions we have had with FDA as a result of the ...
US FDA extends review of Merus' gene-targeting cancer therapy
The U.S. Food and Drug Administration on Tuesday extended the review of Merus NV's gene-targeting cancer therapy to allow ...
U.S. News & World Report1y
Megatrends: Gene Therapy and Editing
"We've seen the pipeline of treatments for genetic diseases like sickle cell disease, but also certain types of cancer – just in December, the FDA approved ... of new gene therapy products ...
FierceBiotech21d
Sangamo slashes time to market for Fabry gene therapy as FDA agrees to accelerated approval package
The biotech pumped the brakes on the Fabry gene therapy, ST-920 ... but has now established a route to a submission for FDA approval in the second half of 2025. Sangamo previously provided ...
BioWorld5d
FDA eases the way for cell and gene therapy companies
Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture ...
Yahoo Finance12d
bluebird bio (BLUE): Promising Gene Editing Stock with FDA-Approved Therapies
At the forefront of modern biotechnology are gene therapy and gene editing. Gene therapies correct genetic defects by introducing new genetic material at the cellular level, often by adding a ...
precisionmedicineonline4d
Rocket Pharma Starts Rolling BLA Submission for Fanconi Anemia Gene Therapy
RP-L102, which the firm designed to treat a form of disease caused by FANCA mutations, is already under review with the European Medicines Agency.
BioWorld13d
BioFuture 2024: FDA eases the way for cell and gene therapy companies
Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is ...