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 ...
pharmaphorum5d
bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval
bluebird bio's Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment ... pharmaceutical industry products is evolving at ...
Hosted on MSN10mon
FDA-approved sickle cell gene therapy could cost patients millions
(KTAL/KMSS) – Two cell-based gene therapies for treating sickle ... Casgevy is the first FDA-approved therapy that utilizes CRISPR/Cas 9, a type of genome editing technology that modifies ...
FiercePharma29d
Pfizer doubles down with its 2nd FDA hemophilia approval in 6 months
Six months after scoring FDA approval for hemophilia B gene therapy Beqvez, Pfizer has earned a nod from the U.S. regulator for another of its products in the indication. On Friday, the FDA signed ...
BioWorld3d
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 ...
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 ...
The Business Journals26d
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 ...
Kaleido Scope11mon
Should FDA Approve Gene Therapy Exa-cell, 3 Key Implementation Challenges Loom
sheds light on the intricate process of implementing new gene therapy treatment for Sickle Cell Disease (SCD). 1. Complex Treatment Roadmap The FDA's potential approval of Exa-cell offers hope for SCD ...
FierceBiotech18d
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 ...
precisionmedicineonline1d
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.
News Medical10d
Gene Therapy News and Research
CRISPR/Cas9 is a gene editing tool that has revolutionized biomedical research and led to the first FDA-approved CRISPR-based gene therapy ... essential life science products for researchers ...
BioWorld10d
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 ...