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Pfizer’s gene therapy for a muscle-wasting disorder misses main goal of late-stage study
WSAU Wausau· 4 days ago(Reuters) - Pfizer said on Wednesday its experimental gene therapy for a type of muscle-wasting...
What good are whizzy new drugs if the world can’t afford them?
The Economist· 2 days agoIN RECENT YEARS biotechnology and pharmaceutical science have produced quantum leaps that both offer great opportunity and pose great risk. Gene therapies for rare conditions and obesity drugs ...
Biomedicine Breakthrough: Complete Gene Insertion Now Possible in Human Cells
SciTechDaily· 3 days agoResearchers at the Broad Institute of MIT and Harvard have enhanced a gene-editing technology that can now efficiently insert or replace entire genes in human cell genomes, potentially making ...
Pfizer's Duchenne Muscular Dystrophy Treatment Misses Endpoints
Morningstar· 4 days agoPfizer's gene therapy being developed to treat Duchenne muscular dystrophy missed its primary endpoint in a late-stage trial. The pharmaceutical company ...
Regenxbio CEO to step down after 15 years
BioPharma Dive via Yahoo Finance· 4 days agoKenneth Mills will become chair of the gene therapy developer's board while Curran Simpson, the current chief operating officer, will take his place as...
Study suggests promising gene therapy for FOXG1 syndrome
UB Reporter· 5 days agoA master regulator gene, FOXG1 is one of the most important genes for early brain development and its impairment can result in profound brain structure abnormalities. The Lees previously established ...
Ultragenyx wins surrogate endpoint debate, securing green light to seek FDA approval of gene therapy
FierceBiotech· 3 days agoUltragenyx Pharmaceutical's work to get the FDA to recognize a biomarker as a surrogate endpoint looks to have paid off. With the agency agreeing the biomarker is a reasonable surrogate endpoint ...
Health Care Roundup: Market Talk
The Wall Street Journal· 3 days agoPublished exclusively on Dow Jones Newswires at 4:20 ET, 12:20 ET and 16:50 ET. 0748 ET – The failure of Pfizer’s proposed gene therapy for Duchenne...
FDA official: The risk of secondary cancer from CAR-T therapy, pioneered at Penn, is less than...
Philly.com· 2 days agoThe risk of secondary cancer after CAR-T therapy, pioneered at Penn, is less than regulators feared...
New tool to detect protein-protein interactions co | Newswise
Newswise· 4 days agoThis could pave the way for innovative medical therapies and advancements to be created using gene therapy – a technique that utilizes harmless viruses ...
