gene-therapy
8 stories tagged "gene-therapy"
China Runs More Drug Trials Than America. The FDA Says It Can No Longer Trust Data From Sites It Can't Inspect.
China went from under 8 percent of the world's drug trials in 2010 to passing the United States by 2020. After three deaths in Chinese studies surfaced late, the FDA says it has sometimes been denied access to the very sites whose data underpins American approvals.
InvestigationsA Chinese Registry Marked the Trial 'Complete.' It Never Mentioned the Boy Who Died.
A boy died in a Chinese gene-therapy trial in August 2025. Months later the registry marked the study 'complete' and never mentioned him. He is one of three deaths now pulling Congress toward the trial data U.S. drugmakers ship home from China, while American inspectors almost never see the sites th
Drugs & FDAA $2.7 Million Gene Therapy, Approved Because Patients Needed Less Cornstarch
Ultragenyx's Genglycos won accelerated approval on a surrogate: a 31 percent cut in daily cornstarch. But the disease is low blood sugar, and by the company's own numbers the treated group spent slightly more time below 70 mg/dL, while liver enzymes spiked in 71 percent and every patient needed ster
Drugs & FDAThe FDA Cleared a CRISPR Cure for Two-Year-Olds Whose Own Trial Enrolled No One That Young
Casgevy's pediatric trials enrolled 11 children aged five and up, yet the FDA extended the $2.2 million therapy down to age two, where the conditioning chemo can permanently sterilize a child too young to bank eggs or sperm.
CancerGlioblastoma Craves Sugar. Oregon State Coated a Gene Therapy in It and Rode Straight Past the Blood-Brain Barrier.
Glioblastoma overexpresses the same sugar transporter that guards the brain. Oregon State researchers used that greed to smuggle a tumor-suppressor gene into tumors, and mouse survival rose 50 percent.
Drugs & FDAThe FDA Cleared a Sickle-Cell Cure for Two-Year-Olds in 53 Days. It Starts With Chemo That Can Sterilize Them.
Casgevy is a real advance against a brutal disease. It is also a $2.2 million therapy that requires sterilizing chemotherapy, saw a child die in trials, and reached toddlers on a 53-day review Makary's FDA hands out as a favor.
Drugs & FDAThe FDA Office That Vets Gene Therapy Has Run Through Its Entire Top Tier in a Year
The office that decides whether gene therapies reach patients has lost its director, her deputy, and the man brought in to replace them, all inside twelve months.
Drugs & FDAFDA's New Gene Therapy Shortcut Leans on 'Platform Knowledge.' Six Months Ago, They Revoked Sarepta's After Three Patient Deaths.
FDA's new gene therapy draft guidance invites sponsors to lean on "platform knowledge" to streamline submissions, six months after the agency revoked Sarepta's AAVrh74 platform designation following three patient deaths.