gene-therapy
12 stories tagged "gene-therapy"
The Sepsis Drug Eli Lilly Withdrew in 2011 Has a Second Life in a Dish of Blood Stem Cells
Soaked in activated protein C for one hour, human blood stem cells divided less, stayed quiescent, and engrafted better after transplant. The catch: the result lives in mice, and the molecule is one Eli Lilly withdrew as a failed sepsis drug in 2011.
Drugs & FDAGenSight's Blindness Gene Therapy Reached the NEJM on the Same Day It Reported €0.7 Million in Cash
Seven of ten patients blinded by retinitis pigmentosa sensed more light through special goggles after one gene injection, in an uncontrolled trial whose sponsor held €0.7 million on September 30 and warned it may not survive the year.
Drugs & FDAThe FDA Approved a $3.95 Million Gene Therapy It Rejected Fourteen Months Ago
In July 2025 the FDA refused to approve this Sanfilippo gene therapy and faulted the factory, not the science. Fourteen months later it is the first treatment for a fatal childhood brain disease, cleared on 17 treated children against a comparison group the maker built, at $3.95 million a dose.
Drugs & FDAThe Data on the First Sanfilippo Gene Therapy Were Ready Last Summer. The FDA Spent the Next Year on the Factory.
The FDA said Ultragenyx's clinical data were robust in July 2025, then rejected its Sanfilippo gene therapy over manufacturing questions. Approval came fourteen months later, for children who lose skills and die on a clock.
Drugs & FDAChina 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.