Fondazione Telethon Announces FDA approval of Waskyra(TM) (etuvetidigene autotemcel), a Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome

ROME, Italy, Dec. 10, 2025 /PRNewswire/ -- Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex ...
Medscape

Could Gene Therapy for RA Soon Be Ready to ‘Take Off’?

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...
Hosted on MSN

First-in-human trial of CRISPR gene-editing therapy safely lowers cholesterol and triglycerides

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, ...
New Atlas

One-off lipid-lowering gene therapy a success in world-first human trial

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...
Observer

This Longevity Startup Is Bringing Anti-Aging Gene Therapy to Human Trials

Boston-based Life Biosciences is testing whether reprogramming human cells can slow or even reverse the effects of aging. Unsplash Life Biosciences, a Boston-based biotechnology company founded in ...
Wired

A New Startup Wants to Edit Human Embryos

In 2018, Chinese scientist He Jiankui shocked the world when he revealed that he had created the first gene-edited babies. Using Crispr, he tweaked the genes of three human embryos in an attempt to ...
Reuters

CRISPR gene therapy slashes 'bad' cholesterol, triglycerides by half in small study

CHICAGO, Nov 8 (Reuters) - A single infusion of CRISPR Therapeutics' (CRSP.BN), opens new tab experimental gene therapy was safe and reduced levels of harmful LDL cholesterol and triglycerides by half ...

This gene therapy costs $5 million a dose. Is it worth the price?

You’re reading an excerpt – sign up to get the whole newsletter in your inbox.Born in 1949, Chip Hailey is old enough to ...
The New York Times

Mutated DNA Restored to Normal in Gene Therapy Advance

The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely targeted infusions. By Gina Kolata Researchers have corrected a disease-causing ...
PharmiWeb

EpilepsyGTx Raises $33 Million Series A to Develop Single Dose Gene Therapy for Focal Refractory Epilepsy

Investment from XGEN Venture, British Business Bank, and a global biopharmaceutical company will support first-in-human Phase ...
Finanznachrichten

Fondazione Telethon Announces FDA approval of Waskyra (etuvetidigene autotemcel), a Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome

WAS affects almost exclusively males, with an estimated incidence of 1 in 250,000 live male births. The therapy represents a major scientific and clinical achievement, offering new hope for patients ...