Teen Beats Deadly Leukemia With Life-Saving Gene-Edited Cell Therapy

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...
STAT

Gene therapy spurs improvement in rare neuromuscular disease

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...
NJBIZ

FDA approves Novartis’ Itvisma gene therapy for SMA

Novartis’ Itvisma wins FDA approval as the first gene replacement therapy for spinal muscular atrophy (SMA) patients age 2 ...
BioSpace

Novartis Expands Spinal Muscular Atrophy Gene Therapy Market With FDA’s Itvisma Greenlight

The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access ...

California boy, 3, receives first-ever gene therapy for his rare disease: ‘It’s just so exciting’

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...
Fierce Pharma

ASH: CSL proves efficacy of hemophilia B gene therapy Hemgenix holds up after 5 years

Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...
Fierce Biotech

Regeneron makes $150M bet on Tessera's rare disease gene writing prospect

Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene writing program, inking a deal that lets it in on a ...

3-Year-Old Boy with Rare Condition Amazes Doctors by Becoming World's First Gene Therapy Patient

Oliver Chu was born with a rare genetic condition called Hunter syndrome that impacts physical and mental development. The toddler has now exceeded doctors' expectations after becoming the first ...
Fred Hutch

Gene therapy delivery system successfully targets CD90+ hematopoietic stem cells in vivo

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...
Hosted on MSN

Columbus doctors turn to gene therapy for patients in continued fight against sickle cell disease

COLUMBUS, Ga. (WRBL) — September is Sickle Cell Awareness Month, and millions of people worldwide are living with the disease. But advances in treatment are being made. WRBL News 3 anchor Cameron ...
Medscape

FDA OKs New Formulation of SMA Gene Therapy Itvisma

Itvisma contains the active ingredient in Zolgensma approved in 2019 for children younger than 2 years but is reformulated for intrathecal administration across age groups.
EurekAlert!

Pharmacy professor receives $1.9 million award to tackle gene therapy challenges

Assistant Professor of Pharmaceutical Sciences KC Mei standing in his lab at the School of Pharmacy at Binghamton University, State University of New York. BINGHAMTON, N.Y. -- A pharmacy professor at ...