University of California

Scientists create cancer-fighting immune cells right in the body

A new form of CAR T kills leukemia, multiple myeloma, and sarcoma in mice, opening the door to a future off-the-shelf cancer treatment without chemotherapy.

An AI-guided gene-editing tool for more precise and safer DNA correction

Researchers at the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) have developed a revolutionary new method to improve compact gene-editing tools known as base ...

Huntington’s gene therapy research has reached a turning point, expert says

An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and challenges with the FDA.

1st Michigan patient undergoes FDA-authorized sickle cell gene therapy

Chantez Sanford Jr., 24, of Southfield, says his life has been transformed after undergoing Lyfgenia gene therapy at Children ...
Frontiers

Next-generation viral vectors for gene therapy: Innovations in engineering, safety, and translational development

Viral vector gene therapy represents a rapidly advancing field at the intersection of molecular medicine, biotechnology, and clinical translation. Viral ...
BioSpace

Denali’s Hunter Syndrome Candidate in the Spotlight After REGENXBIO Rejection

The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO's investigational gene therapy, ...
MPR

Gene Therapy DTX301 Reduces Ammonia in Patients With OTC Deficiency

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.
C&EN

FDA says it wants individualized medicines. Can we get there?

The drug was finally approved in September 2025 through the FDA’s accelerated approval pathway, based on improvement in knee extensor muscle strength, for adults and children with Barth syndrome who ...
New Scientist

First ever inhalable gene therapy for cancer gets fast-tracked by FDA

A first-of-its-kind inhalable gene therapy for lung cancer that genetically modifies people’s lung cells has been fast-tracked towards potential approval after promising clinical trial results. “Very ...
Drexel University

Breakthrough on Gene Therapy for Hereditary Spastic Paraplegia

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...
STAT

Child’s sudden death unnerves a promising area of gene therapy research

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
New York Post

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 ...