"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are common-sense reforms that will address the unique characteristics of cell and ...

GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

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 ...

ELRIG has revealed Drs Stephen Ward (Cell and Gene Therapy Catapult) and Annarita Miccio (Imagine Institute) as keynote ...

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 ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

The global cell and gene therapy market is experiencing explosive growth. Key growth factors include rising chronic/genetic disease prevalence, advancements in gene editing (CRISPR) and viral vectors, ...

ELRIG, a not-for-profit, volunteer-led organisation dedicated to the global drug discovery community, today announced the ...

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

Researchers at the new Center for Brain Health at UT Health San Antonio are studying midlife testing for early-onset dementia with blood-based biomarkers to see if they can detect disease activity up ...

Is Obesity Genetic or Environmental? Get All the Details This article was reviewed by Lynn Marie Morski, MD, JD. Key ...