Experimental gene therapies like AMT‑130 lower toxic huntingtin protein and significantly slow Huntington’s disease, offering ...

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 ...

A free two-day event in Cambridge will unite experts to discuss translational advances in cell and gene therapy, spanning rare diseases, cancer immunotherapy, and genome editing.

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 ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

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, ...

Genetic medicines could treat certain diseases, however, the current delivery systems available—viral vectors and lipid nanoparticles (LNPs)—pose unique challenges for gene therapy applications. For ...

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