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 ...

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 ...

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 ...

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 ...

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, ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

ELRIG, a not-for-profit, volunteer-led organisation dedicated to the global drug discovery community, today announced the keynote speakers for Cell and Gene Therapy 2026. Drs Stephen Ward (Cell and ...

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