"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...

Cell and gene therapy are revolutionary approaches to treating diseases at the molecular level. They involve the manipulation ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

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 ...

An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...

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, ...

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 ...

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 ...

Genetic treatments could be the key to unlocking some rare diseases. But bringing these medicines to patients involves ...

According to DataM Intelligence, the global cell and gene therapy market reached a value of US$13.90 billion in 2024 and is projected to expand significantly to US$105.83 billion by 2033, registering ...