Can a gene therapy work no matter the mutation? While there’s little chance of changing market dynamics, researchers said, one solution is to develop “mutation agnostic” gene therapies ...

Gene therapy is at the cutting edge of medicine, with exciting new products being developed and used to treat a wide variety ...

The new gene therapy works by modifying the patient's own immune cells to help them create the missing CD18 protein needed to fight infections. After being reintroduced into the patient's body ...

In April, the Deparment of Health and Human Services shuttered the committe that oversaw newborn screenings. Advocates hope ...

A gene therapy recently approved by the FDA to treat patients with Sickle Cell Anemia is now being administered in St. Louis.

From connectors and containers to plasmids and platforms, companies work to get these treatments to patients faster and more ...

Siren Biotechnology is trying to raise $5 million via the type of crowdfunding round that’s rarely used by young drugmakers.

A new gene therapy reversed heart failure in pigs by repairing heart function through cBIN1, showing major promise for future ...

The 11-year-old will be the first to receive an experimental antisense oligonucleotide gene therapy to treat NARS1 ... including whether it’ll even work. “I just have a lot of gratitude ...

"We are excited to continue our pioneering gene therapy work in diabetes through this SRA," said Mark Berger, MD, Chief Medical Officer of Genprex. "Our preclinical studies have produced ...