A sickle cell gene therapy treatment has allowed a Long Island man to turn a chapter in his life. He was Sebastien Beauzile ...

Mía had dystrophic epidermolysis bullosa, better known as butterfly skin due to the extreme fragility of this tissue in ...

The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...

Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders.

Twenty-one-year-old Sebastien Beauzile was all smiles as he was serenaded with “Happy Birthday” on what he calls his new birthday at Cohen Children’s Medical ...

In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...

Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...

With MeiraGTx eyeing up how to take the drug through phase 3 and onto commercialization, Hologen has stepped in. The AI-focused company is handing over $200 million as an upfront payment, as well as ...

Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit that the Swiss pharma ...

Cellular therapy, according to the Dana-Farber Cancer Institute, is designed to improve the immune system’s ability to fight ...

Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases by developing a range of next-generation therapeutics. Medera operates via ...

Aaron Scott, of Fayetteville, is the first in North Carolina to receive a new FDA-approved therapy which uses the patient's own cells to relieve the pain of sickle cell disease.