New gene therapy cures 1st New Yorker of sickle cell anemia

In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
Mía had dystrophic epidermolysis bullosa, better known as butterfly skin due to the extreme fragility of this tissue in ...
The venture aims to advance MeiraGTx’s Parkinson’s gene therapy through Phase III trials with up to $430m in funding from ...
The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...
Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...
A promising study out of the United Kingdom has partially restored the vision of multiple small children born legally blind.
Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders.
While initial study results suggest Beam's technology can correct alpha-1 antitrypsin deficiency's genetic roots, shares fell ...