Mía had dystrophic epidermolysis bullosa, better known as butterfly skin due to the extreme fragility of this tissue in ...
Cellular therapy, according to the Dana-Farber Cancer Institute, is designed to improve the immune system’s ability to fight ...
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.
The venture aims to advance MeiraGTx’s Parkinson’s gene therapy through Phase III trials with up to $430m in funding from ...
With MeiraGTx eyeing up how to take the drug through phase 3 and onto commercialization, Hologen has stepped in. The ...
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
The deal ends 2Seventy’s short run as an independent company, during which it restructured, sold off research and lost nearly ...
Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit that the Swiss pharma ...
Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...
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 ...