New gene therapy cures 1st New Yorker of sickle cell anemia
The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...
Mía had dystrophic epidermolysis bullosa, better known as butterfly skin due to the extreme fragility of this tissue in ...
Pharmaceutical Technology on MSN8h
MeiraGTx and Hologen launch AI-backed gene therapy ventureThe venture aims to advance MeiraGTx’s Parkinson’s gene therapy through Phase III trials with up to $430m in funding from ...
Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders.
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit that the Swiss pharma ...
But they are not the same. Gene therapy, according to the FDA, modifies a person’s genes to treat or cure disease, and can ...
The deal ends 2Seventy’s short run as an independent company, during which it restructured, sold off research and lost nearly ...
X-linked severe combined immunodeficiency disease (X-SCID) is a rare genetic disorder characterized by profound defects in ...
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 ...
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