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

EL PAÍS English2d
The most expensive drug ever approved: A gene therapy that cures butterfly skin and could cost $20 million per patient
Mía had dystrophic epidermolysis bullosa, better known as butterfly skin due to the extreme fragility of this tissue in ...
Mutated DNA Restored to Normal in Gene Therapy Advance
The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...
Pharmaceutical Technology5h
MeiraGTx and Hologen launch AI-backed gene therapy venture
The venture aims to advance MeiraGTx’s Parkinson’s gene therapy through Phase III trials with up to $430m in funding from ...
This Founder’s Gene Therapy Company Helped Blind Kids See Again
Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders.
Preclinical study demonstrates improved gene therapy approach to treating a rare and devastating disease in children
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
BioPharma Dive3d
2seventy bio, Bluebird’s cell therapy spinout, sells to Bristol Myers for less than $300M
The deal ends 2Seventy’s short run as an independent company, during which it restructured, sold off research and lost nearly ...
Fierce Pharma3d
Roche overhauls Spark gene therapy unit, recording $2.4B in full impairment
Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit that the Swiss pharma ...
EurekAlert!1d
IL2RG gene therapy for X-SCID
X-linked severe combined immunodeficiency disease (X-SCID) is a rare genetic disorder characterized by profound defects in ...
Healio1d
Q&A: ‘An exciting breakthrough’: Gene transfer therapy promising in Danon disease
Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...