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 ...
LVB1d
Helping Employers Prepare for Gene and Cellular Therapies
Cellular therapy, according to the Dana-Farber Cancer Institute, is designed to improve the immune system’s ability to fight ...
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.
Pharmaceutical Technology on MSN17h
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 ...
Fierce Biotech1d
Hologen AI commits $430M to MeiraGTx Parkinson’s gene therapy
With MeiraGTx eyeing up how to take the drug through phase 3 and onto commercialization, Hologen has stepped in. The ...
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 ...
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 ...
Fierce Pharma4d
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 ...
Medera Completes Patient Dosing in Phase 1/2a MUSIC-HFrEF Trial of SRD-001 Gene Therapy for Heart Failure with Reduced Ejection Fraction
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 ...