Sickle cell anemia patient reunites with Long Island doctors whose gene therapy treatments made him symptom-free
Sebastien Beauzile, 21, became the first person in New York state to be made symptom-free of sickle cell disease after ...
Pharmaceutical Technology on MSN16h
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 ...
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 ...
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 ...
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 ...
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 ...
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 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 ...
EurekAlert!2d
IL2RG gene therapy for X-SCID
X-linked severe combined immunodeficiency disease (X-SCID) is a rare genetic disorder characterized by profound defects in ...
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 ...
Sickle cell gene therapy frees teen from life of pain
Aaron Scott, of Fayetteville, is the first in North Carolina to receive a new FDA-approved therapy which uses the patient's own cells to relieve the pain of sickle cell disease.