Gene Therapy - Search News

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

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 undergoing Lyfgenia, a new type of gene therapy.

Yahoo · 9h

Gene therapy cures man from sickle cell

A sickle cell gene therapy treatment has allowed a Long Island man to turn a chapter in his life. He was Sebastien Beauzile was born with sickle cell anemia and struggled to live a pain free life. However,

WRAL · 2d

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.

WPIX New York City, NY on MSN · 12h

'I'm Unstoppable': New gene therapy cures first New Yorker of sickle cell anemia

Thanks to his medical team, Sebastien is grateful for becoming the first person in New York to receive a new genetic treatment, Lyfgenia, to cure his sickle cell disease.

New York Post on MSN · 1d

Long Island patient gets breakthrough sickle cell treatment called a ‘cure’: ‘I’m not in pain anymore’

He’s got new blood. A Long Island hospital said it administered life-changing Lyfgenia gene therapy treatment and successfully wiped away sickle cell disease from a Laurelton man. Cohen Children’s Medical Center said patient Sebastien Beauzile,

EL PAÍS English1d

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 ...

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.

1don MSN

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 ...

Healio21h

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 ...

BioPharma Dive2d

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 ...

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 ...

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