Gene therapy, childhood blindness

Gene therapy experiment gives children 'life-changing' sight boost
An experimental trial of gene therapy has helped four toddlers - born with one of the most severe forms of childhood blindness - gain "life-changing improvements" to their sight, according to doctors at Moorfields Eye Hospital in London.
6-Year-Old Boy Who Was Born Blind Gets Vision Partially Restored After ‘Life-Changing’ Gene Therapy
Jace, from Connecticut, is one of four children all born with severe childhood blindness who gained 'life-changing improvements' to their vision after an experimental trial of gene therapy.
Blind Children See for the First Time Thanks to a Revolutionary Gene Therapy
A groundbreaking gene therapy has restored sight in four young children born with severe blindness due to a rare genetic deficiency. Scientists at UCL and Moorfields Eye Hospital successfully injected healthy copies of the defective gene into the retina,
3 Gene Therapy Stocks With Game-Changing Potential
Gene therapy stocks are transforming healthcare with revolutionary treatments. Therefore, as this sector continues to evolve, investing in gene therapy stocks like Pfizer (PFE), Sanofi (SNY), and Novartis AG (NVS) could be beneficial.
MeiraGTx gene therapy allows 11 blind children to see as biotech eyes accelerated approval
Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx's investigational gene therapy, data that is prompting the biotech to seek accelerated approval in AIPL1-associated severe retinal dystrophy.
'Life-changing' gene therapy for children born blind
Jace, 6, was born with a rare and aggressive eye condition An experimental trial of gene therapy has helped four toddlers - born with one of the most severe forms of childhood blindness - gain "life-changing improvements" to their sight,

Pfizer Discontinues Hemophilia Gene Therapy Beqvez

Once high-flying Bluebird Bio sells itself to private equity after tough times for the gene therapy maker
Bluebird Bio will sell itself to private equity firms Carlyle and SK Capital for about $30 million, the company said Friday.
What a $2 million per dose gene therapy reveals about drug pricing
Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...
Gene Therapy: Revolutionizing Medicine By Tackling Diseases At Their Genetic Roots
Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...
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Gene therapy breakthrough offers hope for severe Dravet syndrome cases
AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...
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Solid says early data suggest ‘differentiated’ Duchenne gene therapy
The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...
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Envision Summit 2025: Beyond the status quo in gene therapy
At the Envision Summit 2025 in San Juan, Puerto Rico, Mike Farkas, MD, discussed gene therapy research for retinal ...
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Medical Minute: Breakthrough treatment helps children with genetic disorder regain vision
A revolutionary gene therapy treatment in London has improved the vision of children suffering from a rare genetic disorder, ...
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Inhalable Cystic Fibrosis Gene Therapy 24 Years In The Making Enters Clinical Trials
A new inhalable gene therapy for cystic fibrosis (CF) has entered Phase I clinical trials, a major milestone on the journey ...