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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.

People on MSN · 22h

6-Year-Old Boy Who Was Born Blind Gets Vision Partially Restored After ‘Life-Changing’ Gene Therapy

A Connecticut boy is among four children all born with severe childhood blindness who gained “life-changing improvements” to their vision after an experimental trial of gene therapy.

The Mirror US on MSN · 16h

Blind 6-year-old Connecticut boy sees for the first time after groundbreaking gene therapy

Jace, 4, from Connecticut, was registered legally blind and only just able to distinguish between dark and light before undergoing treatment at Moorfields Eye Hospital in London

Newsweek on MSN · 1d

Blind Boy Can See After Groundbreaking Gene Therapy

A young boy, by the name of Harvey, is seen before and after he received gene supplement therapy for a rare inherited eye disorder that caused him to be blind. The after results show a miraculous development in his eyesight.

Pfizer Discontinues Hemophilia Gene Therapy Beqvez

FiercePharma · 1d

Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia B product Beqvez.

Reuters · 1d

Pfizer stops commercialization of hemophilia gene therapy Beqvez

Pfizer said on Thursday it will stop global development and commercialization of its hemophilia gene therapy, Beqvez, citing soft demand from patients and their doctors.

Business Insider · 1d

Pfizer halts work on hemophilia gene therapy, Nikkei reports

Pfizer (PFE) will end global development and commercialization of Beqvez, a gene therapy for hemophilia, Nikkei learned Thursday, after the

23hon MSN

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 ...

ophthalmologytimes6h

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 ...

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 ...

stocknews23h

3 Gene Therapy Stocks With Game-Changing Potential

Gene therapy stocks are transforming healthcare with revolutionary treatments. Therefore, as this sector continues to evolve, ...

IFLScience on MSN1d

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 ...

BioPharma Dive4d

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 ...

21h

Pharma giant's pullback from gene therapy puts spotlight on North Carolina operations

One of the largest pharmaceutical companies in North Carolina is discontinuing a treatment produced in the state.

News Medical on MSN4d

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, ...

FierceBiotech1d

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 ...

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Gene therapy, childhood blindness

Pfizer Discontinues Hemophilia Gene Therapy Beqvez

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