Gene therapy, Born Blind

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,
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
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 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 ...
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 ...
stocknews1d
3 Gene Therapy Stocks With Game-Changing Potential
Gene therapy stocks are transforming healthcare with revolutionary treatments. Therefore, as this sector continues to evolve, ...
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 ...
FiercePharma1d
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. | ...
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 ...
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.
ophthalmologytimes9h
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 ...
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 ...
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.