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The Latest Breaking News from Gene Therapy

Sarepta Crashes 37% After Its Gene Therapy Flops Its Biggest-Ever Test

Sarepta Therapeutics' gene therapy, Elevidys, faces an uphill regulatory journey, analysts said Tuesday on mixed test results.

Investors Business Daily

A new cure for sickle cell disease may be coming. Health advisers will review it next week

There soon may be a new cure for sickle cell disease that attacks the disorder at its genetic source

The Independent UK

Have Million-Dollar Gene Therapies Finally Reached An Inflection Point?

The big names behind biotech stocks no longer want to merely treat diseases, they want to cure them. But it's going to be costly.

Investors Business Daily

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Form of gene therapy offers hope for severe alcohol addiction, study finds

US research linked to dopamine release finds macaques decreased consumption by up to 90% after treatment

The Guardian - UK

Gene therapy injection into the brain causes alcohol use disorder to stop — in monkeys

A small, proof-of-concept study in monkeys reveals the potential of a one-off gene therapy to treat people with alcohol use disorder who haven't responded to other treatments.

LiveScience

Bengaluru-based eye hospital to soon start human clinical trials of indigenous gene therapy for ocular diseases

Narayana Nethralaya to start human clinical trials of indigenous gene therapy for ocular diseases; first such effort in India. Cost of therapy to reduce from ₹7 crore to 1/20th-1/50th of international costs. Meeting of 25 clinicians & scientists held to come out with consensus & establish national registry for rare…

The Hindu

Gene-therapy drops restore teen's vision after genetic disease left his eyes clouded with scars

A teen with dystrophic epidermolysis bullosa, or "butterfly disease," was legally blind but has now had his sight restored with gene therapy.

LiveScience

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Gene therapy eyedrops restored a boy's sight. Similar treatments could help millions

Antonio Vento Carvajal has been legally blind for much of his 14 years

The Independent UK

Harvard scientists have identified a drug combo that may reverse aging in just one week: ‘A step towards affordable whole-body rejuvenation'

The “chemical cocktail” helped reverse aging in mice, but some experts are skeptical about whether it will work in humans.

Fortune

Gene therapy for severe hemophilia is approved by FDA

U.S. health regulators have approved a gene therapy for the most common form of hemophilia

The Independent UK

Up First: Titan implosion; 1 year after Roe; Evan Gershkovich's appeal rejected

All five people aboard the Titan are believed dead after an implosion. Saturday marks a year since the Supreme Court overturned Roe. The FDA approves the first gene therapy for muscular dystrophy.

WEKU

First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids

The U.S. Food and Drug Administration has approved the first gene therapy to treat a deadly form of muscular dystrophy

The Independent UK

Sarepta Plummets Despite Winning FDA Nod For $3.2 Million Gene Therapy

Sarepta won approval Thursday for its highly anticipated Duchenne muscular dystrophy gene therapy. SRPT stock yo-yoed in response.

Investors Business Daily

Uniqure Crashes To Six-Year Low On Muddy Results For Huntington's Gene Therapy

Uniqure unveiled mixed results for its study of a Huntington's disease treatment on Wednesday, and QURE stock crashed to a six-year low.

Investors Business Daily

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The Hindu

Laurus Labs, IIT-Kanpur to collaborate for novel gene therapy

The drug manufacturer will also fund the clinical trials and launch the products in India and emerging markets. Additionally, it will establish a GMP (good manufacturing practice) facility at the Techno Park facility of IIT-K, Laurus said on Monday.

LiveScience

Gene therapy could be used as birth control for cats, small study suggests

A new study shows that gene therapy could be an effective form of birth control for cats.

Inverse

This Cutting-Edge Gene Therapy Could Revolutionize Birth Control — But It's Not for Humans

A new gene therapy that modifies the production of a hormone in cats could make spaying wild cats far easier.

Inverse

This Humble Virus Could Revolutionize How We Treat Genetic Diseases

A new type of gene therapy could be cheaper, safer, and more efficient at treating diseases like muscular dystrophy and sickle cell disease.

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Investors Business Daily

Sarepta Therapeutics Dives On An Unexpected Holdup For Its Gene Therapy

The FDA pushed back its review of a highly anticipated gene therapy from Sarepta Therapeutics on Wednesday, prompting SRPT stock to tumble.

LiveScience

New gene therapy gel is the 1st approved treatment for rare and painful 'butterfly disease'

The U.S. Food and Drug Administration approved a gene therapy for dystrophic epidermolysis bullosa, also known as butterfly disease.

Inverse

Gene Therapy is Upending How Doctors Treat Incurable Conditions

Gene therapy has helped doctors make major strides in treating inherited blindness, and it's now being explored for other conditions.

The Street

FDA Group Backs Gene Therapy to Fight Degenerative Disease

A promising gene therapy got welcome news from regulators.

The Street

Sarepta Therapeutics Stock Skyrockets On FDA Surprise

A promising gene therapy got welcome news from regulators.

Investors Business Daily

Sarepta Stock Catapults Higher After Eking Out A Narrow Win In Its Gene Therapy Battle

Shares of SRPT stock launched Monday after FDA advisors voted late Friday in support of a highly anticipated gene therapy from Sarepta.

The Conversation

Gene therapy helps combat some forms of blindness – and ongoing clinical trials are looking to extend these treatments to other diseases

An estimated 295 million people suffer from visual impairment globally. Around 43 million of those people are living with blindness. While not every form of blindness can be cured, recent scientific breakthroughs have uncovered new ways to treat some forms of inherited blindness through gene therapy.

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