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The Latest Breaking News from Gene Therapy

New gene therapy 'significantly reduces seizures' for children with epilepsy

UCL expect to begin human trials of therapy within five years

Evening Standard

Promising new gene therapies for sickle cell are out of reach in countries where they're needed most

New gene therapies promise a cure for sickle cell disease but experts say they are out of reach in places that need them the most

The Independent UK

Here's What The FDA Approval Of Gene Editing For Sickle Cell Means For CRSP Stock And The Future Of Medicine

Biotech stocks could be at a major turning point after FDA approval of the first gene editing drug using Crispr technology. But gene editing therapies aren’t without risk, and questions remain about the safety and cost effectiveness of these experimental treatments.

Investors Business Daily

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Game-changer: On approval for gene therapy to treat sickle cell disease and beta thalassemia

Landmark decisions that mark the beginning of gene therapy using the CRISPR-Cas9 tool to treat diseases that could otherwise be cured only through bone marrow transplantation

The Hindu

The First CRISPR Gene Therapy Was Finally Approved In The US — Here’s How It Works

Using the gene editing tool CRISPR, the new therapy promises a cure for those with sickle cell anemia and beta thalassemia, but cost could be a barrier to care.

Inverse

UK becomes 1st country to approve gene therapy treatment for sickle cell, thalassemia

Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the U.K. In a statement on Thursday, the Medicines and Healthcare Regulatory Agency said it had approved Casgevy, the…

The Independent UK

Blood disorder breakthrough as world-first gene therapy given green light

The treatment is the first to be licensed using the gene-editing tool known as Crispr, for which its inventors were awarded the Nobel prize in 2020

The Independent UK

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Casgevy: World-first gene therapy for sickle cell disease approved in UK

Regulator's approval comes three years after scientists won Nobel Prize for inventing the treatment

Evening Standard

World-first gene therapy for inherited blood disorders approved in UK

The treatment is the first to be licensed using the gene-editing tool known as Crispr, for which its inventors were awarded the Nobel prize in 2020.

The Independent UK

Goodbye Statins? New Gene Editing Therapy Slashes High Cholesterol

A new gene therapy targeting an enzyme involved in familial hypercholesterolemia slashes bad cholesterol in a small clinical trial.

Inverse

Some children born deaf in China finally able to hear after gene therapy, report says

‘It’s really important that we get the first gene therapy treatment right’

The Independent UK

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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The Guardian - UK

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

LiveScience

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.

The Hindu

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…

LiveScience

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.

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The Independent UK

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

Fortune

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.

The Independent UK

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

WEKU

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.

The Independent UK

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

Investors Business Daily

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.

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