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The Latest Breaking News from Gene Therapy

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

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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

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

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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

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

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

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

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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

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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.

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 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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