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The Latest Breaking News from Gene Therapy

Toddler becomes first child in UK to receive revolutionary gene therapy

Teddi Shaw suffers from deadly metachromatic leukodystrophy (MLD) but has been given the chance of a normal life.

The Independent UK

Girl with deadly inherited condition is cured with gene therapy on NHS

Teddi Shaw, from Northumberland, first recipient on health service of Libmeldy, world’s most expensive drug

The Guardian - UK

Promising gene therapy delivers treatment directly to brain

Scientists say delivering gene therapy directly to the brain holds great promise

The Independent UK

From analysis to the latest developments in health,
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NOT REAL NEWS: A look at what didn't happen this week

Social media users shared a range of false claims this week

The Independent UK

Health: Finds New Infant Gene Therapy To Treat Artemis-SCID Patients

A novel gene therapy developed at UC San Francisco is on track to improve the health of ten young children who were born without functioning immune systems and without the capacity to fight infections. Read on for details:

Dynamite News

Sickle cell cure will be cost-effective if health disparities considered -study finds

(This Dec.13 story has been corrected to remove reference to Sanofi in the fifth paragraph)

Reuters

Revolutionary gene therapy offers hope for untreatable cancers

UK teenager is first person in the world to receive ‘base-edited’ cells that could transform cancer medicine

The Guardian - UK

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World’s most expensive drug approved to treat hemophilia at $3.5 million a dose

U.S. regulators approved CSL Behring’s hemophilia B gene therapy, a one-off infusion that frees patients from regular treatments but costs $3.5 million a dose, making it the most expensive medicine in the world.

Tribune News Service

Gene therapy at $3.5m a dose approved for US adults with hemophilia B

Hemgenix frees patients from repeated treatments but initial cost makes it most expensive medicine in the world

The Guardian - US

The FDA Just Approved Its Most Expensive Drug Ever — Sending UniQure Stock On A Warpath

The FDA just approved its most expensive drug ever — a hemophilia gene therapy from CSL and UniQure — and QURE stock popped Wednesday.

Investors Business Daily

$3.5M gene therapy for hemophilia gets FDA approval

U.S. health regulators have approved the first gene therapy for hemophilia, a blood-clotting disorder with few treatment options

The Independent UK

Krystal Biotech Needs to Feed the Goldfish in 2023

Development delays across the pipeline risks losing interest on Wall Street, which has the attention span of a goldfish.

The Street

Epilepsy: gene therapy technique targeting overactive brain cells shows promise in treating drug-resistant form of the condition

Something like 50 million people worldwide have epilepsy. While the majority of these people are able to use medications to manage and prevent their seizures, around one-third don’t respond well to these treatments. In such cases, the only option available to bring seizures under control is to remove the part…

The Conversation

Death in CRISPR gene therapy study sparks search for answers

The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now trying to figure out what killed him

The Independent UK

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Investors Business Daily

Why IBD Stock Of The Day Sarepta Just Snagged A Price Target Hike Over Pfizer

IBD Stock Of The Day Sarepta will lead the gene therapy market in treating a form of muscular dystrophy, an analyst said Monday.

The Street

Bluebird Bio's 2nd Approval Doesn't Guarantee Survival

Investors hoping for a revival at the struggling biotech might want to temper their expectations.

Wales Online

Four brothers with rare disease who had bone marrow transplants lead call for more research

There is 'promising new gene therapy treatment on the horizon' that could see 'safer and more effective ways to treat patients with XLP'

Tribune News Service

Bluebird’s gene therapy for brain disease receives accelerated approval

Bluebird Bio Inc.’s gene therapy for a brain-wasting disease received accelerated approval from U.S. regulators, making it the first treatment of its kind for children living with cerebral adrenoleukodystrophy.

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Benzinga

FDA Okays bluebird bio's $3M Gene Therapy For Rare Neurological Disorder In Boys: What Investors Should Know

Biotechnology company, bluebird bio, Inc. (NASDAQ: BLUE) announced late Friday that the Food and Drug Administration approved its gene therapy for a rare neurodegenerative disease.

Investors Business Daily

Something Unusual Is Happening Right Now With Biotech Stocks

Biotech stocks have swung sharply higher, buoyed by several factors. Could they be poised to lead the market though the next big upturn?

Liverpool Echo

Gene therapy firm growing its Liverpool footprint with new build

A three-storey lab site was approved earlier today

Benzinga

Recap Of Tuesday's Biotech Catalysts - End Of the Day Summary

Amgen (NASDAQ:AMGN) announced top-line results from the global Phase 3 CodeBreaK 200 trial evaluating once daily oral LUMAKRAS (sotorasib) in patients with KRAS G12C-mutated non-small cell lung cancer (NSCLC), the study met primary endpoint. Amgen shares traded in a range of $238.29 to $240.31 on day volume of 2.43 million shares, closed regular…

Benzinga

Recap Of Wednesday's Biotech Catalysts - End Of the Day Summary

The European Commission (EC) has granted conditional marketing authorization (CMA) to BioMarin Pharmaceutical’s (NASDAQ:BMRN) lead asset ROCTAVIAN (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients. BioMarin shares traded as high as 2.28 percent, in a range of $91.39 to $93.68…

Wales Online

Alzheimer's breakthrough: Scientists reverse disease with gene therapy

In the study, stem cell survival was enhanced by deleting a gene called Bax

The Street

Did Sarepta Therapeutics Just Unveil a Breakthrough?

The leading gene therapy developer just bolstered its technology platform.

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