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The Latest Breaking News from Gene Therapy

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

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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

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.

Investors Business Daily

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

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

The Street

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'

Wales Online

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.

Tribune News Service

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.

Benzinga

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?

Investors Business Daily

Gene therapy firm growing its Liverpool footprint with new build

A three-storey lab site was approved earlier today

Liverpool Echo

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

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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.

The Conversation

Helping cells become better protein factories could improve gene therapies and other treatments – a new technique shows how

The cells in your body are not all the same. Each of your organs has cells with very different functions. For example, liver cells are top-notch secretors, as their job requires them to make and export many of the proteins in your blood. By contrast, muscle cells are tasked with…

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Benzinga

Recap of Wednesday's Biotech Catalysts - End of The Day Summary

IDEAYA Biosciences (NASDAQ:IDYA) has entered into a clinical trial collaboration and supply agreement with Amgen Inc. to evaluate the efficacy and safety of IDE397 with Amgen's AMG 193 in a Phase 1 clinical trial. IDEAYA shares traded in a range of $14.59 to $15.82 on a day volume of 130.58…

Manchester Evening News

Mum thought itchy hands were caused by household cleaner - she was left with a deadly diagnosis costing £16.5K a month

Without treatment she now has just three to six months to live, doctors say

The Independent UK

Gene therapy dramatically cuts risk of bleeding in haemophilia B patients

Haemophilia is a rare condition that affects the blood’s ability to clot.

Benzinga

Recap of Wednesday's Biotech Catalysts - End of The Day Summary

Daily Mirror

Mum thought her hands were itchy from cleaning products before devastating diagnosis

Maria Barry, 58, assumed new cleaning products were to blame for the irritated skin on her hands, but she was later told the crushing news that she had a rare form of cancer

Benzinga

The Daily Biotech Pulse: CDC AdComm Backs Novavax COVID-19 Vaccine, European Approval For PTC Therapeutics' Gene Therapy, Merck's Keytruda Flunks In Head, Neck Cancer Study

Here's a roundup of top developments in the biotech space over the last 24 hours:

The Independent UK

Mother who thought itchy hands were caused by cleaning product given months to live

London nursery manager Maria Barry, 58, was horrified to discover truth behind chronic symptom

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