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The Latest Breaking News from Gene Therapy

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

$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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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

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

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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

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

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…

Benzinga

Alzheimer's breakthrough: Scientists reverse disease with gene therapy

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

Wales Online

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…

The Conversation

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…

Benzinga

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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

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news in one place.

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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

Chicago Sun-Times

Funeral held for Highland Park parade shooting victim Kevin McCarthy, who died after shielding toddler son

McCarthy, 37, worked at a gene therapy startup and graduated from DePaul University; he died alongside his wife, Irina.

Benzinga

Sarepta Reports One Heart Inflammation Case In Duchenne Gene Therapy Trial

Sarepta Therapeutics Inc (NASDAQ:SRPT) shared new functional data across multiple studies from the SRP-9001 (delandistrogene moxeparvovec) program for Duchenne muscular dystrophy. SRP-9001 is an investigational gene therapy under development in partnership with Roche Holdings AG (OTC:RHHBY). Results from Cohort 1 demonstrated a 3.8-point improvement and 3.2-point improvement on the Duchenne functional scale 52 weeks after…

Benzinga

Why Precigen Shares Are Gaining Today

Precigen Inc (NASDAQ:PGEN) has agreed to sell its subsidiary Trans Ova Genetics, an animal reproductive technologies company, to URUS, a holding company with cooperative and private ownership. The deal consideration includes $170 million in upfront cash and up to $10 million earn-out based on the performance of Trans Ova in 2022 and 2023. …

Benzinga

Oyster Point Outlines Restructuring Plan With Savings Of Up To $48M In 2023

Oyster Point Pharma Inc (NASDAQ:OYST) will implement an operating expense streamlining plan to reduce employee and non-employee expenses (up to 50 roles) across the organization. The company plans to continue to drive the launch of Tyrvaya with approximately 150-200 field-based sales resources. The plan is expected to reduce operating expenses by approximately $6…

Benzinga

UniQure Shares Are Up After Low-Dose Cohort Data From Huntington's Gene Therapy Trial

UniQure N.V. (NASDAQ:QURE) announced safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing Phase 1/2 trial of AMT-130 gene therapy for Huntington's disease. Six of the ten enrolled patients received AMT-130, and four received an imitation surgical procedure in this randomized, blinded clinical trial in the U.S.…

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