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The Latest Breaking News from Gene Therapy

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

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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

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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

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

Manchester Evening News

Gene therapy dramatically cuts risk of bleeding in haemophilia B patients

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

The Independent UK

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

Benzinga

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

Daily Mirror

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:

Benzinga

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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. …

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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.…

Benzinga

BridgeBio Pharma Posts Promising Data From Gene Therapy Trial In Ultra-Rare Disease

BridgeBio Pharma Inc (NASDAQ:BBIO) announced promising pharmacodynamic data from the first two participants dosed in CANaspire Phase 1/2 trial of BBP-812, gene therapy for Canavan disease. Canavan disease is an ultra-rare inherited disorder that damages the ability of nerve cells (neurons) in the brain to send and receive messages. Data from the first…

Benzinga

Mizuho Comments On UniQure's Upcoming Low-Dose Huntington's Gene Therapy Trial Data

The much-awaited one-year data for the entire low-dose cohort for UniQure NV's (NASDAQ: QURE) AMT-130 gene therapy in Huntington's disease is expected at any time.

Benzinga

FDA Sounds Positive On Bluebird's One Gene Therapy But Doubts Another

The FDA signaled in briefing documents released that it may approve Bluebird bio Inc's (NASDAQ:BLUE) beti-cel gene therapy to treat β-thalassemia for those who require regular blood transfusions. The Prescription Drug User Fee Act (PDUFA) goal dates for beti-cel and eli-cel are August 19 and September 16. Bluebird bio's blood disorder treatment demonstrates "clinically meaningful"…

Benzinga

SVB Leerink Sees Over 600% Upside For This Penny Biotech Stock

SVB Leerink initiated Selecta Biosciences Inc (NASDAQ:SELB) coverage with an Outperform rating and a price target of $7. The analysts view ImmTOR's approach to mitigate ADAs as de-risked by early clinical data from SEL-212 and believe that the ability to tame the immune response bodes well for ImmTOR's clinical potential in combination with…

Benzinga

Raymond James Says PTC Therapeutics' CHMP Backing Is 'Under Exceptional Circumstances' What's Next

Last week, PTC Therapeutics Inc's (NASDAQ:PTCT) Upstaza (PTC-AADC; AAV2-based DDC gene therapy) received a CHMP opinion for approval under exceptional circumstances, says Raymond James. It maintains Outperform rating on the stock. The CHMP recommended Upstaza for AADC deficiency (AADCD) patients aged 18 months and older with a severe phenotype. According to the analysts, with the…

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