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The Latest Breaking News from Gene Therapy

Sarepta's Shocking Stand: Company Rejects FDA Request To Pull Gene Therapy Elevidys

The ground shook for Sarepta Therapeutics, Inc. (NASDAQ:SRPT) last week as the U.S. Food and Drug Administration (FDA), alarmed by a third patient death tied to its gene therapy platform, pulled a rare move: requesting a voluntary halt to Elevidys shipments. The company’s response? A resounding “no,” igniting a major…

Benzinga

Gene therapy maker Sarepta tells FDA it won't halt shipments despite patient deaths

Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene therapy

The Independent UK

Sarepta's Setback Deepens With Another Gene Therapy Fatality

Sarepta Therapeutics (NASDAQ:SRPT) shares plummeted on Friday following reports of a third patient death linked to its gene therapy pipeline this year, further clouding investor confidence despite the company’s recent efforts to provide clarity on its restructuring and FDA label plans.

Benzinga

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Sarepta Therapeutics Stock Plunges Over 30% In Friday Pre-Market: What's Going On

Shares of Sarepta Therapeutics (NASDAQ:SRPT) plunged over 30% in Friday pre-market after a clinical trial participant died following treatment with a gene therapy, marking the third death linked to the company’s experimental treatments.

Benzinga

Sarepta Therapeutics' Reorganization Boosts Investor Confidence

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) stock is trading higher on Thursday after the company issued an after-hours update on ongoing label expansion efforts for Elevidys, its approved gene therapy for Duchenne muscular dystrophy.

Benzinga

Breakthrough gene therapy jab reverses hearing loss in weeks

Researcher says this type of treatment for deafness is ‘just the beginning’

The Independent UK

Gene therapy restores hearing in toddlers and teenagers born with congenital deafness – new research

Up to three in every 1,000 newborns has hearing loss in one or both ears. While cochlear implants offer remarkable hope for these children, it requires invasive surgery. These implants also cannot fully replicate the nuance of natural hearing.

The Conversation

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Three-year-old becomes youngest patient to receive groundbreaking gene therapy

IN FULL

The Independent UK

Girl, 3, becomes youngest patient to receive groundbreaking treatment

It is the first NHS England commissioned gene therapy in the UK infused directly into the brain

The Independent UK

Mother’s delight as gene therapy offers hope to three-year-old

Gunreet Kaur has a rare inherited condition which can be fatal.

The Independent UK

Fractyl Health Stock Is Trading Lower Monday: What's Going On?

Fractyl Health, Inc. (NASDAQ:GUTS) shares are trading lower. The company announced new preclinical data from its Rejuva Smart GLP-1 pancreatic gene therapy platform.

Benzinga

Second patient death reported with gene therapy for muscular dystrophy

Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene therapy for muscular dystrophy

The Independent UK

Boy with ‘death sentence’ disease living a normal life after breakthrough gene therapy

Four-year-old Eisa can now play football and go to school – things his family feared he may never do

The Independent UK

London hospital gene therapy breakthrough gives boy with ‘death sentence’ disease a normal life

Eisa Hussein took part in a clinical trial testing the new gene therapy at Great Ormond Street Hospital

Evening Standard

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MarketBeat

Pharma Frenzy: Volatility Ignites Biotech Sector

Two biotech companies have surged on recent developments, illustrating biotech's volatility and prompting investors to evaluate potential long-term value.

The Independent UK

Breakthrough for baby with rare illness in world-first gene therapy trial

Fourteen-month-old Tomas was diagnosed with ornithine transcarbamylase (OTC) deficiency when he was a few weeks old

The Independent UK

Baby given new gene therapy in groundbreaking trial

It is hoped that the new therapy will work as well as a liver transplant for some patients.

StockNews.com

3 Gene Therapy Stocks With Game-Changing Potential

Gene therapy stocks are transforming healthcare with revolutionary treatments. Therefore, as this sector continues to evolve, investing in gene therapy stocks like Pfizer (PFE), Sanofi (SNY), and Novartis AG (NVS) could be beneficial. Read on….

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Evening Standard

Gene therapy experiment gives children 'life-changing' sight boost at London hospital

Pioneering gene therapy at Moorfields Eye Hospital has helped improve sight of four young children

The Independent UK

Medics preserve sight in children with rare genetic eye condition in world first

Specialists selected patients from overseas for the initial procedures, as the condition is so rare.

The Independent UK

Parents of boy with rare eye condition hail ‘amazing’ results of gene therapy

Jace was selected by specialists from Moorfields Eye Hospital and UCL Institute of Ophthalmology.

Salon

Behind $2 million per dose gene therapy

How did a drug rooted in government seed money and spurred by grassroots fundraising end up with such a price tag?

The Guardian - UK

This new sickle cell treatment could be a gamechanger – if the NHS really starts to care about black people’s health

Dating, holidays, work: this genetic disease changes everything. Gene therapy could be a lifeline, if it’s accessible to all, says Tobi Oredein, founder and CEO of Black Ballad

The Guardian - UK

‘Groundbreaking’ sickle cell disease treatment approved for NHS use in England

Clinical trials find one-time gene therapy exa-cel offers ‘functional cure’ in 96.6% of patients

MarketBeat

MaxCyte: Building the Future of Cell and Gene Therapy Innovation

MaxCyte's proprietary Flow Electroporation technology enables companies like Vertex and CRISPR Therapeutics to get FDA approved for their gene-editing therapy.

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