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The Latest Breaking News from Gene Therapy

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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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.

MarketBeat

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.

The Independent UK

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….

StockNews.com

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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

Evening Standard

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.

The Independent UK

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?

Salon

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

The Guardian - UK

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.

MarketBeat

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Benzinga

A Glimpse Into The Expert Outlook On Voyager Therapeutics Through 5 Analysts

Across the recent three months, 5 analysts have shared their insights on Voyager Therapeutics (NASDAQ:VYGR), expressing a variety of opinions spanning from bullish to bearish.

MarketBeat

3 Small-Cap Stocks That Are Ready to Rocket Higher

With lower interest rates and lower corporate taxes more likely, it's a good time to consider three small-cap stocks that present a buying opportunity

The Conversation

RNA editing is the next frontier in gene therapy – here’s what you need to know

The United States Food and Drug Administration has just approved the first-ever clinical trial that uses CRISPR-Cas13 RNA editing. Its aim is to treat an eye disease called wet age-related macular degeneration that causes vision loss in millions of older people worldwide.

StockNews.com

3 Biotech Stocks Predicted to Surge on Medical Innovation

Biotech stocks are surging as groundbreaking neuroscience and gene therapy innovations take center stage. Amid this backdrop, investors might consider adding fundamentally strong biotech stocks like Regeneron Pharmaceuticals (REGN), Biogen (BIIB), and Gilead Sciences (GILD) to their portfolio for an impactful investment. Continue reading….

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news in one place.

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StockNews.com

3 Pharma Stocks to Watch as Gene Therapy Gains Momentum

The pharma market shows promise as gene therapy gains momentum through rapid advancements that address complex diseases and improve treatment options. Given this favorable trend, it could be wise to watch strong pharma stocks such as Vertex Pharmaceuticals (VRTX), Wave Life Sciences (WVE), and Poseida Therapeutics (PSTX). Read more.

Barchart

2 'Strong Buy'-Rated Stocks That Could Rally 39% to 48% in 2025

Both of these growth stocks are positioned for long-term success.

The Guardian - UK

NHS to offer ‘life-changing’ gene therapy for blood disorder thalassaemia

Treatment may be offered to hundreds with severe form of disease, most often found in people of Mediterranean, Asian and Middle Eastern heritage

International Business Times UK

Goldman Sachs Expects Market Sentiment To Support Smaller Firms, Sees Upside of up to 165% for Two Biotech Stocks

Goldman Sachs analysts believe small- and equal-weight benchmarks will keep outperforming unless Q2 reports of big tech companies lead analysts to bump their sales forecasts for the latter half of this year and 2025.

The Independent UK

Takeaways from AP's report on access to gene therapies for rare diseases

The promise of gene therapy looms large for families dealing with rare, genetic disorders

The Independent UK

Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate

The promise of gene therapy looms large for families dealing with rare, genetic disorders

The Conversation

When there’s no commercial incentive to develop gene therapy – hospitals will try to fill the gap

Individually, rare diseases are rare, but there are so many rare diseases that over 400 million people are affected worldwide. Together, rare diseases aren’t so rare.

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