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The Latest Breaking News from Gene Therapy

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.

The Conversation

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….

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.

StockNews.com

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

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

Barchart

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

The Guardian - 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.

International Business Times 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

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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 Independent UK

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.

The Conversation

Children born deaf can hear in both ears in world-first gene therapy trial

Given as an infusion into the ear, the treatment works by replacing the faulty DNA which causes a type of inherited deafness known as DFNB9.

The Independent UK

Gene therapy trial gives deaf children hearing in both ears

Study participants born unable to hear could locate sound sources, recognise speech and dance to music after treatment

The Guardian - UK

Two deaf children can hear in both ears after world-first treatment

After the gene therapy, the children went from being profoundly deaf to having mild to moderate hearing loss

The Independent UK

Demystifying the maze: How genetic testing lights the path in India's fight against rare diseases

For countless families in India, rare diseases are a crippling burden. Affecting less than 1 in 50,000 individuals, these conditions often leave patients and doctors entangled in a frustrating diagnostic labyrinth. Traditional methods can lead to delays and misdiagnosis, further jeopardizing timely intervention. However, a beacon of hope emerges in…

The Times of India

Argentinian couple moves to US to allow their toddler to join gene therapy trial

The Lovatos relocated from Guatemala to Ohio for their son, who has the rare genetic disorder Canavan disease

The Guardian - US

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LiveScience

Deaf baby can hear after 'mind-blowing' gene therapy treatment

Seven months after her treatment, the baby girl can now respond to her parents' voices without the aid of a cochlear implant.

The Guardian - UK

Great Ormond Street hoping to license gene therapy for ‘bubble baby’ syndrome

Hospital to take unprecedented step after drug firm pulled out despite successful trial of treatment

The Guardian - UK

UK toddler has hearing restored in world first gene therapy trial

Opal Sandy can hear almost perfectly after groundbreaking surgery that took just 16 minutes

The Hindu

Pfizer reports patient death in Duchenne gene therapy study

Pfizer's gene therapy trial for Duchenne muscular dystrophy resulted in a young patient's death from cardiac arrest

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Barchart

3 Standout Penny Stocks to Watch This April

Investing in penny stocks carries substantial risk, but also offers the potential for significant returns. These penny stocks, all with bullish analyst ratings, have considerable upside potential, according to Wall Street.

Top News

Gene Therapy Breakthroughs In The 1990S Revolutionized Medical Treatment.

Explore the pivotal era of gene therapy in the 1990s, from groundbreaking advancements to significant challenges, shaping modern regenerative medicine.

Top News

Kyowa Kirin Gene Therapy: Most Expensive US Drug At .3M

Kyowa Kirin's .3 million gene therapy sparks debate on accessibility and pricing of innovative treatments in the US.

Top News

Kyowa Kirin's Gene Therapy: Most Expensive US Drug

Kyowa Kirin's .3 million gene therapy sparks debates on accessibility and affordability in healthcare.

Top News

Ensuring Quality Control In Cell And Gene Therapy Manufacturing

Learn about the importance of quality control in cell and gene therapy manufacturing for patient safety and market success.

The Hindu

German firm Miltenyi Biotec opens office, plans Cell and Gene Therapy CoE

Miltenyi Biotec launches operations in Hyderabad, investing in a Cell and Gene Therapy centre of excellence.

Top News

Exploring The Top 10 Genetic Engineering Breakthroughs

Explore the latest breakthroughs in genetic engineering, from CRISPR-Cas9 to gene therapy, and their impact on healthcare and conservation.

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