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Researchers design genetic therapy to prevent brain deterioration

Drug Discovery World

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. Doctors believed the calcium deposits were non-specific signs of brain damage, but this research challenges this view.

Therapies 147
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How to advance AAV-based gene therapies

Drug Discovery World

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

Therapies 162
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Genetic biomarkers could personalise therapy for TNBC

Drug Discovery World

The study, by researchers at The Institute of Cancer Research, London, shows that certain characteristics within patients’ primary tumours can predict how they will respond to different treatments after their tumours have spread around the body.

Therapies 130
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Sloane’s 1st ASO treatment

KIF1A

Fortunately, Sloane does not currently have epilepsy, and Megan hopes this treatment will prevent it’s onset as her disease progresses. On the day of the treatment, they checked into the hospital where Sloane was administered sedation medication. If all goes well, her treatments will continue on a quarterly basis thereafter.

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No sign of stopping: The rise of cell and gene therapy

Drug Discovery World

Ryan Leahy , a science communication expert in cell and gene therapy, at Phacilitate, says there is a lot to celebrate but also much to assess, question and challenge in this sector. There are questions regarding whether enough doctors are able to recommend advanced therapies, as well as where to send patients to receive treatment.

Therapies 130
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Immuno-oncology innovations redefining cancer treatment in 2024

Drug Target Review

What innovative approaches and technologies are anticipated to reshape immuno-oncology treatments in 2024? Immuno-oncology, notably through checkpoint inhibitors, has significantly reshaped cancer treatment over the past decade.

Treatment 121
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Combination therapy halts lung cancer tumour growth in mice

Drug Discovery World

A pathway to better therapies. The findings suggest a pathway to better therapies for one of the least treatable forms of cancer. The problem is that these tumours respond to treatment initially, but then they come back. One of the challenges we will face is convincing doctors to go back to an old drug.

Therapies 130