Category GENE THERAPY RARE DISEASES

Gene therapy appears more cost effective than current treatments for severe hemophilia B

St. Jude Children’s Research Hospital provides the first gene therapy manufacturing and distribution cost report for hemophilia B. A St. Jude Children’s Research Hospital analysis found a major gap between the cost to manufacture and distribute hemophilia B gene therapy and the $2 million-plus price reportedly under consideration for hemophilia gene therapy now in development. The study appeared

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Gene therapy offers potential cure to children born with immunodeficiency due to adenosine deaminase deficiency.

Study shows treatment developed by international team restored immune function in more than 95% of patients in three clinical trials An experimental form of gene therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited

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Gene therapy using “zinc fingers” may help treat Alzheimer’s disease, animal study shows

Key Takeaways A single injection of a gene therapy involving what are called zinc fingers dramatically reduced levels of tau, a protein implicated in Alzheimer’s disease, in mice with the condition. The treatment was long lasting, caused no side effects, and reduced Alzheimer’s-related damage in the brain. The technology worked just the way we had

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Retroviral gene therapy with a view on previous experience and future perspectives

Gene therapy can be used to restore cell function in monogenic disorders or to endow cells with new capabilities, such as improved killing of cancer cells, expression of suicide genes for controlled elimination of cell populations, or protection against chemotherapy or viral infection. While gene therapies were originally most often used to treat monogenic diseases

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Could Gene Therapy Cure Sickle Cell Disease? Two New Studies Raise Hopes

A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both therapies work by switching on a gene that promotes production of fetal hemoglobin, said Dr. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association

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Researchers of NEI use gene therapy and a novel light-sensing protein to restore vision in mice

NIH-funded therapy will now be tested in humans A newly developed light-sensing protein called the MCO1 opsin restores vision in blind mice when attached to retina bipolar cells using gene therapy. The National Eye Institute, part of the National Institutes of Health, provided a Small Business Innovation Research grant to Nanoscope, LLC for development of

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Clinical Development of Gene Therapies: The First Three Decades and Counting.

In the past three decades the field of gene therapy has made remarkable progress surging from mere laboratory experiments to FDA-approved products which bring significant reduction in disease burden to patients who previously had no therapeutic options for their serious conditions. In this paper it’s reviewed the evolution of the gene therapy clinical research landscape

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362 cell and gene therapies in US pipeline, finds report

New research has found that there are 362 cell and gene therapies in clinical pipelines in the US, an increase from 2018. A new report from America’s Biopharmaceutical Companies has revealed that there are 362 cell and gene therapies in development in the US. Roughly a third of the therapies, 132, are potential treatments for rare diseases.  The research

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Scientists at Oregon edit genes inside the body using CRISPR for a hereditary blindness disorder, in a trial called BRILLIANCE.

A person with a genetic condition that causes blindness has become the first to receive a CRISPR–Cas9 gene therapy administered directly into their body. The treatment is part of a landmark clinical trial to test the ability of CRISPR–Cas9 gene-editing techniques to remove mutations that cause a rare condition called Leber’s congenital amaurosis 10 (LCA10).

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