Remove Gene Remove Gene Expression Remove Gene Therapy
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Gene Therapy and Pharmacokinetics

Camargo

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Astellas licenses companion drug for Pompe gene therapy

pharmaphorum

Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe disease. The post Astellas licenses companion drug for Pompe gene therapy appeared first on.

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Researchers speed identification of DNA regions that regulate gene expression

Scienmag

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Credit: St. Jude Children’s Research Hospital St.

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Selecta Biosciences files patent for enhanced gene therapy using viral vectors and immunosuppressants

Pharmaceutical Technology

Enhance gene expression and reduce immune responses with Selecta Biosciences' patented method using viral vectors and nanocarriers attached to immunosuppressants. Learn more about this innovative approach for improved therapeutic outcomes.

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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. This treatment activates the OTC gene so that ammonia can be removed from the blood.

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US FDA clears Transcenta’s IND for Rett syndrome treatment

Pharmaceutical Technology

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

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Gene editing: beyond the hype

pharmaphorum

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. The exact mechanism depends on the disease in question.