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

Pharmaceutical Technology

In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. David Bumcrot PhD, CSO, CAMP4 Therapeutics.

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US FDA clears Transcenta’s IND for Rett syndrome treatment

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted clearance for Neurogene’s investigational new drug (IND) application for NGN-401 to treat Rett syndrome. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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bluebird seeks gene therapy trial restart after cancer scare

pharmaphorum

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. Shares in bluebird ticked up following the announcement.

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Gene editing: beyond the hype

pharmaphorum

Since that discovery, a flurry of gene-editing focused biopharma companies have launched – including Intellia Therapeutics, CRISPR Therapeutics, Caribou Biosciences and Mammoth Biosciences – and the first drug therapies based on the technology are now in human testing for diseases like cancer. billion in funding.

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Angelman syndrome Pipeline: Unmet needs call for a robust pipeline

Delveinsight

Ovid Therapeutics is running Phase III clinical trials for its drug, Gaboxadol ( OV101 ). The drug is a small molecule, which selectively activates GABAA receptors located on the surface of nerve cells and away from the synapse. OV101 is considered as the first compound that targets disrupted tonic inhibition.