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Risk Assessment for use of Engineered Genetic Materials in Clinical Research

Advarra

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

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Ginkgo acquires machine learning biotech Patch Biosciences

Drug Discovery World

Ginkgo Bioworks has acquired Patch Biosciences, with the intention to strengthen its gene therapy services, cell therapy services, and RNA therapeutics services. The collaboration achieved its goals of enhancing the AAV production titres of Biogen’s gene therapy manufacturing processes.

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Scientists engineer safe, virus-resistant E coli for research

Drug Discovery World

In a step forward for genetic engineering and synthetic biology, US researchers have modified E coli bacteria to be immune to infection by all natural viruses tested so far. The team used two safeguard methods to prevent the bacteria and their modified genes from escaping into the wild.

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Spotlighting Lupus Awareness Month: CAR-T Technology Creates New Avenues for Treatment of a Devastating Disease

WCG Clinical

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. WCG has many ways to support cell and gene therapy clinical trials for lupus.

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Gene Switch: A Novel Platform for Switching Genes On and Off

Roots Analysis

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

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Clinical Catch-Up: January 4-8 | BioSpace

The Pharma Data

FBX-101 is a first-in-human AAV gene therapy. Krabbe disease is a rare and fatal pediatric leukodystrophy caused by mutations in the galactosylceramidase (GALC) gene. The drug is an RNA interference (RNAi) therapeutic. ENPP1 deficiency is a rare, chronic, systemic, and progressive disease. Most Read Today. Source link.

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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

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