Remove DNA Remove Gene Therapy Remove Genetic Engineering Remove Genetics
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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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Researchers Halt Trial of Promising Sickle Cell Treatment

NY Times

Two patients in a gene therapy study developed cancer years after treatment. It is not clear whether the therapy was responsible.

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IBC vs. IRB: What’s the Difference?

Advarra

Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. What Does an IBC Review? How is IBC Membership Composed?

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Leading innovators in transcription factors for AAV for the pharmaceutical industry

Pharmaceutical Technology

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.

Gene 262
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Gene Switch: A Novel Platform for Switching Genes On and Off

Roots Analysis

Gene switches can be regulatory proteins or specific DNA sequences that act to either switch on or off the expression of a gene. Basic Components of Gene Switch Gene switches are composed of noncoding DNA sequences and transcription factors.

Gene 40
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mRNA Therapeutics and mRNA Vaccines Industry: Current Scenario and Future Trends

Roots Analysis

Messenger ribonucleic acid (mRNA) is a single-stranded molecule that is complementary to a gene’s DNA. It is important in the process of protein synthesis because mRNA is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein.

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Operation Warp Speed for Rare Diseases: Expected Boom in Drug Development and Approval

Advarra

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. From a technical standpoint, such diseases should be easier to treat with current gene therapy technology.