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Start-up aims to advance transformative genetic medicines

Drug Discovery World

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. The post Start-up aims to advance transformative genetic medicines appeared first on Drug Discovery World (DDW).

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The Power of Lipids: Enhancing Drug Delivery with Nanoparticles

Roots Analysis

LNPs comprise of a lipid bilayer that surrounds a hydrophobic core, which can be loaded with therapeutic agents ( such as drugs, genetic material and proteins ). The average size of LNPs typically ranges from 40 to 1,000 nanometers, which allows efficient cellular uptake and intracellular delivery.

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Global advances in synthetic biology

Drug Discovery World

Lu Rahman selects some recent synthetic biology innovations and the potential they hold to benefit drug discovery. . The rapidly growing area of synthetic biology – including molecular biology, biotechnology, biophysics, and genetic engineering – is having a marked impact on the drug discovery landscape.

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How to advance AAV-based gene therapies

Drug Discovery World

Such therapies have become an appealing drug modality for multiple conditions, including monogenic disorders, cancers, and infectious diseases. As such, they offer highly stable expression of human genes, mRNA, and proteins. Food & Drug Administration (2022) FDA Approves First Gene Therapy to Treat Adults with Hemophilia B.

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Considerations for Neuroscience Trials with Intrathecal Delivery and Other Methods of Direct CNS Administration

XTalks

One of the biggest challenges with developing therapies to treat CNS disorders is the delivery of systemically administered investigational products (IPs) to the brain, which is limited by the blood-brain barrier. Some methods of direct CNS administration include intrathecal, intraparenchymal and intracerebroventricular (ICV) delivery.

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

Roots Analysis

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. In the last few years, researchers have become interested in using in vitro transcribed (IVT) mRNA as a drug delivery agent.

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mRNA Synthesis: ManufacturingProcess of Modern Revolutionary Molecule

Roots Analysis

Messenger ribonucleic acid (mRNA) is a single-stranded molecule, which carries coding sequence and plays a prominent role in protein synthesis. It transfers genetic information form to DNA to ribosomes, a specialized structure, or organelle, which decodes genetic information into a protein.