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How to advance AAV-based gene therapies

Drug Discovery World

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

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Innovate UK grant to support development of powdered gene therapy

Drug Discovery World

The funding will support the development of the company’s gene therapy in a powder, a non-viral gene delivery system to improve tissue regeneration following surgery. The technology allows rapid transfection of patients’ cells with the gene(s) of interest.

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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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Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

The Pharma Data

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics. Financial details were not disclosed.

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Turning science into business: An optimised alternative to antibodies

Drug Discovery World

As the industry’s awareness of alternatives to traditional antibodies continues to grow, there is increasing demand for these alternatives, which we are seeing particularly within the therapeutic sector, to enable targeted therapeutics and enable the emerging gene therapy market. DS: The company now has clients across big pharma.

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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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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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