A new frontier in
medicine with robust cell & gene therapy delivery
At the forefront of personalized medicine, cell and gene
therapy are areas of biomedical research that aim to treat the underlying cause of both genetic and acquired
diseases.
Advancements in the field of human genomics, transcriptomics, and proteomics have given rise
to high-throughput, affordable, and potentially life-changing cell and gene therapies to treat individuals with
genetic diseases. Cell therapy works by introducing healthy cells into a patient's body to replace or repair
damaged cells, and gene therapy works by introducing genetic material to target cells via a vector to modify the
expression of a gene. Cell and gene therapies can be combined and typically offer longer-lasting effects than
traditional medicines. As our understanding of the human genome continues to improve, more sophisticated
gene-editing tools become available, promising a new era of personalized medicine.
In this resource, we
explore the different approaches available for safe and effective delivery of cell and gene therapies into target
cells.
Nucleic acids, DNA and RNA, are essential to all known forms of life,
playing a primary role in the storage and expression of genomic information.
Recent developments in human genomics, transcriptomics, and proteomics have led
to the advancement of nucleic acid-based therapeutics. With an increasing number of nucleic acid
therapeutics now available, gene-based therapies are promising a new era of genetic medicine.
There
are a number of different approaches to nucleic acid-based therapeutics, which can provide long-lasting and
effective effects via gene editing, insertion, replacement, or inhibition. These specific approaches require
the delivery of a nucleic acid sequence into target cells, and there are several delivery approaches
available, such as conjugation with lipid nanoparticles (LNPs) or encapsidating in viral vectors for
effective targeted delivery. The effectiveness of their translation, however, depends on the delivery tools
and technologies.
Discover the technology designed to reduce adsorption of oligonucleotides with
metal surfaces, improving quantification, sensitivity, accuracy, and robustness compared to standard
instrumentation.
Explore the free resources below to find out more:
Lipid-mediated delivery systems have been developed to facilitate the
transfer of large macromolecules such as nucleic acids across the plasma membrane.
LNPs provide a protected mode of transport for nucleic acids, as their size, lipid
composition, charge, and surface chemistry allow them to cross the plasma membrane and eliminate the risk of
nuclease degradation and endosomal digestion, enabling nucleic acids to enter the cell. The delivery approach
can be optimized for different types of nucleic acid and the selection of specific lipid characteristics
enables targeted delivery to certain cell types or tissues.
Each component that makes up the LNP
delivery system must be meticulously analyzed and controlled as it has been shown that impurities can lead to
mRNA degradation and lower potency. It is therefore imperative to implement impurities screening to ensure
drug safety and efficacy.
Download the free resources below to find out more about rapid LNP
analysis:
Another approach to delivering nucleic acid sequences into cells is by
encapsidating them in viral vectors.
Modified viruses are used as a vehicle to deliver nucleic acid across the nuclear
membrane and into cells due to their natural ability to infect cells and release their genetic payload. Once
the genetic information is released into the host cell, the gene product is expressed.
Viral vector
delivery systems offer advantages such as long-term gene expression, however, to ensure safety and efficacy,
careful monitoring of critical quality attributes across development, manufacturing, and QC is crucial.
Analytical methods such as anion exchange (AEX) chromatography, post-translational modification (PTM)
analyses, reversed-phase (RP) chromatography, and size exclusion chromatography (SEC) are just some of the
methods available to monitor the nucleic acid-based therapeutic.
Explore our free resources on the analysis of adeno-associated virus
(AAV) preparations:
Eliminating doubt in liquid
chromatography
Due to the complex nature of cell and gene therapies, managing residual
impurities in downstream processing is a challenging yet critical step to ensure the safety and efficacy of
these biotherapeutics.
From LC-optical workflows to support LNP development to pipetting instruments and
chromatography columns, Waters offers a comprehensive portfolio of compliance-ready LC-MS solutions to support
cell and gene therapy development. Find out more about Waters key products designed to eliminate doubt in your
LC-MS workflows:
Watch the video below to discover how Waters strives to provide you with the
confidence that your method is consistent and reproducible, batch-to-batch, year-to-year.
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