Page 194 - CW E-Magazine (24-10-2023)

Special Report Special Report

but the kinetics of plasmid delivery are virus-like particles(2). With viral vector is determining how to package enough pharmaceutical formulations. The com- lopment arm supporting clinical pro- 4. Pharmaceutics. 19: 1669–1686 2022.
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slower than those for mRNA. delivery, the CRISPR/Cas9-encoding material into the LNPs. Research is pany is focused on empowering biologics grams. 5. Kenjo, Eriya et al. “Low immuno-
sequences are integrated into the viral therefore focused on developing LNP drug delivery through its Adjuvant genicity of LNP allows repeated
Regardless of the approach, deli- genome, resulting in release of the RNP delivery vehicles that can carry larger Systems and Small Molecule, Protein, In addition to supplying materi- administrations of CRISPR-Cas9
ChemImpex
IndiaChemTrade
very is challenging, owing to the large complex into the targeted cells(1). There payloads. Increasing LNP delivery and Nucleic Acid Delivery platforms. als currently in use for the delivery mRNA into skeletal muscle in
size of the Cas9 protein and the over- are safety concerns (e.g., mutations, effi ciency and precision are other primary With a wide range of solutions for both of CRISPR/Cas9 gene-editing com- mice.” Nature Communications.
For subscribers only
Free for subscribers of Chemical Weekly
all CRISPR/Cas9 system, as the com- carcinogenesis, immune response) and goals. With gene-editing, it is necessary the human and animal health markets, ponents, Croda is pursuing – in coope- 12: 7101 (2021).
IndiaChemTrade provides consolidated trade statistics
ChemImpex, the trade database, tracks, on a daily
ponents must reach the cell nucleus. packaging limitations with certain viral to deliver the components to a large the pharmaceutical portfolio is unsur- ration with its customers – efforts to
of India. The information provided here is based on the
basis, exports and imports of chemicals and related
Stability is also an issue. The low vectors. Exosomes (membrane-bound number of cells; high delivery effi ciency passed in its excellence. Croda Pharma’s develop innovative lipid products that ABOUT THE AUTHOR
products through the major ports in India — a vital
annual trade data published by the Directorate General
intracellular delivery effi ciency must be vesicles) are another natural alternative is therefore necessary to enable cost- products, along with its in-house for- can address some of the issues that have Stephen Burgess, Ph.D., serves as
of Commercial Intelligence and Statistics, Government
source of commercial intelligence. Find out on-line
overcome to achieve effective gene- for CRISPR/Cas9 delivery that are effective doses. Preventing off-target mulation and regulatory expertise, al- arisen with LNPs for mRNA vaccines Managing Director of Avanti Polar Lip-
how international markets are moving; where your
of India. The database can be searched by: Chemical
editing therapeutics(1). There are also being explored because of their biocom- editing is equally crucial. low the company to meet its customers’ around PEG lipids and other compo- ids, and he is Head of Lipid Technology
and Nucleic Acid Delivery for Croda
competitors are exporting and at what price; where
concerns about the ability to safely and patibility and low immunogenicity. most demanding formulation needs. nents. Name/Harmonised Code to obtain the consolidated
effectively deliver repeated treatments, LNPs with lipid-coated polymer cores The company is committed to enabling trade (import or export) in that item; or by Country to
Pharma, supporting the group’s vision to
imports are coming from and how their prices are
become the global leader in empowering
which for many large tissues and Delivery of CRISPR gene-editing Hybrid LNPs present a potential the next generation of drug delivery REFERENCES obtain the trade (import or export) in all chemicals and
moving. Download all this data, seamlessly, for a
organs will be necessary using current tools can also be achieved with chemi- solution. They have solid polymeric cores systems. 1. Duan, Li et al. “Nanoparticle Deli- biologics delivery. Over the past 30 years,
related products between India and that country
small fee — online, anytime, with no hassles.
delivery technologies(2). The need for cal methods, including liposomes, that sequester genetic material and an very of CRISPR/Cas9 for Genome he has worked with numerous researchers
repeat administration has the potential lipid nanoparticles (LNPs), polymeric external lipid coating comprising lipid Avanti Polar Lipids, now part of ChemXchange to develop novel lipids and lipid formu-
Editing.” Front. Genet. 12 May 2021.
to result in immunogenicity on the one nanoparticles, gold nanoparticles, and components that are biocompatible and Croda Pharma, has been supplying 2. Taha, Eman A., Joseph Lee, and lations to address specifi c experimental
Open to all registered users — no subscription required
hand and the development of immunity other nanoscale solutions(1,2). LNPs are make it easier to cross the lipid mem- lipids for therapeutic applications, in- Akitsu Hotta. “Delivery of CRISPR- challenges and improve drug delivery. He
ChemXchange — the chemical exchange with a difference — is where you can identify your buyers and sup-
on the other. attractive because they have low branes of cells. They are highly effi - cluding lipids used for gene therapies, Cas tools for in vivo genome editing has played a key role in a diverse list of
plyers on-line, any time. This unique platform is free to all advertisers in the print version of Chemical Weekly.
immunogenicity and provide greater cient at sequestering and protecting the for more than 30 years. Researchers therapy: Trends and challenges.” projects, including drug delivery develop-
Why LNPs are attractive delivery fl exibility with respect to the cargos for genetic material and enabling its inter- investigating both viral vector and Journal of Controlled Release. 342: ment, gene therapy, wound healing, and
For more information contact: 022-24044477. Fax: 022-24044450
vehicles which they can facilitate delivery and cellular uptake. Even the non-natural nonviral delivery systems have been 345–361 (2022). vaccine development. His background in
Email: corporate@chemicalweekly.com or log on to chemicalweekly.com
Several different approaches have the organs or cells that can be targe- ionizable lipids, depending on how reliably provided with high-quality, 3. Ross, Colin J.D. et al. “Lipid- lipid synthesis, lipid biophysical proper-
been employed for the delivery of ted(1,3). They can also be formulated with they are manufactured, are biocompati- well-characterized materials. Croda Nanoparticle-Based Delivery of ties, and membrane dynamics has been
CRISPR gene-editing tools, and they biodegradable components as lipid-like ble and metabolizable and thus do not has added the ability to supply lipids CRISPR/Cas9 Genome-Editing instrumental in resolving problems asso-
can generally be classifi ed as physical, nanoparticles (LLNs) with reduced lipo- present toxicity issues. In addition, the at commercial scale to Avanti’s deve- Components.” Mol. ciated with these projects.
biological, or chemical(2). some biological toxicity. polymeric core means that less lipid
material is needed, which further reduces
Physical delivery methods include In one recent example, a CRISPR- the toxicity. Furthermore, it is possible
electroporation, microinjection, and Cas9/sgRNA system developed as a to tether a targeting agent – a peptide,
sonoporation. Electroporation can be gene-editing therapy to treat Duchenne protein, or antibody – to the lipid to en-
effective at delivering Cas9 RNP into muscular dystrophy (DMD) was able the targeting of these therapies to
hard-to-transfect cells, including in- achieved using a chemically defi ned specifi c areas. The key issue, then, is
duced pluripotent stem cells(2). It is inef- LNP solution that enabled safe and designing the polymeric core so that Missed a copy !!!
fi cient for in vivo delivery, however, so repeated injection of the therapy into it can sequester the large gene-editing
most uses have been in ex vivo applica- skeletal muscle tissue in a mouse model(4). components while maintaining the For Digital Edition of this month’s issue & all other past issues
tions. Microinjection involves injection Overall, LNPs increase the effi ciency desired delivery effi ciencies. Advances in
of the CRISPR components into cells of in vivo CRISPR/Cas9 component the development of polymeric cores for
under a microscope and thus has limi- delivery and reduce toxicity compared hybrid LNPs will enable the effi cient, Visit www.hpicindia.com
ted throughput. In addition, while there with viral vector technology while targeted delivery of greater quantities
is no cargo size limitation, it does maintaining the necessary precision to of genetic material with reduced toxi- Mrs. Usha S. - usha@hpicindia.com
require specialized equipment and exper- achieve therapeutic effects. city, making polymeric core design a PDF copies available for download
tise and is largely focused on the zygote key area of research and development
stage, which is not appropriate for Packaging, effi ciency, and precision in LNP production. Register Now
human therapeutics. Sonoporation com- challenges
bines ultrasound with injection and has While LNPs are attractive as delivery A long-term lipid innovations partner
been investigated in a few development vehicles for CRISPR/Cas9 gene-editing Croda Pharma is a leading partner Contact: For Subscription : Mrs. Usha S. - usha@hpicindia.com
products. Biological delivery largely components, there are challenges that for the development of excipients and For Advertising : Mr. Vijay Raghavan - vijay@hpicindia.com
involves the use of viral vectors and must be overcome. One difficulty the supply of high-purity materials for
194 Chemical Weekly October 24, 2023 Chemical Weekly October 24, 2023 195
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