Page 193 - CW E-Magazine (24-10-2023)

For your requirement of Special Report
Acetone
Benzene
Toluene Lipid technology for delivery of gene-editing therapies
Chloroform
Soya Lecithin ene-editing technology is mov- STEPHEN BURGESS, Ph.D.
ing from the research lab to the
I.P.A. Gclinic – one of the most eagerly Croda Pharma
Methyl Salicylate I.P. anticipated biopharma developments Further improvements are anticipated
Ortho & Mix Xylene in recent history. One of the biggest as candidates move through preclini-
hurdles to the development of success- cal and clinical development and more
Contact: ful gene-editing therapeutics is effec- knowledge is gained.
Pure Solvi-Chem India tive delivery of the large and sensitive

297-B, Old Post Offi ce Lane, Mangaldas Market, components needed to execute the editing A number of companies that have
Bldg. No. 3-D Mangaldas Road, Mumbai 400 002.
Tel.: 022-22019080 / 22014846 * Fax: 022-22014846 functions. Lipid-based nanoparticles been exploring this approach are cur-
email: info@puresolvichem.com present promising solutions to these phenotype by introducing the gene for rently seeing rapid acceleration of
Website: www.puresolvichem.com
challenges. a functional protein. Today, however, their programs. For instance, Verve
many therapeutic candidates based Therapeutics and Intellia Therapeutics
Manufacturers of: The potential of therapeutic CRISPR on gene-editing technologies are now have begun clinical trials. Many new
Saccharin Insoluble
Sodium Saccharin gene-editing advancing through the clinic. companies are entering the space as
Sodium Saccharin Powder 6% Gene-editing technologies are well, quite a number of them focused
Ortho Toluene Sulphonamide used to manipulate DNA and RNA The developers of the fi rst version on in vivo therapies. As gene-editing
Ortho Para Toluene Sulphonamide sequences by cutting, inserting, or replac- of CRISPR/Cas9 gene-editing tech- systems are optimized and become
Para Toluene Sulphonyl Chloride ing genetic sequences in the genome. nology received the 2020 Nobel Prize more precise and effi cient, the ability
Para Toluene Sulphonamide Three primary technologies have been in Chemistry for their work. A host of to address new and different diseases
developed to date: zinc fi nger nuclease advances have been made since the will become possible, and there will
®
(ZNF); transcription activator-like original CRISPR editing tools were be continued movement toward in vivo
effector nuclease (TALEN); and clus- introduced – primarily via genetic therapeutics.
SWATI PETRO PRODUCTS PRIVATE LIMITED tered regularly interspaced short palin- engineering – with some of the current
(Regd. Off.) Plot No B-18, Chemical Zone, MIDC,
& Unit 1: Ambernath (W) - 421501 dromic repeat (CRISPR)(1). Initially, candidates leveraging second-gene- Two CRISPR-Cas9 components to
Tel: +91-251-2682581, 2684285, 7276422244, 8446053566 this technology was primarily used ration technology and others relying deliver
Unit 2: Plot No. 15,16&21/9, Morivali, MIDC,
Ambernath (W) 421501 for research purposes to identify genes on third-generation approaches that Unlike more traditional gene thera-
Tel: +91-251-2684286, 8446853565 involved in disease pathways – either have addressed some of the earlier pies that involve delivery of a single
Unit 3: Plot No. 15,16&21/14, Morivali, MIDC,
Ambernath (W) 421501  Tel: +91-251-2684287 assessing the consequences of a gene issues with the precision and persistence gene of interest, CRISPR/CRISPR-
Email: marketing@swatipetro.in, swati.saccharin@gmail.com knockout/knockdown or reversing a required for therapeutic applications. associated system (Cas9) gene-editing
Website: www.swatipetro.in
requires co-delivery of the Cas9 endo-
nuclease protein and the single guide
Industrial Land and shed RNA (sgRNA) that targets the editor
Available on Rent / Sell / protein to a specifi c genomic location.
Suitable Business Proposal
The Cas9 protein and sgRNA can
1. Location - Kurkumbh MIDC be delivered separately or complexed
2. Area - 12,000 square meter together as a ribonucleoprotein (RNP),
3. Built up Area - 2790 square which is the active species that per-
meter forms the genome editing within the
4. MIDC approval for chemical cell(1). Alternatively, plasmid DNA can
activity be delivered to the cell, where it is tran-
5. Well connected to Solapur scribed into messenger RNA (mRNA)
highway and then translated into Cas9 protein(2).
Another option involves the use of
Suitable for Factory, Workshop or Godown Cas9 mRNA, which must be translated
Please Contact: into Cas9 protein once it is inside the
Email: directors@halideschemicals.com cell. DNA is more stable than mRNA,
Mobile: 9921179184, 9881397116
19/13
Chemical Weekly October 24, 2023 193

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