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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
       192                                                                   Chemical Weekly  October 24, 2023       Chemical Weekly  October 24, 2023                                                               193

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