Gene editing technologies allow the researchers to change the genetic material of an organism.
This change can be due to any addition, deletion or alteration at specific location in the genome.
Basically, there are three ways in which genes can be manipulated, namely:[1]Gene Insertion:Â This involves the addition of new attributes to a gene through the incorporation of nucleotide sequences.Gene Repair:Â This refers to the replacement of a defective gene sequence by a functional sequence.Gene Inactivation:Â This involves the use of specific nucleotide sequences or regulatory elements to prevent the expression of a target gene.
Table 10.1 provides information on the companies that are using / have developed proprietary gene editing technology platforms that can be applied for the production of gene therapies.
Table 10.1 Gene Editing Technology PlatformsS.
No.Company NameHQTechnologyNumber of Molecules in Pipeline[2]Highest Phase of Development1Beam TherapeuticsUSCRISPR[3]1Undisclosed2Bioverativ, Sangamo TherapeuticsUSZFN Technology[4]1I/II3bluebird bioUSHoming endonuclease and megaTAL[5]1Preclinical4Caribou BiosciencesUSCRISPR / Cas[6]NANA5CellectisFranceTALEN[7], [8]NANA6CRISPR TherapeuticsUSCRISPR / Cas9[9]5I/II7EdiGeneChinaCRISPR[10]4I/II (Planned)8Editas MedicineUSCRISPR / Cas9, TALEN[11], [12]6I/II9Exonics TherapeuticsUSCRISPR / Cas9[13]1Preclinical10Homology MedicinesUSProprietary Technology[14]4Preclinical11Horizon Discovery GroupEnglandProprietary Technology[15]NANA12Intellia TherapeuticsUSCRISPR / Cas9[16]5Preclinical13LogicBio TherapeuticsUSGeneRide[17], [18]4Preclinical14NovartisSwitzerlandProprietary AAV-mediated editing by Directed Homologous Recombination[19]NANA15Poseida TherapeuticsUSCas-CLOVER[20]NANA16Precision BioSciencesUSARCUS[21], [22]NANA17Sangamo TherapeuticsUSZFN Technology[23]5I/II18Sarepta Therapeutics, Duke UniversityUSCRISPR / Cas9[24]NADiscovery19TransposagenUSFootprint-Free[25], [26]NANA20Verve TherapeuticsUSCRISPR[27]1PreclinicalAbbreviations: HQ: Headquarters; ZFN: Zinc Finger Nuclease; TALEN: Transcription Activator-like Effector Nuclease Source: Roots AnalysisÂ