Gene Therapy Market – Gene Editing Technologies

Gene editing technologies allow the researchers to change the genetic material of an organism. This change can be due to any addition, deletion or alt

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: This involves the addition of new attributes to a gene through the incorporation of nucleotide sequences.
  • Gene Repair: This refers to the replacement of a defective gene sequence by a functional sequence.
  • Gene Inactivation: 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 Platforms

S. No. Company Name HQ Technology Number of Molecules in Pipeline[2] Highest Phase of Development
1 Beam Therapeutics US CRISPR[3] 1 Undisclosed
2 Bioverativ, Sangamo Therapeutics US ZFN Technology[4] 1 I/II
3 bluebird bio US Homing endonuclease and megaTAL[5] 1 Preclinical
4 Caribou Biosciences US CRISPR / Cas[6] NA NA
5 Cellectis France TALEN[7][8] NA NA
6 CRISPR Therapeutics US CRISPR / Cas9[9] 5 I/II
7 EdiGene China CRISPR[10] 4 I/II (Planned)
8 Editas Medicine US CRISPR / Cas9, TALEN[11][12] 6 I/II
9 Exonics Therapeutics US CRISPR / Cas9[13] 1 Preclinical
10 Homology Medicines US Proprietary Technology[14] 4 Preclinical
11 Horizon Discovery Group England Proprietary Technology[15] NA NA
12 Intellia Therapeutics US CRISPR / Cas9[16] 5 Preclinical
13 LogicBio Therapeutics US GeneRide[17][18] 4 Preclinical
14 Novartis Switzerland Proprietary AAV-mediated editing by Directed Homologous Recombination[19] NA NA
15 Poseida Therapeutics US Cas-CLOVER[20] NA NA
16 Precision BioSciences US ARCUS[21][22] NA NA
17 Sangamo Therapeutics US ZFN Technology[23] 5 I/II
18 Sarepta Therapeutics, Duke University US CRISPR / Cas9[24] NA Discovery
19 Transposagen US Footprint-Free[25][26] NA NA
20 Verve Therapeutics US CRISPR[27] 1 Preclinical

Abbreviations: HQ: Headquarters; ZFN: Zinc Finger Nuclease; TALEN: Transcription Activator-like Effector Nuclease

 

Source: Roots Analysis

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