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
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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