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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 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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