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Human Gene Editing

Reinventing gene therapies with next-generation technologies

Overview

Gene editing refers to the modification of DNA in the human genome, either by directly delivering gene-editing components into the body or by delivering cells edited in a specialized laboratory to the patient's body. It is often applied in the areas of oncology, hematology, neurology, and genetic disease. 

Amongst a variety of gene editing technologies utilized by biotech companies, the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR/Cas9) method has gained traction in recent times due to its simplicity, versatility, and accuracy. Other comparatively old technologies include transcription activator-like effector-based nucleases (TALEN), zinc finger nucleases (ZFN), and meganucleases, which have certain benefits of their own such as low cost (ZFN), higher specificity (TALEN), and low toxicity (meganucleases). At present, gene editing therapies have not been commercialized, but several have already reached Phase I and II clinical trials, with the majority of them based on the CRISPR technology.

Note: Currently, we do not actively cover this industry, but we have identified it as a potential industry to cover.

More details

Market Mapping


Incumbents
Growth
Early
Seed
Pre-Seed
R&D therapies: CRISPR/Cas9 technology
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R&D therapies: Other technologies
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Diagnostic and delivery systems
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Gene editing: SaaS platforms
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Gene editing support services
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Biotherapeutics
Biotherapeutics
Biotherapeutics
Biotherapeutics
Biotherapeutics
Moderna
Bristol-Myers Squibb
Pfizer
Vertex Pharmaceuticals
Merck
Eli Lilly
Amgen
Bayer
Novartis
Sarepta Therapeutics
AstraZeneca
CRISPR Therapeutics
Editas Medicine
Repare Therapeutics
Caribou Biosciences
Excision BioTherapeutics
Intellia Therapeutics
Sana Biotechnology
Beam Therapeutics
Century Therapeutics
Poseida Therapeutics
Prime Medicine
Mammoth Biosciences
Arbor Biotechnologies
Verve Therapeutics
Graphite Bio
SparingVision
Scribe Therapeutics
Qihan Biotech
Spotlight Therapeutics
Recombinetics
Emendo Biotherapeutics
SNIPR Biome
Eligo Bioscience
Refuge Biotechnologies
Flash Therapeutics
Acrigen Biosciences
Lempo Therapeutics
Carver Biosciences
Amgen
Sangamo Therapeutics
Bluebird Bio
Precision Biosciences
Cellectis
2seventy Bio
Tessera Therapeutics
Metagenomi
eGenesis
Homology Medicines
Obsidian Therapeutics
LogicBio Therapeutics
Recombinetics
iECURE
Flash Therapeutics
Genespire
Cytovia Therapeutics
Exacis Biotherapeutics
Google
Sherlock Biosciences
Poseida Therapeutics
Mammoth Biosciences
LogicBio Therapeutics
GenEdit
Vesigen Therapeutics
Hopewell Therapeutics
Ligandal
Mediphage Bioceuticals
Hunterian Medicine
CASPR Biotech
CyGenica
CellFE
Twelve Bio
ElevateBio
Inscripta
Avectas
Homology Medicines
Locus Biosciences
Watchmaker Genomics
Integra Therapeutics
Biopalette
PerkinElmer
Thermo Fisher Scientific
Danaher
Synthego
ElevateBio
Sangamo Therapeutics
Inscripta
Biocytogen
Homology Medicines
Origene Technologies
Definigen
TigerQ
Hera BioLabs
GeneLancet Biosciences

The Disruptors


R&D therapies: CRISPR/Cas9 technology

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Disruptors

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Funding in USD Millions
CRISPR Therapeutics
Public - Market cap USD 5.1 bn
Intellia Therapeutics
Public - Market cap USD 4.3 bn
Beam Therapeutics
Public - Market cap USD 3.4 bn
Verve Therapeutics
Public - Market cap USD 2.1 bn
Sana Biotechnology
Public - Market cap USD 1.1 bn
Editas Medicine
Public - Market cap USD 841.3 mn
Caribou Biosciences
Public - Market cap USD 641.9 mn
Century Therapeutics
Public - Market cap USD 582.3 mn
Repare Therapeutics
Public - Market cap USD 508.7 mn
Poseida Therapeutics
Public - Market cap USD 302.8 mn
Graphite Bio
Public - Market cap USD 184.5 mn
Prime Medicine
315
Mammoth Biosciences
265
Arbor Biotechnologies
231
SparingVision
128
Scribe Therapeutics
120
Qihan Biotech
100
Spotlight Therapeutics
81
Excision BioTherapeutics
78
Recombinetics
68
Emendo Biotherapeutics
61
SNIPR Biome
60
Eligo Bioscience
40
Refuge Biotechnologies
34
Flash Therapeutics
22
Lempo Therapeutics
Unknown
Watchlist
?
Acrigen Biosciences
Carver Biosciences

R&D therapies: Other technologies

?

Disruptors

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Funding in USD Millions
Sangamo Therapeutics
Public - Market cap USD 767.1 mn
2seventy Bio
Public - Market cap USD 551.1 mn
Bluebird Bio
Public - Market cap USD 488.2 mn
Precision Biosciences
Public - Market cap USD 144.1 mn
Cellectis
Public - Market cap USD 108.6 mn
Homology Medicines
Public - Market cap USD 92.4 mn
LogicBio Therapeutics
Public - Market cap USD 9.0 mn
Tessera Therapeutics
532
Metagenomi
277
eGenesis
265
Obsidian Therapeutics
165
Recombinetics
68
iECURE
50
Flash Therapeutics
22
Cytovia Therapeutics
Unknown
Exacis Biotherapeutics
Unknown
Watchlist
?
Genespire

Diagnostic and delivery systems

?

Disruptors

?
Funding in USD Millions
Poseida Therapeutics
Public - Market cap USD 302.8 mn
LogicBio Therapeutics
Public - Market cap USD 9.0 mn
Mammoth Biosciences
265
Sherlock Biosciences
136
GenEdit
44
Vesigen Therapeutics
29
Hopewell Therapeutics
15
CASPR Biotech
3
Watchlist
?
CellFE
Ligandal
Mediphage Bioceuticals
Hunterian Medicine
CyGenica
Twelve Bio

Gene editing: SaaS platforms

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Disruptors

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Funding in USD Millions
Homology Medicines
Public - Market cap USD 92.4 mn
ElevateBio
845
Inscripta
459
Locus Biosciences
129
Avectas
39
Integra Therapeutics
7
Watchlist
?
Watchmaker Genomics
Biopalette

Gene editing support services

?

Disruptors

?
Funding in USD Millions
Sangamo Therapeutics
Public - Market cap USD 767.1 mn
Homology Medicines
Public - Market cap USD 92.4 mn
ElevateBio
845
Synthego
460
Inscripta
459
Biocytogen
335
Origene Technologies
69
Definigen
13
Watchlist
?
TigerQ
GeneLancet Biosciences
Hera BioLabs

CRISPR Therapeutics

CRISPR Therapeutics specializes in developing gene editing-based novel therapeutics for a variety of areas including immuno-oncology and hemoglobinopathies. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 enzymes, and focuses predominantly on treating somatic cells that do not transfer DNA to the next generations, preventing future generations from contracting a genetic disease. 

The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of June 2022, the company  had three internal and two collaborative drug candidates in its pipeline, all of which were in the clinical stage. It was also researching other in vivo approaches for four disease indications. 

Key customers and partnerships

The company has secured strategic collaborations with a number of companies to co-develop and co-commercialize gene editing medicines that include Vertex Pharmaceuticals (2015) to develop a therapy (CTX001) for various indications including beta-thalassemia, sickle cell disease, and Duchenne muscular dystrophy; and ViaCyte (September 2018) to develop VCTX210, a regenerative medicine for diabetes.

Big pharma Bayer also signed an option agreement in 2019 with the company to co-develop and co-commercialize two candidates, after entirely handing over the joint venture Casebia Therapeutics to CRISPR.  

Funding and financials

Founded in 2013, CRISPR has been listed on the Nasdaq since 2016. For 2021, the company reported a revenue of USD 913.1 million earned via milestone payments, collaborations, and licensing.

Segment:
R&D therapies: CRISPR/Cas9 technology
Total funding:
USD 127.0 million
Disruptor Funding History

R&D therapies: CRISPR/Cas9 technology:

CRISPR Therapeutics
Intellia Therapeutics
Beam Therapeutics
Verve Therapeutics
Sana Biotechnology
Editas Medicine
Caribou Biosciences
Century Therapeutics
Repare Therapeutics
Poseida Therapeutics
Graphite Bio
Prime Medicine
Mammoth Biosciences
Arbor Biotechnologies
SparingVision
Scribe Therapeutics
Qihan Biotech
Spotlight Therapeutics
Excision BioTherapeutics
Recombinetics
Emendo Biotherapeutics
SNIPR Biome
Eligo Bioscience
Refuge Biotechnologies
Flash Therapeutics
Acrigen Biosciences

R&D therapies: Other technologies:

Sangamo Therapeutics
2seventy Bio
Bluebird Bio
Precision Biosciences
Cellectis
Homology Medicines
LogicBio Therapeutics
Tessera Therapeutics
Metagenomi
eGenesis
Obsidian Therapeutics
Recombinetics
iECURE
Flash Therapeutics
Genespire

Diagnostic and delivery systems:

Poseida Therapeutics
LogicBio Therapeutics
Mammoth Biosciences
Sherlock Biosciences
GenEdit
Vesigen Therapeutics
Hopewell Therapeutics
CASPR Biotech
CellFE
Ligandal
Mediphage Bioceuticals
Hunterian Medicine
CyGenica
Twelve Bio

Notable Investors


No investor data is available

Funding data are powered by Crunchbase
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