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:

• 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 Pipeline2.

Highest Phase of Development

1

Beam Therapeutics

US

CRISPR3.

1

Undisclosed

2

Bioverativ, Sangamo Therapeutics

US

ZFN Technology4.

1

I/II

3

bluebird bio

US

Homing endonuclease and megaTAL5.

1

Preclinical

4

Caribou Biosciences

US

CRISPR / Cas6.

NA

NA

5

Cellectis

France

TALEN7., 8.

NA

NA

6

CRISPR Therapeutics

US

CRISPR / Cas99.

5

I/II

7

EdiGene

China

CRISPR10.

4

I/II (Planned)

8

Editas Medicine

US

CRISPR / Cas9, TALEN11., 12.

6

I/II

9

Exonics Therapeutics

US

CRISPR / Cas913.

1

Preclinical

10

Homology Medicines

US

Proprietary Technology14.

4

Preclinical

11

Horizon Discovery Group

England

Proprietary Technology15.

NA

NA

12

Intellia Therapeutics

US

CRISPR / Cas916.

5

Preclinical

13

LogicBio Therapeutics

US

GeneRide17., 18.

4

Preclinical

14

Novartis

Switzerland

Proprietary AAV-mediated editing by Directed Homologous Recombination19.

NA

NA

15

Poseida Therapeutics

US

Cas-CLOVER20.

NA

NA

16

Precision BioSciences

US

ARCUS21., 22.

NA

NA

17

Sangamo Therapeutics

US

ZFN Technology23.

5

I/II

18

Sarepta Therapeutics, Duke University

US

CRISPR / Cas924.

NA

Discovery

19

Transposagen

US

Footprint-Free25., 26.

NA

NA

20

Verve Therapeutics

US

CRISPR27.

1

Preclinical

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

Source: Roots Analysis

The "Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy (Ex vivo and In vivo), Type of Vectors used (Adeno Associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Plasmid DNA, Retrovirus and Others), Target Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Dermatological Disorders, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebellar, Intradermal, Intramuscular, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal and Others), and Key Geographical Regions (US, EU5 and rest of the world): Industry Trends and Global Forecasts, 2020-2030" report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market. Amongst other elements, the report features:

• A detailed review of the overall market landscape of gene therapies and gene editing therapies, including information on phase of development (marketed, clinical, preclinical and discovery) of pipeline candidates, key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene, therapeutic approach (gene augmentation, oncolytic viral therapy and others), type of gene therapy (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any).
• A detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain.
• An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors.
• A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies.
• Detailed profiles of marketed and late stage (phase II/III and above) gene therapies, including development timeline of the therapy, information on the current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, as well as details related to the developer company.

Roots Analysis provides market research and consulting in the pharmaceutical industry. Focussed on providing an informed and impartial view on key issues facing the industry, our research is primarily driven by an in-depth analysis.