Top 10 Gene Editing Companies To Look For In 2019

Since the dawn of civilization, humans have always been curious, and eager to discover new things. Today, science and technology have reached such a time where we are able to modify nature and living organisms too. Gene editing is a technique of changing the phenotype of an organism by introducing, repairing or deleting a specific gene in them. It can be done with various tools like CRISPR Cas9, adenovirus, and so on. Various applications of gene editing are gene therapy to treat various genetically caused diseases; production of superior crops in agriculture; etc. With billions of dollars being invested in the research for gene editing and biopharmaceuticals, there are a lot of blooming gene editing companies in today’s market.

Gene Editing Companies With High Prospects In 2019

Amongst numerous biotech companies and startups in 2019, some companies focus on gene editing. Some of the most promising gene editing companies are listed below.

1. Editas Medicine

  • Annual Revenue: $31.9M
  • Headquarters: Cambridge, Massachusetts
  • Number of Employees: 133
  • Total Funding: $257.4M
  • Status: Public Independent Company

Established in 2013 by Feng Zhang, David Liu, George Church, J. Keith Joung, and Jennifer Doudna, Editas Medicine operates in the field of pharmaceuticals. The company has aimed at treating patients with genetically defined diseases (e.g. Leber Congenital Amaurosis LCA) by using CRISPR- based gene therapy. One of the major projects the company has been undertaking is the treatment of LCA (causing childhood blindness) by eliminating the mutation in the CEP290 gene along with other muscle, blood, liver and lung diseases.

2. Intellia Therapeutics

  • Annual Revenue: $29.9 M
  • Headquarters: Cambridge, USA
  • Number of Employees: 195
  • Total Funding: $193M
  • Status: Public Subsidiary of CCH Small Firm Services

Intellia Therapeutics was founded in 2014 by Jennifer Doudna and colleagues at the University of California. It has been developing biopharmaceuticals using a CRISPR gene editing system. It has it’s own in vivo and ex vivo programs. The in vivo programs focuses on the use of Lipid Nanoparticles (LNPs0 for delivery of CRISPR/ Cas9 complex to the liver targeting transthyretin amyloidosis (ATTR).The ex vivo programs are partnered and focus on chimeric antigen receptor R cells (“CAR T cells”) and hematopoietic stem cells. The company has partnerships with Novartis and Regeneron.

3. Homology Medicines

  • Annual Revenue: $285.3 M
  • Headquarters:  Bedford, Massachusetts, United States
  • Number of Employees: 86
  • Total Funding: $271M
  • Status: Public Independent Company

Homology Medicines was established in 2015. Since its establishment, Homology Medicines’ has been researching and generating outstanding results in the field of gene therapy. It’s lead program is an AAVHSC-mediated gene therapy for adults with the rare disease phenylketonuria. The gene editing is based on a novel set of adeno-associated virus vectors derived from human hematopoietic stem cells (AAVHSCs) that are designed to precisely and efficiently deliver genetic medicines in vivo either through gene therapy or by harnessing the body’s natural DNA repair process of homologous recombination through nuclease-free gene editing.

4. Spark Therapeutics

  • Annual Revenue: $29.9 M
  • Headquarters: Philadelphia, USA
  • Number of Employees: 195
  • Total Funding: $193M
  • Status: Public Subsidiary of F. Hoffmann-La Roche Ltd.

Spark Therapeutics was established in 2013 by Katherine High and Jeffrey Marrazzo in an effort to commercially develop treatments against hemophilia. Spark Therapeutics has been using adeno-associated viral (AAV) vectors to design research programs against strategically selected cell targets like the retina, liver, and CNS. One of the major products of Spark Therapeutics is Voretigene neparvovec, marketed under the tradename Luxturna: a gene therapy approved by the FDA for treatment of Leber’s congenital amaurosis, a rare genetic eye disease. In February 2019, Roche announced it would acquire the company for $4.3 billion.

5. Precision Biosciences

 

 

 

Precision Biosciences

Source

  • Annual Revenue: $10.9 M
  • Headquarters: Durham, North Carolina
  • Status: Private Independent Company
  • Number of Employees: 127
  • Total Funding: $117.3M

Precision Biosciences was founded in 2006. It has been engaging in the development and commercialization of therapeutics and services based on genomic molecular biology. The gene editing tool used in Precision Biosciences is ARCUS which has been derived from a natural genome editing enzyme called homing endonuclease. The ARC nuclease; similar but improved homing endonuclease can be customized to recognize a DNA sequence within any target gene. Precision Biosciences has been using ARCUS for Gene therapy, Food, and Cancer Immunotherapy.

6. Synthego

 

  • Headquarters: Redwood City, California
  • Status: Private Independent Company
  • Annual Revenue: $3 M
  • Number of Employees: 130
  • Total Funding: $159.7M

Synthego was founded in 2012. It was aimed at providing genome engineering solutions for CRISPR editing and research. Some of the services provided by Synthego are CRISPR Design Tools (for knockout and optimization), NGS quality results tool in the field of Bioinformatics. Additionally, It has developed various CRISPRevolutionary Kits (Gene Knockout Kit, Synthetic cr:tracr RNA kit, Synthetic sgRNA, etc.).

7. Horizon Discovery

  • Headquarters: Cambridge, England
  • Status: Private Independent Company
  • Employees: 500
  • Funding: $21.2M
  • Revenue: $35M

Horizon Discovery was founded in 2007 by….. A UK-based company, Horizon Discovery provides cell panel screening, data analysis, model generation, apoptosis assays and custom breeding solutions for drug manufacturers. Some of the services provided by Horizon Discovery are CRISPR Screening, CRISPRa Screening, CRISPRi Screening, Cell Line Engineering (HAP1 cell lines, immune cells), iPSC services, etc. It is estimated to be worth over $20Billion.

8. NightStar Therapeutics

 

  • Headquarters: London, England
  • Status: Public Subsidiary of Biogen, Inc. NASDAQNITE  800M $ bought on 2019
  • Funding Raised: $75M
  • Revenue: $2.1M

NightStar Therapeutics is a biopharmaceutical company that utilized vectors based on adeno-associated virus (AAV) for the treatment of genetically defined diseased like choroideremia (CHM). It was established in 2013. Its lead retinal gene therapy product candidate NSR-REP1 is being developed for the same. Recently, in March 2019, NightStar Therapeutics has been acquired by Biogen, Inc for $800M.

9. Crispr Therapeutics

  • Headquarters: Zug, Switzerland
  • Status: Public Independent Company
  • Funding Collected: $218M
  • Revenue: $3.1M
  • Number of Employees: 188

CRISPR Therapeutics was founded in 2013. It has been utilizing CRISPR/CAS9 technology for the treatment of genetic diseases, either in-vivo or ex-vivo. Their lead program is in the treatment of hemoglobinopathies that uses ex-vivo gene editing. Also, the company has been researching Immunooncology and Regenerative Medicines.

10. Pairwise Plants

Pairwise Plants

  • Headquarters: San Diego, California
  • Status: Private Independent Company
  • Total Capital Raised:$125M

Pairwise Plants focuses on solving agricultural problems, and creating new crops and improving existing ones using gene editing. Pairwise has been using CRISPR proteins for gene editing.  Major investor of Pairwise is Deerfield and MGV while it has also been working on a collaboration with Harvard University.

The financial information has been generated from their respective latest SEC filings.* (derived from owler.com)

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