Gene Editing Companies

CRISPR Therapeutics
CRISPR Therapeutics AG
Sector: Healthcare
Industry: Biotechnology & Medical Research
Employees: 188
 
CRISPR Therapeutics AG is a Switzerland-based gene-editing company. It focuses on the development of transformative gene-based medicines for serious diseases. The Company develops its products using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 gene-editing platform, which allows for precise directed changes to genomic deoxyribonucleic acid (DNA). The Company has a portfolio of therapeutic programs in a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine and rare diseases. Its lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from transfusion-dependent beta thalassemia or severe sickle cell disease in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. The Company has business operations in London and the United Kingdom, as well as research and development operations in the United States.
Editas
Editas Medicine Inc.
Sector: Healthcare
Industry: Biotechnology & Medical Research
Employees: 133
 
Editas Medicine, Inc. is a genome editing company. It is engaged in treating patients with genetically defined diseases by correcting their disease-causing genes. It operates through developing and commercializing genome editing technology segment. It is developing a genome editing platform based on clustered, regularly interspaced short palindromic repeats (CRISPR) technology. CRISPR uses a protein-ribonucleic acid (RNA) complex composed of an enzyme, including either CRISPR associated protein 9 (Cas9) or CRISPR from Prevotella and Francisella 1 (Cpf1), bound to a guide RNA molecule designed to recognize a particular deoxyribonucleic acid (DNA) sequence. Its platform consists of four components: nuclease engineering, delivery, control and specificity, and directed editing. Its programs include Eye Diseases, Engineered T Cell Therapies for Immuno-Oncology and additional research programs. It is developing a genome editing therapeutic for Leber Congenital Amaurosis type 10 (LCA10).
Intellia
Intellia Therapeutics Inc.
Sector: Healthcare
Industry: Biotechnology & Medical Research
Employees: 211
 
Intellia Therapeutics, Inc. is a genome editing company. The Company is focused on developing therapeutics utilizing a biological tool known as CRISPR/Cas9. The CRISPR/Cas9 genome editing system includes two components: the Cas9 protein and the guide RNA sequence. The Cas9 protein acts like a pair of molecular scissors that initiates the natural cellular repair process to knockout, repair or insert a gene. The guide RNA sequence recognizes and directs the Cas9 to a specific target deoxyribonucleic acid (DNA) sequence. The Company’s sentinel in vivo programs focus on the use of Lipid Nanoparticle (LNPs) for delivery of the CRISPR/Cas9 complex to the liver. The Company’s division, eXtellia Therapeutics, is focused on the application of CRISPR/Cas9 genome editing in the fields of immuno-oncology and autoimmune and inflammatory diseases. The Company’s subsidiary is Intellia Securities Corp. As of September 30, 2016, the Company had not generated any revenue.