Editas Medicine, Inc., (formerly Gengine, Inc.), is a clinical-stage
biotechnology
Biotechnology is the integration of natural sciences and engineering sciences in order to achieve the application of organisms, cells, parts thereof and molecular analogues for products and services. The term ''biotechnology'' was first used b ...
company which is developing therapies for rare diseases based on
CRISPR gene editing
CRISPR gene editing (pronounced "crisper") is a genetic engineering technique in molecular biology by which the genomes of living organisms may be modified. It is based on a simplified version of the bacterial CRISPR-Cas9 antiviral defense sys ...
technology.
Editas headquarters is located in
Cambridge, Massachusetts
Cambridge ( ) is a city in Middlesex County, Massachusetts, United States. As part of the Boston metropolitan area, the cities population of the 2020 U.S. census was 118,403, making it the fourth most populous city in the state, behind Boston, ...
and has facilities in
Boulder, Colorado
Boulder is a home rule city that is the county seat and most populous municipality of Boulder County, Colorado, United States. The city population was 108,250 at the 2020 United States census, making it the 12th most populous city in Color ...
.
History
Editas Medicine was originally founded with the name "Gengine, Inc." in September 2013 by
Feng Zhang
Feng Zhang (; born October 22, 1981) is a Chinese-American biochemist. Zhang currently holds the James and Patricia Poitras Professorship in Neuroscience at the McGovern Institute for Brain Research and in the departments of Brain and Cognitive ...
of the
Broad Institute
The Eli and Edythe L. Broad Institute of MIT and Harvard (IPA: , pronunciation respelling: ), often referred to as the Broad Institute, is a biomedical and genomic research center located in Cambridge, Massachusetts, Cambridge, Massachusetts, U ...
,
Jennifer Doudna
Jennifer Anne Doudna (; born February 19, 1964) is an American biochemist who has done pioneering work in CRISPR gene editing, and made other fundamental contributions in biochemistry and genetics. Doudna was one of the first women to share a ...
of the
University of California, Berkeley
The University of California, Berkeley (UC Berkeley, Berkeley, Cal, or California) is a public land-grant research university in Berkeley, California. Established in 1868 as the University of California, it is the state's first land-grant u ...
, and
George Church,
David Liu, and
J. Keith Joung of
Harvard University
Harvard University is a private Ivy League research university in Cambridge, Massachusetts. Founded in 1636 as Harvard College and named for its first benefactor, the Puritan clergyman John Harvard, it is the oldest institution of higher le ...
, with funding from
Third Rock Ventures
Third Rock Ventures is a venture capital firm based in Boston
Boston (), officially the City of Boston, is the state capital and most populous city of the Commonwealth of Massachusetts, as well as the cultural and financial center of ...
,
Polaris Partners
Polaris Partners is a venture capital firm active in the field of healthcare and biotechnology companies. The company has offices in Boston, Massachusetts, New York, NY and San Francisco, California.
History
Polaris Partners was founded in 1996 ...
and
Flagship Ventures; the name was changed to the current "Editas Medicine" two months later. Doudna quit in June 2014 over legal differences concerning intellectual property of
Cas9
Cas9 (CRISPR associated protein 9, formerly called Cas5, Csn1, or Csx12) is a 160 kilodalton protein which plays a vital role in the immunological defense of certain bacteria against DNA viruses and plasmids, and is heavily utilized in genetic e ...
.
[
In August 2015, the company raised $120 million in ]Series B
A venture round is a type of funding round used for venture capital financing, by which startup companies obtain investment, generally from venture capitalists and other institutional investors. The availability of venture funding is among the ...
funding from Bill Gates
William Henry Gates III (born October 28, 1955) is an American business magnate and philanthropist. He is a co-founder of Microsoft, along with his late childhood friend Paul Allen. During his career at Microsoft, Gates held the positions ...
and 13 other investors. it went public on 2 February 2016,[ with an IPO that raised $94 million.
The company entered into a strategic collaboration with ]Juno Therapeutics
Juno Therapeutics Inc is an American biopharmaceutical company founded in 2013 through a collaboration of the Fred Hutchinson Cancer Research Center, Memorial Sloan-Kettering Cancer Center and pediatrics partner Seattle Children's Research Instit ...
in 2015 to combine its CRISPR-Cas9 technology with Juno's experience in creating chimeric antigen receptor
In biology, chimeric antigen receptors (CARs)—also known as chimeric immunoreceptors, chimeric T cell receptors or artificial T cell receptors—are receptor proteins that have been engineered to give T cells the new ability to target a specific ...
and high-affinity T cell receptor
The T-cell receptor (TCR) is a protein complex found on the surface of T cells, or T lymphocytes, that is responsible for recognizing fragments of antigen as peptides bound to major histocompatibility complex (MHC) molecules. The binding b ...
therapeutics to the end of developing cancer therapeutics. Juno was later acquired by Celgene
Celgene Corporation is a pharmaceutical company that makes cancer and immunology drugs. Its major product is Revlimid ( lenalidomide), which is used in the treatment of multiple myeloma, and also in certain anemias. The company is incorporated in ...
, which was in turn acquired by Bristol Myers Squibb
The Bristol Myers Squibb Company (BMS) is an American multinational pharmaceutical company. Headquartered in New York City, BMS is one of the world's largest pharmaceutical companies and consistently ranks on the ''Fortune'' 500 list of the lar ...
.
The company announced in 2015 that it was planning a clinical trial in 2017 using CRISPR gene editing techniques to treat Leber congenital amaurosis
Leber congenital amaurosis (LCA) is a rare inherited eye disease that appears at birth or in the first few months of life.
It affects about 1 in 40,000 newborns. LCA was first described by Theodor Leber in the 19th century. It should not be co ...
type 10 (LCA10), a rare genetic illness that causes blindness. On 30 November 2018, the FDA
The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food ...
gave permission to start the trials, under the investigational name EDIT-101 (also known as AGN-151587). In September 2021, a statement from Editas claimed that preliminary results from clinical trials were promising and support clinical benefits of EDIT-101 treatment.
In March 2020, Editas, in partnership with Allergan
Allergan plc is an American, Irish-domiciled pharmaceutical company that acquires, develops, manufactures and markets brand name drugs and medical devices in the areas of medical aesthetics, eye care, central nervous system, and gastroenterology. ...
, was the first to use CRISPR to try to edit DNA inside a person's body (''in vivo
Studies that are ''in vivo'' (Latin for "within the living"; often not italicized in English) are those in which the effects of various biological entities are tested on whole, living organisms or cells, usually animals, including humans, and ...
''). As part of the clinical trial, a patient who was nearly blind as a result of Leber's congenital amaurosis received an intravitreal injection
Intravitreal injection is the method of administration of drugs into the eye by injection with a fine needle. The medication will be directly applied into the vitreous humor. It is used to treat various eye diseases, such as age-related macular d ...
containing a harmless virus carrying CRISPR gene-editing instructions. Five months later, Editas reworked its deal with Allergan's owner AbbVie
AbbVie is an American publicly traded biopharmaceutical company founded in 2013. It originated as a spin-off of Abbott Laboratories.
History
On October 19, 2011, Abbott Laboratories announced its plan to separate into two publicly traded compan ...
and regained full rights to their range of eye disease treatment therapies, including EDIT-101 for the treatment of LCA10.
In 2019, the company was building new chemistry facilities in Boulder, Colorado
Boulder is a home rule city that is the county seat and most populous municipality of Boulder County, Colorado, United States. The city population was 108,250 at the 2020 United States census, making it the 12th most populous city in Color ...
.
Katrine Bosley was CEO until 2019, when she was replaced by board member Cynthia Collins. Collins was replaced in 2021 by James Mullen, who had been board chairman. Gilmore O'Neill, former CMO of Sarepta Therapeutics
Sarepta Therapeutics, Inc. () is a medical research and drug development company with corporation, corporate offices and research facilities in Cambridge, Massachusetts, United States. Incorporated in 1980 as AntiVirals, , archiveurl = http://ww ...
, became CEO on June 1, 2022, with Mullen staying on as executive chairman of the board.
Research
Editas works with two different CRISPR nucleases
A nuclease (also archaically known as nucleodepolymerase or polynucleotidase) is an enzyme capable of cleaving the phosphodiester bonds between nucleotides of nucleic acids. Nucleases variously effect single and double stranded breaks in their ta ...
, Cas9 and Cas12a
Cas12a (CRISPR associated protein 12a, previously known as Cpf1) is an RNA-guided endonuclease of that forms part of the CRISPR system in some bacteria and is used by scientists to modify DNA. It originates as part of a bacterial immune mechan ...
.
EDIT-101 is a CRISPR based gene therapy for treatment of Leber congenital amaurosis, which is currently in clinical trials.
EDIT-301 is an experimental potential treatment utilizing the firm's CAS 12a editing technology for sickle cell disease
Sickle cell disease (SCD) is a group of blood disorders typically inherited from a person's parents. The most common type is known as sickle cell anaemia. It results in an abnormality in the oxygen-carrying protein haemoglobin found in red blo ...
and beta-thalassemia
Beta thalassemias (β thalassemias) are a group of inherited blood disorders. They are forms of thalassemia caused by reduced or absent synthesis of the beta chains of hemoglobin that result in variable outcomes ranging from severe anemia to cl ...
. In 2019 the firm reported early success in research on the drug;. In December 2020, it filed an IND application for treatment of sickle cell disease. In January 2021, it said it had received clearance from the FDA for phase 1 safety studies.
References
{{reflist
Pharmaceutical companies of the United States
Companies based in Cambridge, Massachusetts
Companies listed on the Nasdaq
Gene therapy
Health care companies based in Massachusetts
Pharmaceutical companies established in 2013
Biotechnology companies of the United States