Editas Medicine

Last updated
Editas Medicine, Inc.
FormerlyGengine, Inc.
Company type Public
Industry
FoundedNovember 2013;10 years ago (2013-11), in Cambridge, Massachusetts
Founders
Headquarters
Cambridge, Massachusetts
,
United States
Number of locations
2
Key people
RevenueDecrease2.svgUS$19.7 million (2022)
Decrease2.svgUS$−226 million (2022)
Decrease2.svgUS$−220 million (2022)
Total assets Decrease2.svgUS$514 million (2022)
Total equity Decrease2.svgUS$361 million (2022)
Number of employees
226 (2023)
Website editasmedicine.com
Footnotes /references
[1]

Editas Medicine, Inc., (formerly Gengine, Inc.), is a clinical-stage biotechnology company which is developing therapies for rare diseases based on CRISPR gene editing technology. [2] [3] Editas headquarters is located in Cambridge, Massachusetts and has facilities in Boulder, Colorado. [4] [5] [6]

Contents

History

Editas Medicine was originally founded with the name "Gengine, Inc." in September 2013 by Feng Zhang of the Broad Institute, Jennifer Doudna of the University of California, Berkeley, [7] and George Church, David Liu, and J. Keith Joung of Harvard University, with funding from Third Rock Ventures, Polaris Partners 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. [8] [9] [10]

In August 2015, the company raised $120 million in Series B funding from Bill Gates and 13 other investors. [11] [12] it went public on 2 February 2016, [2] via an initial public offering that raised $94 million. [13] [14]

The company entered into a strategic collaboration with Juno Therapeutics in 2015 to combine its CRISPR-Cas9 technology with Juno's experience in creating chimeric antigen receptor and high-affinity T cell receptor therapeutics to the end of developing cancer therapeutics. [15] Juno was later acquired by Celgene, [16] which was in turn acquired by Bristol Myers Squibb. [17]

The company announced in 2015 that it was planning a clinical trial in 2017 using CRISPR gene editing techniques to treat Leber congenital amaurosis type 10 (LCA10), a rare genetic illness that causes blindness. [18] [10] On 30 November 2018, the FDA gave permission to start the trials, under the investigational name EDIT-101 (also known as AGN-151587). [19] [20] In September 2021, a statement from Editas claimed that preliminary results from clinical trials were promising and support clinical benefits of EDIT-101 treatment. [21]

In March 2020, Editas, in partnership with Allergan, was the first to use CRISPR to try to edit DNA inside a person's body ( in vivo ). As part of the clinical trial, a patient who was nearly blind as a result of Leber's congenital amaurosis received an intravitreal injection containing a harmless virus carrying CRISPR gene-editing instructions. [22] [23] Five months later, Editas reworked its deal with Allergan's owner AbbVie and regained full rights to their range of eye disease treatment therapies, including EDIT-101 for the treatment of LCA10. [24]

In 2019, the company was building new chemistry facilities in Boulder, Colorado. [5]

Katrine Bosley was CEO until 2019, when she was replaced by board member Cynthia Collins. [25] [26] Collins was replaced in 2021 by James Mullen, who had been board chairman. [27] Gilmore O'Neill, former CMO of Sarepta Therapeutics, became CEO on June 1, 2022, with Mullen staying on as executive chairman of the board. [28]

Research

Editas works with two different CRISPR nucleases, Cas9 and Cas12a. [29]

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 and beta-thalassemia. In 2019 the firm reported early success in research on the drug;. [30] [31] 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. [32]

Related Research Articles

<span class="mw-page-title-main">Gene therapy</span> Medical field

Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.

<span class="mw-page-title-main">Human genetic enhancement</span> Technologies to genetically improve human bodies

Human genetic enhancement or human genetic engineering refers to human enhancement by means of a genetic modification. This could be done in order to cure diseases, prevent the possibility of getting a particular disease, to improve athlete performance in sporting events, or to change physical appearance, metabolism, and even improve physical capabilities and mental faculties such as memory and intelligence. These genetic enhancements may or may not be done in such a way that the change is heritable.

<span class="mw-page-title-main">Designer baby</span> Genetically modified human embryo

A designer baby is a baby whose genetic makeup has been selected or altered, often to exclude a particular gene or to remove genes associated with disease. This process usually involves analysing a wide range of human embryos to identify genes associated with particular diseases and characteristics, and selecting embryos that have the desired genetic makeup; a process known as preimplantation genetic diagnosis. Screening for single genes is commonly practiced, and polygenic screening is offered by a few companies. Other methods by which a baby's genetic information can be altered involve directly editing the genome before birth, which is not routinely performed and only one instance of this is known to have occurred as of 2019, where Chinese twins Lulu and Nana were edited as embryos, causing widespread criticism.

Leber congenital amaurosis (LCA) is a rare inherited eye disease that appears at birth or in the first few months of life.

<span class="mw-page-title-main">Cellectis</span> French biopharmaceutical company

Cellectis is a French biopharmaceutical company. It develops genome-edited chimeric antigen receptor T-cell technologies for cancer immunotherapy. It has offices in Paris, New York City, and Raleigh, North Carolina.

<span class="mw-page-title-main">Juno Therapeutics</span> American biopharmaceutical company

Juno Therapeutics Inc was 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 Institute. The company was launched with an initial investment of $120 million, with a remit to develop a pipeline of cancer immunotherapy drugs. The company raised $300 million through private funding and a further $265 million through their IPO.

Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis.

<span class="mw-page-title-main">Intellia Therapeutics</span> American biotechnology company

Intellia Therapeutics, Inc. is an American clinical-stage biotechnology company focused on developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. The company's in vivo programs use intravenously administered CRISPR as the therapy, in which the company's proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellia's ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases.

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<span class="mw-page-title-main">He Jiankui affair</span> 2018 scientific and bioethical controversy

The He Jiankui affair is a scientific and bioethical controversy concerning the use of genome editing following its first use on humans by Chinese scientist He Jiankui, who edited the genomes of human embryos in 2018. He became widely known on 26 November 2018 after he announced that he had created the first human genetically edited babies. He was listed in the Time's 100 most influential people of 2019. The affair led to ethical and legal controversies, resulting in the indictment of He and two of his collaborators, Zhang Renli and Qin Jinzhou. He eventually received widespread international condemnation.

<span class="mw-page-title-main">CRISPR gene editing</span> Gene editing method

CRISPR gene editing 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 system. By delivering the Cas9 nuclease complexed with a synthetic guide RNA (gRNA) into a cell, the cell's genome can be cut at a desired location, allowing existing genes to be removed and/or new ones added in vivo.

Locus Biosciences is a clinical-stage pharmaceutical company, founded in 2015 and based in Research Triangle Park, North Carolina. Locus develops phage therapies based on CRISPR–Cas3 gene editing technology, as opposed to the more commonly used CRISPR-Cas9, delivered by engineered bacteriophages. The intended therapeutic targets are antibiotic-resistant bacterial infections.

bluebird bio, Inc., based in Somerville, Massachusetts, is a biotechnology company that develops gene therapies for severe genetic disorders.

BeiGene, Ltd. is a global oncology company that specializes in the development of drugs for cancer treatment. Founded in 2010 chief executive officer by John V. Oyler and Xiaodong Wang, the multinational company headquartered in Cambridge, Massachusetts has offices in North America, Europe, South America, Asia and Australia. BeiGene has a large presence in Chinese market. BeiGene has developed several pharmaceuticals, including tislelizumab, a checkpoint inhibitor, and zanubrutinib, a Bruton's tyrosine kinase inhibitor.

<span class="mw-page-title-main">CRISPR Therapeutics</span> Swiss-American biotechnology company

CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland. It was one of the first companies formed to utilize the CRISPR gene editing platform to develop medicines for the treatment of various rare and common diseases. The company has approximately 500 employees and has offices in Zug, Switzerland, Boston, Massachusetts, San Francisco, California and London, United Kingdom. Its manufacturing facility in Framingham, Massachusetts won the Facilities of the Year Award (FOYA) award in 2022. The company’s lead program, exagamglogene autotemcel, or exa-cel, was granted regulatory approval by the US Food and Drug Administration (FDA) in December 2023.

Excision BioTherapeutics is a biopharmaceutical company based in San Francisco focused on developing gene therapies against HIV infection.

Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease and transfusion-dependent beta thalassemia. It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.

Victoria Gray was the first patient ever to be treated with the gene-editing tool CRISPR for sickle-cell disease.

The Innovative Genomics Institute (IGI) is a nonprofit scientific research institute founded by Nobel laureate and CRISPR gene editing pioneer Jennifer Doudna and biophysicist Jonathan Weissman. The institute is based at the University of California, Berkeley, and also has member researchers at the University of California, San Francisco, UC Davis, UCLA, Lawrence Berkeley National Laboratory, Lawrence Livermore National Laboratory, Gladstone Institutes, and other collaborating research institutions. The IGI focuses on developing real-world applications of genome editing to address problems in human health, agriculture and climate change.

Samarth Kulkarni is a biotechnology executive. Kulkarni is the chief executive officer and chairman of the board at CRISPR Therapeutics. He has served as CEO of CRISPR Therapeutics since 2017. He was appointed to CEO and Chairman of CRISPR Therapeutics in 2023.

References

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