Editas_Medicine

Editas Medicine

Editas Medicine

Discovery-phase pharmaceutical company

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]

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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]

References

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    "2022 Annual Report (Form 10-K)". U.S. Securities and Exchange Commission. February 22, 2023.
  2. [2]
    "The week in science: 5–11 February 2016". Nature. 530 (7589). Business: CRISPR goes public. 10 Feb 2016. Bibcode:2016Natur.530..134.. doi:10.1038/530134a.Open access icon
  3. [3]
    "Editas Wins FDA Approval for IND of CRISPR Treatment for LCA10". Genetic Engineering & Biotechnology News. 30 November 2018. Retrieved 20 August 2020.
  4. [4]
    Werley, Jensen (5 September 2019). "How Boulder biotech companies are putting Colorado on the gene-editing map". Denver Business Journal. Retrieved 27 October 2020.
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    Symington, Steve (20 August 2020). "Editas Medicine Remains on Track". The Motley Fool. Retrieved 20 August 2020.
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    "Who We Are". Editas Medicine. Retrieved 2022-02-25.
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    Rockoff, Jonathan (2015-06-29). "Why Gene-Editing Technology Has Scientists Excited". Wall Street Journal. ISSN 0099-9660. Retrieved 2021-05-27.
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    Isaacson, Walter (2021). The Code Breaker. Simon & Schuster. pp. 209–212. ISBN 978-1-9821-1585-2.
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    John Carroll (Nov 25, 2013). "Biotech pioneer in 'gene editing' launches with $43M in VC cash". FierceBiotech.
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    Regalado, Antonio (2015-11-05). "CRISPR Gene Editing to Be Tested on People by 2017, Says Editas". MIT Technology Review. Retrieved 2016-06-21.
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    Nowogrodzki, Anna (10 August 2015). "Gene-Editing Startup Raises $120 Million to Apply CRISPR to Medicine". MIT Technology Review. Retrieved 12 August 2020.
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    Loria, Kevin (12 April 2018). "Bill Gates says it would be a 'tragedy' to pass up a controversial, revolutionary gene-editing technology". Business Insider.
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    Pflanzer, Lydia (2 February 2016). "A Bill Gates-backed startup that wants to edit your genes just raised nearly $100 million". Business Insider.
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    Fidler, Ben (2 February 2016). "CRISPR Hits Wall Street as Editas Bags $94M in IPO". Xconomy. Retrieved 12 August 2020.
  15. [15]
    "Juno, Editas Launch Up-to-$737M+ Cancer Therapy Collaboration". Genetic Engineering & Biotechnology News. 27 May 2015. Retrieved 2016-02-11.Open access icon
  16. [16]
    Lombardo, Cara (2018-01-22). "Celgene to Buy Juno Therapeutics for $9 Billion". Wall Street Journal. ISSN 0099-9660. Retrieved 2018-01-22.
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    Bristol-Myers Squibb Completes Acquisition of Celgene, Creating a Leading Biopharma Company, PM BMS, November 20, 2019; retrieved May 20, 2020
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    Kuchler, Hannah (6 January 2020). "Crispr puts first human in-body gene editing to test". Financial Times.
  19. [19]
    "First CRISPR therapy dosed". Nature. 38 (4): 382. 7 April 2020. doi:10.1038/s41587-020-0493-4. PMID 32265555.
  20. [20]
    Sheridan, Cormac (14 December 2018). "Go-ahead for first in-body CRISPR medicine testing". Nature. doi:10.1038/d41587-018-00003-2. S2CID 91818387. Retrieved 21 December 2018.
  21. [21]
    "Editas Medicine Announces Positive Initial Clinical Data From Ongoing Phase 1/2 BRILLIANCE Clinical Trial Of EDIT-101 For LCA10". Editas Medicine. 29 September 2021. Archived from the original on 2021-09-29. Retrieved 5 November 2021.
  22. [22]
    Stein, Rob (4 March 2020). "In A 1st, Scientists Use Revolutionary Gene-Editing Tool To Edit Inside A Patient". NPR. Retrieved 12 August 2020.
  23. [23]
    Terry, Mark (4 March 2020). "Allergan and Editas Dose First Patient in Historic CRISPR Trial for Inherited Blindness". BioSpace. Retrieved 12 August 2020.
  24. [24]
    Fidler, Ben (7 August 2020). "Editas, AbbVie rework gene editing deal as pioneering CRISPR trial resumes". BioPharma Dive. Retrieved 12 August 2020.
  25. [25]
    Dearment, Alaric (22 January 2019). "Editas Medicine CEO steps down as company moves into product development stage". MedCity News. Retrieved 12 August 2020.
  26. [26]
    DeAngelis, Allison (6 August 2019). "Editas became Cindy Collins. CEO". Boston Business Journal.
  27. [27]
    "Editas Medicine Announces Appointment Of James C. Mullen As Chief Executive Officer" (Press release). Editas Medicine. 8 February 2021.
  28. [28]
    Keown, Alex (14 April 2022). "Gilmore O'Neill "Excited" to Take the Reins at Editas Medicine". BioSpace. Retrieved 2 June 2022.
  29. [29]
    Pickar-Oliver, Adrian; Gersbach, Charles A. (August 2019). "The next generation of CRISPR–Cas technologies and applications". Nature Reviews Molecular Cell Biology. 20 (8): 490–507. doi:10.1038/s41580-019-0131-5. ISSN 1471-0080. PMC 7079207. PMID 31147612.
  30. [30]
    Rees, Victoria (20 June 2019). "Experimental treatment for sickle cell disease success". Drug Target Review. Retrieved 20 August 2020.
  31. [31]
    Wong, Sandi (10 December 2019). "Editas shows better gene editing using Cas9 alternative for sickle cell, thalassemia". BioCentury. Retrieved 20 August 2020.
  32. [32]
    Carvalho, Joana. "FDA Clears Way for Trial of Gene Editing Therapy for Severe SCD". Retrieved 2021-03-29.
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