Editas Medicine

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.[3][4] Editas headquarters is located in Cambridge, Massachusetts and has facilities in Boulder, Colorado.[5][6][7]

Editas Medicine
TypePublic company
Nasdaq: EDIT
Russell 2000 Component
Industry
FoundedNovember 2013 (2013-11) in Cambridge, Massachusetts
FoundersJennifer Doudna, Feng Zhang, George Church (geneticist), David R. Liu, J. Keith Joung
Headquarters
Cambridge, Massachusetts
,
United States
Number of locations
2
Key people
Gilmore O'Neill (President and CEO)[1]
Total assetsUS$508,885,000 (2019)
Total equityUS$262,437,000 (2019)
Number of employees
208[2] (2020)
Websitewww.editasmedicine.com

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,[8] 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.[9][10][11]

In August 2015, the company raised $120 million in Series B funding from Bill Gates and 13 other investors.[12][13] it went public on 2 February 2016,[3] with an IPO that raised $94 million.[14][15]

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.[16] Juno was later acquired by Celgene,[17] which was in turn acquired by Bristol Myers Squibb.[18]

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.[19][11] On 30 November 2018, the FDA gave permission to start the trials, under the investigational name EDIT-101 (also known as AGN-151587).[20][21] In September 2021, a statement from Editas claimed that preliminary results from clinical trials were promising and support clinical benefits of EDIT-101 treatment.[22]

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.[23][24] 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.[25]

In 2019, the company was building new chemistry facilities in Boulder, Colorado.[6]

Katrine Bosley was CEO until 2019, when she was replaced by board member Cynthia Collins.[26][27] Collins was replaced in 2021 by James Mullen, who had been board chairman.[28] Gilmore O'Neill, former CMO of Sarepta Therapeutics, became CEO on June 1, 2022, with Mullen staying on as executive chairman of the board.[29]

Research

Editas works with two different CRISPR nucleases, Cas9 and Cas12a.[30]

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;.[31][32] 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.[33]

References

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