Jump to content

Intellia Therapeutics

From Wikipedia, the free encyclopedia

This is an old revision of this page, as edited by Amakuru (talk | contribs) at 09:21, 25 April 2018 (Reverted edits by Amakuru (talk) to last version by Kvng). The present address (URL) is a permanent link to this revision, which may differ significantly from the current revision.

  • Comment: Normally stock market noise is considered WP:ROUTINE and does not establish notability but there's enough smoke here to establish notability even if there is no fire. Waiting for speedy deletion of existing WP:REDIRECT before this can be accepted. ~Kvng (talk) 02:51, 24 April 2018 (UTC)
  • Comment: Does not yet have an approved product. Please do not try to get this article into WP until it does. DGG ( talk ) 10:00, 22 January 2018 (UTC)

Intellia Therapeutics

Intellia Therapeutics
Type
Public
Traded as
NASDAQ: NTLA
Industry
Biotherapeutics
Founded
2014
Headquarters
Cambridge, MA, United States
Website
www.intelliatx.com
Senior Leaders
John Leonard MD (President and Chief Executive Officer); Graeme Bell (Executive Vice President, Chief Financial Officer); Thomas Barnes (Senior Vice President, Innovative Sciences); José Rivera (Executive Vice President, General Counsel)

Intellia Therapeutics is a biotherapeutics company developing therapies using the CRISPR gene editing systems.[1] The aim is to make permanent or non-permanent edits to disease-associated genes with a single treatment course.[2]

CRISPR gene editing technology was described as “the breakthrough of the year” by the journal Science[3] and the “biggest biotechnology discovery of the century” by MIT Technology Review.[4]

The company was founded in May 2014, backed by Atlas Venture, and scientists who pioneered and shaped the field of CRISPR biology.[5] The company’s scientific founders include Jennifer Doudna Ph.D., Rodolphe Barrangou, Ph.D, Rachel Haurwitz Ph.D., Luciano Marraffini, Eric Sontheimer, Ph.D., and Derek Rossi, Ph.D.

Intellia’s Series A round investment in November 2014 raised $15 million.[6] In September 2015, a Series B round secured $70 million.[7] In May 2016, Intellia announced the closing of its initial public offering which raised approximately $112.1 million.[8][9][10]

In December 2016, the company moved to its new 80,000 sq. ft. laboratory and office space in Cambridge, Massachusetts.[11]

Disease Areas

Intellia and its partners have described preclinical programs focused on liver disorders.[12] The company is validating its approach in this organ and says it is using the knowledge gained to expand application of CRISPR therapies to other organs and diseases. Disclosed in vivo programs are in transthyretin amyloidosis (ATTR) (with partner, Regeneron), hepatitis B, alpha-1 antitrypsin deficiency (AATD), and inborn errors of metabolism primary hyperoxaluria (Ph-1). Disclosed ex vivo applications include Novartis-partnered programs in hematopoietic stem cells (HSC) and chimeric antigen receptor T cells (CAR-T).

Intellia has access to Novartis’ lipid nanoparticle (LNP) library to develop a unique therapeutic delivery system, a key differentiator of its in vivo programs versus peers.[13][14]

Data Presented

In August 2016, Intellia disclosed preclinical data showing in vivo gene editing using CRISPR/Cas9 and the company’s proprietary LNP delivery system.[15] The data demonstrated the clinical potential of LNP-delivered CRISPR/Cas9.

Intellia was the first company to offer in vivo data demonstrating efficient delivery and rapid clearance of CRISPR/Cas9 components by LNPs in a target organ.[16]

In 2017, three further presentations supported the approach.[16][17] For the first time, data showed high levels of consistent, dose-dependent gene editing. Intellia also demonstrated another first – 97 percent reduction in serum transthyretin (TTR) protein (associated with liver disease), following a single dose, using LNP-delivered CRISPR/Cas9.[15] Both studies were in rodent models. Intellia recently reported that one year after receiving a single dose of LNP-formulated CRISPR/Cas9, rodents retained TTR reduction and gene editing levels, demonstrating consistent results and durability of the CRISPR/Cas9 system.[16][17]

Later in 2017, Intellia released top-line data from studies in non-human primates. These demonstrate, for the first time, liver genome editing using CRISPR/Cas9 delivered through a liquid nanoparticle (LNP) system, including observing over 20 per cent editing in certain animals. Redosing produced increased levels of editing, with no adverse events observed. Further studies will optimize editing efficiency, explore dosing parameters, and lead toward the selection of a development candidate.

eXtellia

eXtellia Therapeutics is a division of Intellia, focused on ex vivo applications of CRISPR/Cas9 therapies.[18] For the ex vivo approach, cells are removed from a patient or donor, and edited in culture. The engineered cells are then infused into the patient, where they address the disease. Intellia is working to simplify this procedure, to enhance outcomes and improve the patients’ experience.

In June 2017, eXtellia and San Raffaele University and Research Hospital in Italy announced a research collaboration to discover innovative tools to target tough-to-treat cancers.[19][20]

References

  1. ^ "Gene editing comes of healthcare age". Financial Times. February 16, 2015.
  2. ^ "Why Gene Editing Has Scientists Excited". Wall Street Journal. June 28, 2015.
  3. ^ "Breakthrough of the Year: CRISPR makes the cut". Science. December 17, 2015.
  4. ^ "Who Owns the Biggest Biotech Discovery of the Century?". MIT Technology Review. December 4, 2014.
  5. ^ "Novartis joins Atlas in launching a CRISPR Cas biotech with a $15M bankroll". Fierce Biotech. November 18, 2014.
  6. ^ "Intellia Therapeutics Raises $15M Series A for Gene Editing Therapies". Wall Street Journal. November 18, 2014.
  7. ^ "Intellia raises $70 million for gene-editing treatments". Boston Globe. September 1, 2015.
  8. ^ "Form 10-K". United States Securities and Exchange Commission.
  9. ^ "Stock in Biotech Company Intellia Rises 23% After Upsized IPO". Wall Street Journal. May 6, 2016.
  10. ^ "Shares of biotech firm Intellia Therapeutics soar in debut". New York Post. May 7, 2016.
  11. ^ "Exclusive: Fast-growing gene-editing biotech expanding near Kendall Square". Boston Business Journal. January 22, 2016.
  12. ^ "Intellia Therapeutics' Preclinical Data Show Continued Progress in In Vivo Gene Editing With Systemic Lipid Nanoparticle Delivery of CRISPR/Cas9 components". Nasdaq Global Newswire. August 18, 2016.
  13. ^ "First of its Kind Biotech Deal From Novartis, Intellia". Nature. January 30, 2015.
  14. ^ "Novartis secures first CRISPR pharma collaborations". Xconomy. January 7, 2017.
  15. ^ a b ' We’re nearly ready to use CRISPR to target far more diseases, https://www.newscientist.com/article/2149129-were-nearly-ready-to-use-crispr-to-target-far-more-diseases/, New Scientist, October 2, 2017
  16. ^ a b c ' Intellia R&D head says new gene-editing data shows path to human trials, http://www.bizjournals.com/boston/news/2017/03/08/intellia-r-d-head-says-new-gene-editing-data.html, Boston Business Journal, March 8, 2017
  17. ^ a b ' Intellia Therapeutics Demonstrates Pioneering CRISPR/Cas9 Genome Editing Efficiency Data Using Lipid Nanoparticle Delivery Technology, https://globenewswire.com/news-release/2017/03/08/933303/0/en/Intellia-Therapeutics-Demonstrates-Pioneering-CRISPR-Cas9-Genome-Editing-Efficiency-Data-Using-Lipid-Nanoparticle-Delivery-Technology.html, Nasdaq, March 8, 2017
  18. ^ "Intellia Therapeutics Forms New Division eXtellia for Joint Programs with Novartis". BIO.IT World. January 14, 2016.
  19. ^ "Intellia Therapeutics and San Raffaele University and Research Hospital to Combine CRISPR/Cas9 Genome Editing with Enhanced Cell Therapies to Fight Cancer". Ospedale San Raffaele website.
  20. ^ "Intellia, San Raffaele Partner on CRISPR/Cas9-Edited Anticancer T-Cell Therapies". Generic Engineering and Biotechnology News. June 6, 2017.
Retrieved from "https://en.wikipedia.org/w/index.php?title=Intellia_Therapeutics&oldid=838164432"