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Editas Medicine, Inc. (EDIT)

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Last Post: 8/31/2017 8:28:46 AM - Followers: 25 - Board type: Free - Posts Today: 0

We are a leading genome editing company dedicated to treating patients with genetically defined diseases by correcting their disease-causing genes. We believe that we have entered a new era of genomic medicine as the growth of genomic information in recent decades has significantly expanded the understanding of genetically defined diseases. A new technology known as CRISPR (clustered, regularly interspaced short palindromic repeats)/Cas9 (CRISPR associated protein 9) has the potential to achieve precise, directed changes in DNA. The confluence of these two streams of scientific endeavor, understanding genetic defects and having the tools to be able to address them, creates the opportunity for us to achieve a longstanding goal of medicine: to treat the root causes of diseases at the genetic level. While genetic defects are now recognized as the causes of many diseases, the vast majority of these diseases lack effective treatments. Of the estimated 6,000 diseases that are known to be caused by genetic mutations, we believe fewer than 5% are served by approved therapies. Our mission is to translate the promise of our science into a broad class of transformative genomic medicines to benefit the greatest number of patients. We are developing a proprietary genome editing platform based on CRISPR/Cas9 technology. CRISPR/Cas9 uses a protein-RNA complex composed of the Cas9 enzyme bound to a guide RNA molecule designed to recognize a particular DNA sequence. The RNA molecule guides the Cas9 complex to the location in the genome that requires repair. Once there, the complex makes a specific cut in the DNA, ultimately triggering the cell's DNA repair machinery to address the genetic defect. Our platform consists of four interrelated components: nuclease engineering, delivery, control and specificity, and directed editing. These components are designed to develop medicines that specifically address a wide variety of genetic targets, reach the site of disease safely and effectively, tightly and specifically control the editing process, and drive the right kind of genetic repair. Our preclinical drug discovery platform uses the flexibility of CRISPR/Cas9 technology to enable rapid reprogramming of the Cas9-guide RNA complex with the potential to direct it to almost any site in the human genome. Using this platform, we aim to develop and advance a broad range of therapies for genetically defined diseases. Our product development strategy is to target genetically defined diseases with an initial focus on debilitating illnesses where there are no approved treatments and where the genetic basis of disease is well understood. We are advancing over a dozen discovery research programs that we have selected based on our proprietary assessment criteria. Our most advanced research program is designed to address Leber Congenital Amaurosis type 10, or LCA10, a specific genetic form of progressive blindness with no available therapies or potential treatments in clinical trials in either the United States or European Union. The localization of LCA10 disease in the eye allows us to efficiently apply our technology in a context that is confined and relatively uncomplicated compared to many of the systemic illnesses we also anticipate treating over time. We have tested combinations of Cas9 and guide RNA pairs in cells that were taken from patients with a specific mutation that causes LCA10 and demonstrated restoration of normal messenger RNA and protein expression, suggesting that we successfully corrected the LCA10 gene defect in these cells. We aim to initiate a clinical trial in this program in 2017. We believe achievement of proof-of-concept in a disease of the eye has the potential to validate our platform technology, including its potential application to other organs and diseases. Our additional research programs address genetic, infectious, and oncologic diseases of the liver, lung, blood, eye, and muscle. For example, we believe our genome editing technologies have the potential to improve the characteristics of cellular therapies, including engineered T cells to treat cancer. To realize this potential, in May 2015, we entered into a collaboration with Juno Therapeutics, a leader in the emerging field of immuno-oncology. We believe that our genome editing technology has the potential to improve T cell persistence and overcome signals in the tumor microenvironment that reduce T cell activity. In an in vitro study under this collaboration, Cas9-guide RNA complexes directed against what we believe is an important T cell target gene demonstrated approximately 90% editing on average. By working with Juno Therapeutics, we hope that together we will be able to discover and develop the next generation of engineered T cell therapies that have the potential to substantially advance the field of cancer immunotherapy. We believe this collaboration exemplifies our strategy of selectively establishing alliances with leaders in their fields to realize the full therapeutic potential of genome editing. Our company was founded by world leaders in genome editing who have collectively made many fundamental discoveries in the field and have enabled the translation of CRISPR from its origins in bacterial systems to its application in mammalian cells. Through their service as consultants and advisors, our founders were instrumental in defining the initial scientific vision for our company. Among our founders, Drs. Feng Zhang, George Church, David Liu, and J. Keith Joung continue to provide important scientific guidance and insights to us through ongoing consulting and advisory arrangements. Their discoveries, along with inventions by scientists at our company, have led to our broad portfolio of intellectual property, including the patent estates licensed from those founders' institutions. Our portfolio includes 21 issued U.S. and European patents and over 200 pending patent applications. We believe the breadth and depth of our patent estate is a substantial asset and has the potential to provide us with a durable competitive position in the marketplace. The lifeblood of our company is exceptional scientists and company-builders with experience across leading biopharmaceutical companies and academic research laboratories. Our company is distinguished by our leaders' substantial experience in translating groundbreaking scientific platforms into therapeutic products and product candidates at many successful biopharmaceutical companies. We believe that our team and our culture are critical to our success, and we are building a company with the values and people needed to realize the potential of our platform and develop medicines for patients with many different genetically defined diseases. Every decade over the past 40 years, an important class of medicines has emerged, such as recombinant proteins, monoclonal antibodies, and RNA-based drugs. These new categories of medicines have brought forth important therapies for previously untreated diseases. In our view, genome editing with CRISPR/Cas9 has the potential to be one of the next major new categories. At Editas Medicine, we believe we can make that potential a reality.
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EDIT News: Current Report Filing (8-k) 12/07/2017 04:24:53 PM
EDIT News: Prospectus Filed Pursuant to Rule 424(b)(5) (424b5) 12/07/2017 04:20:59 PM
EDIT News: Editas Medicine, Inc., Announces Pricing of $50 Million Offering of Common Stock 12/06/2017 07:00:00 AM
EDIT News: Prospectus Filed Pursuant to Rule 424(b)(5) (424b5) 12/05/2017 04:21:14 PM
EDIT News: Editas Medicine, Inc., Announces Offering of Common Stock 12/05/2017 04:17:01 PM
PostSubject
#39   prediction def has come to pass, and then some... halpern6 08/31/17 08:28:46 AM
#38   Jumped in today will see 20s again soon enough. longhorn4lif 06/01/17 10:56:59 AM
#37   Regarding competing CRISPRP technology utilization...European Patent Agency awar Justfactsmam 03/30/17 08:53:53 AM
#36   Excited To Be Invested In EDIT , ACTcrazy 03/29/17 10:56:00 PM
#35   CRISPR Turbocharges CAR T Cells, Boosts Cancer Immunotherapy Justfactsmam 03/29/17 08:08:18 AM
#34   "Upgrade this AM by Chardan...Price Target: $65.00 (triple Justfactsmam 03/28/17 07:37:03 AM
#33   EDIT Shorts look likely they drove down SP Justfactsmam 03/27/17 09:58:27 AM
#32   AGN, EDIT ink drug-discovery collaboration for eye diseases: #msg-129491806. DewDiligence 03/14/17 09:30:45 AM
#31   Editas bags $90M in gene editing R&D pact ciciagt 03/14/17 09:22:38 AM
#30   Sad that money can even corrupt the USPTO. Leezee Life Strategies 02/16/17 09:38:46 AM
#29   Thanks, yesterday, when NTLA and CRSP were Dan_P 02/16/17 09:00:20 AM
#28   Congrats to all holding EDITAS, the licensing is ACTcrazy 02/16/17 01:53:38 AM
#27   This article summary is quite good and tells ciciagt 02/15/17 11:14:19 PM
#26   Would certainly think the NTLA crew will ciciagt 02/15/17 02:59:41 PM
#25   What are the implications for CRSP and NTLA? Dan_P 02/15/17 02:04:00 PM
#24   Patent Office Hands Win in CRISPR Battle to ciciagt 02/15/17 01:31:20 PM
#23   EDITAS has the cpf1 patent improvements going for ACTcrazy 02/09/17 11:16:47 PM
#22   EDITAS has the cpf1 patent improvements going for ACTcrazy 02/09/17 11:16:47 PM
#21   Thanks for the other article. Here's a ciciagt 02/09/17 03:07:34 PM
#20   Oh i know. It's a crazy case. BioIT.com Leezee Life Strategies 02/09/17 03:04:12 PM
#19   I know, but look at the article I ciciagt 02/09/17 03:02:19 PM
#18   I really don't see they're gonna win. NT*A Leezee Life Strategies 02/09/17 01:57:26 PM
#17   CRISPR Heavyweights Battle in U.S. Patent Court ciciagt 02/09/17 01:13:07 PM
#16   The CRISPR patent fight could open the flood ciciagt 02/09/17 12:16:18 PM
#15   Not sure. Still too early IMO. Not in yet. lgonber 02/05/17 12:07:27 PM
#14   EDIT on the move, Market cap now exceeds ACTcrazy 02/05/17 01:22:27 AM
#13   COMPARING GENE EDITING COMPANIES lgonber 04/15/16 06:55:50 AM
#12   CRISPR is huge! InvestKid36 03/15/16 01:46:06 PM
#10   Most people on ihub would rather trade subpenny protagonist12 03/12/16 12:23:27 PM
#9   How is no one talking about this stock? PennyJoe 03/11/16 01:55:27 PM
#8   Two IPOs to Kick off 2016 in Biotech nyctraydr 02/10/16 01:51:14 PM
#7   Boom time T695 02/03/16 01:40:35 PM
#6   The demand for EDIT seems to be sparking protagonist12 02/03/16 11:31:13 AM
#5   In for a few shares at 16.15 T695 02/03/16 11:21:24 AM
#4   Loaded up EDIT @ 16.06 crudeoil24 02/03/16 11:10:58 AM
#3   Editas Medicine Announces Pricing of Initial Public Offering protagonist12 02/02/16 11:51:38 PM
#2   Ditto. Hopefully the broader biotech sector doesn't drag T695 02/02/16 09:04:14 PM
#1   EDIT Will be watching this IPO closely tomorrow. protagonist12 02/02/16 05:05:32 PM
PostSubject