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Editas Medicine, Inc. (EDIT)

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Last Post: 4/15/2016 6:55:50 AM - Followers: 11 - 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
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

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: Statement of Changes in Beneficial Ownership (4) 12/08/2016 05:10:19 PM
EDIT News: Statement of Changes in Beneficial Ownership (4) 12/08/2016 05:10:19 PM
EDIT News: Statement of Changes in Beneficial Ownership (4) 12/07/2016 04:46:23 PM
EDIT News: Statement of Changes in Beneficial Ownership (4) 12/07/2016 04:44:38 PM
EDIT News: Statement of Changes in Beneficial Ownership (4) 11/28/2016 04:39:00 PM
#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