Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome
editing company focused on the development of curative therapeutics
using CRISPR/Cas9 technology both in vivo and ex vivo, announced
one oral presentation and four poster presentations were accepted
for the 27th Annual Congress of the European Society of Gene and
Cell Therapy (ESGCT) taking place October 22-25, 2019, in
Barcelona, Spain.
Intellia’s data includes important updates about the company’s
programs and platform development activities:
Oral Presentation:
“In Vivo Gene Knockout Followed by Targeted Gene
Insertion Results in Simultaneous Reduced Mutant Protein Levels and
Durable Transgene Expression”
Intellia will present data on its alpha-1 antitrypsin deficiency
(AATD) program, which uses a modular hybrid delivery system
combining lipid nanoparticle (LNP) encapsulated CRISPR/Cas9 with an
adeno-associated virus (AAV) donor DNA template. Intellia’s gene
knockout approach eliminates the production of the faulty PiZ
variant of the protein, while targeted insertion of a wild-type
gene copy facilitates production of a functional circulating
protein. This builds on Intellia’s similar approach for targeted
gene insertion of Factor 9, which achieved increased levels of
circulating human Factor IX protein through two months in non-human
primates and sustained through 12 months in mice.
Presenter: Anthony
Forget, Ph.D.Abstract number: OR48Session
5b: New delivery systems and
technologiesPresentation date/time: Friday,
October 25, 2019, 11:30 a.m. – 1:30 p.m.
CETLocation: Room 113-115
Poster Presentations:
“In Silico, Biochemical and Cell-Based Integrative
Genomics Identifies Precise CRISPR/Cas9 Targets for Human
Therapeutics”
This poster presentation will highlight Intellia’s approach to
assess off-target activity to identify highly specific CRISPR/Cas9
guides. Researchers demonstrated that potential off-target editing
profiles discovered through empirical data from biochemical
approaches were the most sensitive and accurate.
Presenter: Daniel
O’Connell, Ph.D.Poster ID Number:
P655Date: Wednesday, October 23, 2019
“Generation of a Library of WT1-Specific T Cell
Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute
Leukemia”
This poster presentation focuses on Intellia’s ongoing research
collaboration with IRCCS Ospedale San Raffaele to develop
CRISPR/Cas9-edited T cell therapies to address intractable cancers,
such as acute myeloid leukemia (AML). Researchers have successfully
established a protocol enabling consistent and efficient
tumor-specific TCR isolation and characterization from healthy
donors. Based on these results, Intellia has selected multiple lead
TCRs, which are undergoing development candidate evaluation.
Presenter: Erica
Carnevale, Ph.D., Ospedale San RaffaelePoster ID
Number: P111Date: Wednesday, October 23,
2019
“Engineering of Highly Functional and Specific
Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated
In-Locus Insertion Combined with Endogenous TCR
Knockout”
This poster presentation focuses on the company’s T cell
engineering technology, which is being applied in its Wilms’ Tumor
1 (WT1) lead ex vivo program. Intellia has identified an efficient
CRISPR/Cas9-mediated process that inserts tumor-specific TCRs with
high yield into the TRAC locus. Simultaneous knockout of the TRBC1
and TRBC2 loci substantially eliminates production of the
endogenous T cell receptors.
Presenter: Birgit
Schultes, Ph.D.Poster ID Number:
P162Date: Thursday, October 24, 2019
“CRISPR/Cas9-Mediated Gene Knockout to Address Primary
Hyperoxaluria”
This poster presentation will demonstrate the effects of
independent CRISPR/Cas9-mediated knockout of each of two target
genes involved in oxalate formation, lactate dehydrogenase A (LDHA)
and hydroxyacid oxidase 1 (HAO1), to address primary hyperoxaluria
type 1 (PH1).
Presenter: Sean Burns, M.D.Poster ID
Number: P552Date: Thursday, October 24,
2019
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on developing curative therapeutics using the CRISPR/Cas9
system. Intellia believes the CRISPR/Cas9 technology has the
potential to transform medicine by permanently editing
disease-associated genes in the human body with a single treatment
course, and through improved cell therapies that can treat cancer
and immunological diseases, or can replace patients’ diseased
cells. The combination of deep scientific, technical and clinical
development experience, along with its leading intellectual
property portfolio, puts Intellia in a unique position to unlock
broad therapeutic applications of the CRISPR/Cas9 technology and
create a new class of therapeutic products. Learn more
about Intellia Therapeutics and CRISPR/Cas9
at intelliatx.com and follow us on Twitter
@intelliatweets.
Forward-Looking Statements
This press release contains “forward-looking statements”
of Intellia Therapeutics, Inc. (“Intellia” or the
“Company”) within the meaning of the Private Securities Litigation
Reform Act of 1995. These forward-looking statements include, but
are not limited to, express or implied statements regarding
Intellia’s beliefs and expectations regarding its planned
submission of an IND application for NTLA-2001 in mid-2020; its
plans to generate preclinical and other data necessary to nominate
a first engineered cell therapy development candidate for its AML
program by the end of 2019; its plans to advance and complete
preclinical studies, including non-human primate studies for its
ATTR program, AML program and other in vivo and ex
vivo programs; develop our proprietary LNP/AAV hybrid delivery
system to advance our complex genome editing capabilities, such as
gene insertion; its presentation of additional data at upcoming
scientific conferences regarding CRISPR-mediated, targeted
transgene insertion in the liver of NHPs, using F9 as a model gene,
via the Company’s proprietary LNP-AAV delivery technology, and
other preclinical data by the end of 2019; the advancement and
expansion of its CRISPR/Cas9 technology to develop human
therapeutic products, as well as maintain and expand its related
intellectual property portfolio; the ability to demonstrate its
platform’s modularity and replicate or apply results achieved in
preclinical studies, including those in its ATTR and AML programs,
in any future studies, including human clinical trials; its ability
to develop other in vivo or ex vivo cell
therapeutics of all types, and those targeting WT1 in AML in
particular, using CRISPR/Cas9 technology; the impact of its
collaborations on its development programs, including but not
limited to its collaboration with Regeneron Pharmaceuticals,
Inc. or Ospedale San Raffaele; statements regarding the timing of
regulatory filings regarding its development programs; and the
ability to fund operations into the second half of 2021.
Any forward-looking statements in this press release are based
on management’s current expectations and beliefs of future events,
and are subject to a number of risks and uncertainties that could
cause actual results to differ materially and adversely from those
set forth in or implied by such forward-looking statements. These
risks and uncertainties include, but are not limited to: risks
related to Intellia’s ability to protect and maintain our
intellectual property position, including through our arbitration
proceedings against Caribou; risks related to Intellia’s
relationship with third parties, including our licensors; risks
related to the ability of our licensors to protect and maintain
their intellectual property position; uncertainties related to the
initiation and conduct of studies and other development
requirements for our product candidates; the risk that any one or
more of Intellia’s product candidates will not be successfully
developed and commercialized; the risk that the results of
preclinical studies will not be predictive of future results in
connection with future studies; and the risk that Intellia’s
collaborations with Novartis or Regeneron or its
other ex vivo collaborations will not continue or will
not be successful. For a discussion of these and other risks and
uncertainties, and other important factors, any of which could
cause Intellia’s actual results to differ from those contained in
the forward-looking statements, see the section entitled “Risk
Factors” in Intellia’s most recent annual report on Form 10-K as
well as discussions of potential risks, uncertainties, and other
important factors in Intellia’s other filings with
the Securities and Exchange Commission. All information in
this press release is as of the date of the release,
and Intellia undertakes no duty to update this information
unless required by law.
Intellia Contacts:
Media: Jennifer Mound Smoter Senior Vice
President External Affairs & Communications +1 857-706-1071
jenn.smoter@intelliatx.com
Investors: Lina Li Associate Director Investor
Relations +1 857-706-1612 lina.li@intelliatx.com
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