Intellia Therapeutics Announces First Quarter 2022 Financial Results and Highlights Recent Company Progress
05 Mai 2022 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing potentially curative therapies
leveraging CRISPR-based technologies, today reported operational
highlights and financial results for the first quarter ended March
31, 2022.
“Intellia is successfully executing on its 2022 strategic
priorities as we advance our proprietary CRISPR-based drug
discovery and development platform. We recently shared updated
interim data from our landmark study of NTLA-2001, which
demonstrated that treatment with NTLA-2001 in people with ATTR
amyloidosis with polyneuropathy was generally well-tolerated and
delivered rapid, consistent, dose-dependent reductions in serum
TTR. In addition to achieving a mean reduction of 93% at the 1.0
mg/kg dose, we were particularly pleased that reductions in serum
TTR levels persisted, further bolstering our confidence in
NTLA-2001 as a potentially durable, one-time treatment for ATTR
amyloidosis. In June, we plan to share additional durability data
from the dose-escalation portion of the polyneuropathy arm and data
supporting our fixed dose selection for Part 2,” said Intellia
President and Chief Executive Officer John Leonard, M.D. “Alongside
the progress of our lead program, we continued to advance our
second in vivo candidate, NTLA-2002, which benefits from the
modularity of our platform. We look forward to another important
clinical milestone in the second half of this year when we expect
to present initial data from the NTLA-2002 first-in-human study.
Finally, Intellia remains well-funded to drive forward our robust
portfolio and to support continued investment in platform
innovation as we build upon our leadership position in genome
editing.”
First Quarter 2022 and Recent Operational
Highlights
In Vivo Program Updates
Transthyretin (ATTR) Amyloidosis
- NTLA-2001:
NTLA-2001 is the first investigational CRISPR-based therapy to be
systemically delivered to edit genes inside the human body and has
the potential to be the first single-dose treatment for ATTR
amyloidosis. Delivered with the Company’s in vivo lipid
nanoparticle (LNP) technology, NTLA-2001 offers the possibility of
halting and reversing the disease by driving a deep, potentially
lifelong reduction in transthyretin (TTR) protein after a single
dose. NTLA-2001 is subject to a co-development/co-promotion
agreement between Intellia, the lead party for this program, and
Regeneron Pharmaceuticals, Inc.
- In February, Intellia presented
updated interim clinical data from 15 patients with hereditary ATTR
amyloidosis with polyneuropathy (ATTRv-PN) treated across four
single-ascending dose cohorts of the ongoing Phase 1 study.
Treatment with NTLA-2001 led to dose-dependent reductions in serum
TTR and achieved maximal reductions by day 28, with mean reductions
of 86% (n=3) and 93% (n=6) in the 0.7 mg/kg and 1.0 mg/kg dose
group, respectively. The maximum TTR reduction was 98% at 1.0
mg/kg. Mean serum TTR reductions were durable through the
observation period, with patient follow-up ranging from two to 12
months following a single dose. NTLA-2001 was generally
well-tolerated at all dose levels. The most frequent adverse events
included headache, infusion-related reactions, back pain, rash and
nausea.
- Based on these data, Intellia is
evaluating a fixed dose of 80 mg in Part 2 of the Phase 1 study,
which is expected to deliver a similar exposure to the 1.0 mg/kg
dose. The Company announced today that the first patient has been
dosed in Part 2, a single-dose expansion cohort, in the
polyneuropathy arm.
- The Company plans to present
additional interim data from Part 1, the single-ascending dose
portion, of the polyneuropathy arm at the European Association for
the Study of the Liver (EASL) International Liver Congress™ 2022,
to be held June 22-26.
- Intellia also continues to dose
patients in the cardiomyopathy arm of its expanded Phase 1 study,
which is currently evaluating NTLA-2001 in dose-escalation cohorts
of patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM).
The Company’s goal is to present the first interim data from the
cardiomyopathy arm in the second half of 2022. Enrollment across
both ATTRv-PN and ATTR-CM patient populations is expected to
complete in 2022.
Hereditary Angioedema (HAE)
- NTLA-2002:
NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery
technology to knock out the KLKB1 gene in the liver with the
potential to permanently reduce total plasma kallikrein protein and
activity, a key mediator of HAE. This investigational approach aims
to prevent attacks for people living with HAE by providing
continuous reduction of plasma kallikrein activity following a
single dose and to eliminate the significant treatment burden
associated with currently available HAE therapies.
- Intellia is progressing the
single-ascending dose portion of its Phase 1/2 study evaluating the
safety, tolerability and activity of NTLA-2002 in adults with Type
I or Type II HAE. The Company has completed dosing in the first
dose-escalation cohort (25 mg fixed dose) and has begun enrolling
patients in the second dose-escalation cohort (75 mg fixed
dose).
- The Company anticipates presenting
interim data in the second half of 2022 from the first-in-human
study, with initial results expected to characterize the emerging
safety and activity profile of NTLA-2002, and potentially
demonstrate preliminary proof-of-concept.
Alpha-1 Antitrypsin Deficiency (AATD)
- NTLA-3001 for associated
lung disease: NTLA-3001 is a wholly owned, first-in-class
CRISPR-mediated in vivo targeted gene insertion development
candidate for the treatment of AATD-associated lung disease. It is
designed with the aim to precisely insert a healthy copy of the
SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT)
protein, with the potential to restore permanent expression of
functional A1AT protein to therapeutic levels after a single dose.
This approach seeks to improve patient outcomes, including
eliminating the need for weekly IV infusions of A1AT augmentation
therapy or lung transplant in severe cases.
- Intellia is conducting
Investigational New Drug (IND)-enabling activities for NTLA-3001,
with plans to file an IND or IND-equivalent in 2023.
- NTLA-2003 for associated
liver disease: NTLA-2003 is a wholly owned in vivo
knockout development candidate for the treatment of AATD-associated
liver disease. It is designed to inactivate the SERPINA1 gene
responsible for the production of abnormal A1AT protein in the
liver. This approach aims to halt the progression of liver disease
and eliminate the need for liver transplant in severe cases.
- Intellia is initiating IND-enabling
activities for NTLA-2003.
Ex Vivo Program Updates
Acute Myeloid Leukemia (AML)
- NTLA-5001:
NTLA-5001 is an investigational autologous T cell receptor (TCR)-T
cell therapy engineered to target the Wilms’ Tumor 1 (WT1) antigen
for the treatment of all genetic subtypes of AML.
- In March, Intellia announced the
first patient was dosed in its Phase 1/2a study evaluating
NTLA-5001 for the treatment of AML. The Company continues to enroll
patients in the ongoing study.
- In March, Intellia announced that
the U.S. Food and Drug Administration (FDA) granted orphan drug
designation to NTLA-5001 for the treatment of AML.
CD30+ Lymphomas
- NTLA-6001:
NTLA-6001 is a wholly owned, allogeneic CAR-T development candidate
targeting CD30 for the treatment of CD30-expressing hematologic
cancers, including relapsed or refractory classical Hodgkin
lymphoma (cHL). NTLA-6001 is the first candidate developed using
Intellia’s proprietary allogeneic cell engineering platform, which
leverages a novel combination of sequential gene edits to protect T
cells from immune rejection by both host T and natural killer (NK)
cells.
- Intellia is initiating IND-enabling
activities for NTLA-6001.
- At the 2022 Keystone Symposia’s
Precision Genome Engineering Conference, Intellia presented
preclinical data leading to the development of NTLA-6001. The data
demonstrated that Intellia’s proprietary allogeneic solution
created T cells that not only avoided immune recognition by host
CD4 and CD8 T cells, but also were protected from NK cell-mediated
killing in in vitro and in vivo mouse models. Furthermore,
allogeneic T cells engineered sequentially with LNPs retained high
viability, cell expansion, memory phenotype, cytotoxic and cytokine
secretion characteristics.
Research and Corporate Updates
- Modular Platform and
Pipeline Expansion: Intellia is expanding its
industry-leading genome editing platform and scientific leadership
through editing, delivery and cell engineering innovations that may
enable broader in vivo and ex vivo applications.
- Intellia plans to advance at least
one additional new in vivo development candidate by the end of
2022.
- The Company plans to highlight
additional advances to its proprietary technology capabilities,
including both genome editing and delivery tools, at upcoming
scientific conferences in 2022.
- Collaboration
Updates
- In February, Intellia announced a
collaboration agreement with ONK Therapeutics Ltd. for the
development of allogeneic CRISPR-edited NK cell therapies for the
treatment of cancer.
- In January, Intellia announced a
licensing and collaboration agreement with Kyverna Therapeutics,
Inc. for the development of KYV-201, an allogeneic CD19 CAR-T cell
investigational candidate for the treatment of select autoimmune
diseases.
- Corporate Updates
- In May, Intellia announced the
appointment of Muna Bhanji, R.Ph., to its Board of Directors. Ms.
Bhanji brings more than 30 years of strategic and operational
experience in the biopharmaceutical industry to Intellia’s board,
including a proven track record of driving growth across a broad
portfolio of medicines and vaccines.
- In February, Intellia completed the
acquisition of Rewrite Therapeutics, Inc. (Rewrite), a private
biotechnology company focused on advancing novel DNA writing
technologies. Rewrite’s DNA writing technology may enable a range
of editing strategies, including targeted corrections, insertions,
deletions and the full range of single-nucleotide changes.
- In February, Intellia announced a
lease agreement to develop a 140,000-square-foot manufacturing
facility in Waltham, Massachusetts, to support the manufacturing of
key components for its CRISPR-based investigational therapies. The
new manufacturing facility will be Good Manufacturing Practice
compliant and support the preclinical through commercial supply for
key components of Intellia’s CRISPR-based therapies.
Upcoming Events
The Company will participate in the following events during the
second quarter of 2022:
- RBC Capital Markets Global
Healthcare Conference, May 17, New York
- Jefferies Healthcare Conference,
June 9, New York
- EASL International Liver Congress™
2022, June 22-26, London
Upcoming Milestones
The Company has set forth the following for pipeline
progression:
In Vivo
- NTLA-2001 for ATTR amyloidosis:
- Report additional interim data from
ATTRv-PN arm of Phase 1 study in June 2022
- Present interim data from ATTR-CM
arm of Phase 1 study in 2H 2022
- Complete enrollment of Phase 1
study for both ATTRv-PN and ATTR-CM subjects in 2022
- NTLA-2002 for HAE: Present interim
data from Phase 1/2 study in 2H 2022
- NTLA-3001 for AATD: File an IND or
IND-equivalent in 2023
- Advance at least one additional new
in vivo development candidate by the end of 2022
Ex Vivo
- NTLA-5001 for AML: Continue to
enroll patients in Phase 1/2a study in 2022
Modular Platform
- Advance additional novel platform
capabilities in 2022
First Quarter 2022 Financial Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $994.7 million as of March 31, 2022,
compared to $1.1 billion as of December 31, 2021. The decrease was
driven by cash used to fund operations of approximately $95.7
million as well as the acquisition of Rewrite for $45.0 million.
The decrease was offset in part by $38.9 million in net equity
proceeds raised from the Company’s “At the Market” (ATM) agreement
and $8.4 million in proceeds from employee-based stock plans.
- Collaboration
Revenue: Collaboration revenue increased
by approximately $4.8 million to $11.3 million during the first
quarter of 2022, compared to $6.4 million during the first quarter
of 2021. The increase was primarily driven by our joint venture
with AvenCell.
- R&D
Expenses: Research and development
expenses increased by $93.8 million to $133.1 million during the
first quarter of 2022, compared to $39.3 million during the first
quarter of 2021. This increase was primarily driven by $56.0
million of expense related to the acquisition of Rewrite, which
includes a $45.0 million upfront payment and $10.5 million related
to a potential stock-based earnout payment. The remaining $37.8
million was driven by the advancement of our lead programs,
research personnel growth to support these programs, and expansion
of the development organization.
- G&A
Expenses: General and administrative
expenses increased by $8.8 million to $22.4 million during the
first quarter of 2022, compared to $13.6 million during the first
quarter of 2021. This increase was primarily related to employee
related expenses, including stock-based compensation of $5.3
million.
- Net
Loss: The Company’s net loss was $146.9
million for the first quarter of 2022, compared to $46.2 million
during the first quarter of 2021.
Conference Call to Discuss First Quarter 2022
Results
The Company will discuss these results on a conference call
today, Thursday, May 5, at 8 a.m. ET.
To join the call:
- U.S. callers should dial 1-833-316-0545 and international
callers should dial 1-412-317-5726, approximately five minutes
before the call. All participants should ask to be connected to the
Intellia Therapeutics conference call.
- Please visit this link for a simultaneous live webcast of the
call.
A replay of the call will be available through the Events and
Presentations page of the Investors & Media section on
Intellia’s website at intelliatx.com, beginning on May 5, at 12
p.m. ET.
About Intellia TherapeuticsIntellia
Therapeutics, a leading clinical-stage genome editing company, is
developing novel, potentially curative therapeutics leveraging
CRISPR-based technologies. To fully realize the transformative
potential of CRISPR-based technologies, Intellia is pursuing two
primary approaches. The company’s in vivo programs use
intravenously administered CRISPR as the therapy, in which
proprietary delivery technology enables highly precise editing of
disease-causing genes directly within specific target tissues.
Intellia’s ex vivo programs use CRISPR to create the therapy by
using engineered human cells to treat cancer and autoimmune
diseases. Intellia’s deep scientific, technical and clinical
development experience, along with its robust intellectual property
portfolio, have enabled the company to take a leadership role in
harnessing the full potential of genome editing to create new
classes of genetic medicine. Learn more at intelliatx.com. Follow
us on Twitter @intelliatx.
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
concerning: its ability to successfully extend its leadership
position and harness the full potential of genomic medicines to
bolster its genome editing capabilities and pipeline; the safety,
efficacy, success and advancement of its clinical programs for
NTLA-2001 for the treatment of transthyretin amyloidosis, NTLA-2002
for the treatment of hereditary angioedema, and NTLA-5001 for the
treatment of acute myeloid leukemia pursuant to its clinical trial
applications (“CTA”) and IND submissions, including the expected
timing of data releases, regulatory filings, and the initiation,
enrollment, and completion of clinical trials; the advancement of
development candidates such as NTLA-3001 for the treatment of
alpha-1 antitrypsin deficiency-associated lung disease, NTLA-2003
for AATD-associated liver disease and NTLA-6001 for CD30+
lymphomas, including the success of its IND-enabling studies; the
ability to generate data to initiate clinical trials and the timing
of CTA and IND submissions; the expansion of its CRISPR/Cas9
technology and related technologies to advance additional
development candidates and timing expectations of advancing such
development candidates; its ability to maintain and expand its
related intellectual property portfolio; expectations of the
potential impact of the coronavirus disease pandemic, including the
impact of any variants, on strategy, future operations and timing
of its clinical trials; its ability to successfully employ
Rewrite’s DNA writing technology in enabling a range of precise
editing strategies and the resulting therapeutic potential of such
technology; the potential benefits and contributions of the new
manufacturing facility in Waltham to the Company’s CRISPR-based
therapies; and its ability to optimize the impact of its
collaborations on its development programs, including, but not
limited to, its collaboration with Regeneron Pharmaceuticals, Inc.
and their co-development program for ATTR amyloidosis and its
collaborations with ONK Therapeutics Ltd. and Kyverna Therapeutics,
Inc.
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 its
intellectual property position; risks related to Intellia’s
relationship with third parties, including its licensors and
licensees; risks related to the ability of its licensors to protect
and maintain their intellectual property position; uncertainties
related to the authorization, initiation and conduct of studies and
other development requirements for its product candidates,
including uncertainties related to regulatory approvals to conduct
clinical trials; 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 or
clinical studies will not be predictive of future results in
connection with future studies; the risk that clinical study
results will not be positive; the risk that the Rewrite acquisition
may not result in the development of a writing technology or
otherwise result in enabling precise editing strategies; and the
risk that Intellia’s collaborations with Regeneron or its other
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 (“SEC”). 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.
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INTELLIA THERAPEUTICS, INC. |
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CONSOLIDATED STATEMENTS OF OPERATIONS
(UNAUDITED) |
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(Amounts in thousands, except per share data) |
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Three Months Ended March 31, |
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2022 |
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2021 |
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Collaboration revenue |
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$ |
11,252 |
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$ |
6,445 |
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Operating expenses: |
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Research and development |
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133,095 |
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39,276 |
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General and administrative |
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22,403 |
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13,594 |
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Total operating expenses |
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155,498 |
|
|
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52,870 |
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Operating loss |
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|
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(144,246 |
) |
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(46,425 |
) |
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Other (expense) income, net: |
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Interest income |
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540 |
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220 |
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Loss from equity method investment |
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(2,745 |
) |
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- |
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Change in fair value of contingent consideration liability |
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(421 |
) |
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- |
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Total other (expense) income, net |
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(2,626 |
) |
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220 |
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Net loss |
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$ |
(146,872 |
) |
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$ |
(46,205 |
) |
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Net loss per share, basic and diluted |
$ |
(1.96 |
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$ |
(0.69 |
) |
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Weighted average shares outstanding, basic and diluted |
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74,751 |
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67,183 |
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INTELLIA THERAPEUTICS, INC. |
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CONSOLIDATED BALANCE SHEET DATA (UNAUDITED) |
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(Amounts in thousands) |
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March 31,2022 |
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December 31,2021 |
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Cash, cash equivalents and marketable securities |
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$ |
994,737 |
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$ |
1,086,049 |
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Total assets |
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1,202,788 |
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1,294,464 |
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Total liabilities |
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249,034 |
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254,220 |
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Total stockholders' equity |
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953,754 |
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1,040,244 |
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Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.comLina
LiDirector, Investor Relations and Corporate
Communications+1-857-706-1612lina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communications+1-917-640-7930media@intelliatx.com
mcrenson@tenbridgecommunications.com
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