Intellia Therapeutics Announces First Quarter 2019 Financial Results and Company Update Remains on track to submit investigational new drug application in 2020 for its first systemically delivered CRISPR/Ca

Intellia Therapeutics Announces

First Quarter 2019 Financial Results and Company Update

CAMBRIDGE, Mass., May 2, 2019 (GLOBE NEWSWIRE) Intellia Therapeutics, Inc. (NASDAQ:NTLA), reported operational highlights and financial results

for the first quarter ended March 31, 2019. Additionally, the Company highlighted important corporate milestones for 2019.

productive start in support of our mission to advance genome editing to treat a range of severe and life-threatening diseases. We are excited by our achievements in gene knockout and insertion across both our in vivo and engineered cell

therapy efforts. In particular, we presented data at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy showing unprecedented CRISPR-mediated, targeted gene insertion in the

liver of non-human primates, achieving normal circulating human levels of protein production, said Intellia President and Chief Executive Officer John Leonard, M.D. These achievements highlight

Intellia s leadership in CRISPR/Cas9 genome editing as we advance our pipeline towards the clinic. Looking ahead, we remain on track to file an IND application next year for NTLA-2001, our lead in vivo candidate for the treatment of

transthyretin amyloidosis, and expect to nominate a development candidate in our first engineered cell therapy program for acute myeloid leukemia by year-end.

First Quarter 2019 and More Recent Operational Highlights

Today, Intellia announced plans to begin

IND-enabling toxicology studies of NTLA-2001 in mid-2019 and that it remains on track to submit an IND application in 2020. NTLA-2001 is being co-developed with Regeneron Pharmaceuticals, Inc. (Regeneron), with Intellia as the lead development and commercialization party.

LDHA and HAO1 are enzymes involved in oxalate production. In people with PH1, mutations in a specific liver enzyme cause the production of a

surplus of oxalate, which can combine with calcium to form insoluble deposits in the kidney and throughout the body, leading to damage of the kidneys, heart, eyes and skeletal system. An approximate 30% reduction in urinary oxalate in patients with

PH1 is considered to be therapeutically relevant (source: Nephrology Dialysis Transplantation 1999; 14:2556-2558). In collaboration with the University of Alabama at Birmingham, Intellia researchers found that a CRISPR-mediated knockout of the

Ldha gene in a PH1 mouse model disrupts LDHA protein production and reduces urinary oxalate levels by 63%. Researchers also observed that a CRISPR-mediated knockout of the Hao1 gene disrupts glycolate-to-glyoxylate conversion, resulting in a urinary oxalate level reduction of 57% in a PH1 mouse model. In each individual knockout approach, these reduced levels of urinary oxalate were sustained for

Today s presentation, titled CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria, is

accessible through the Events and Presentations page of the Investor Relations section of Intellia s website.

The Company has set forth the following for 2019 pipeline progression:

First Quarter 2019 Financial Results

Intellia expects that its cash, cash equivalents and marketable securities as of March 31, 2019, as well as technology access and funding from Novartis

and Regeneron, will enable Intellia to fund its anticipated operating expenses and capital expenditure requirements into the first half of 2021. This expectation excludes any potential milestone payments or extension fees that could be earned and

distributed under the collaboration agreements with Novartis and Regeneron or any strategic use of capital not currently in the Company s base-case planning assumptions.

Conference Call to Discuss First Quarter 2019 Earnings

The Company will discuss these results on a conference call today, May 2, 2019, at 8 a.m. ET. The investor presentation may be downloaded starting at 7:30

a.m. ET from the Events and Presentations page of the Investor Relations section of Intellia s website at intelliatx.com.

A replay of the call will be available on Intellia s

website, beginning on May 2, 2019 at 12 p.m. ET.

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

Forward-Looking Statements

This press release contains

forward-looking statements of Intellia Therapeutics, Inc. ( Intellia ) 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 ability to advance and expand the CRISPR/Cas9 technology to develop into human therapeutic products, as well as our intellectual property portfolio; our ability to achieve stable or effective genome

editing; our ability to administer our CRISPR/Cas9 product candidates; the potential timing and advancement of our preclinical studies, including continuing non-human primate studies for our ATTR program and

other programs, animal studies for our AML program, and clinical trials; the timing and potential achievement of milestones to advance our pipeline including nominating development candidates and filing INDs; the modularity of our platform and our

ability to replicate or apply results achieved in our preclinical studies, including those in our ATTR and AML programs, in any future studies, including human clinical trials; the potential development of our proprietary LNP/AAV hybrid delivery

system to advance our complex genome editing capabilities; the potential development of other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9

technology; our ability to initiate and conduct successful IND-enabling toxicology studies of NTLA-2001, our lead ATTR development candidate, and subsequently submitting an IND application in 2020 that will be

accepted by the regulatory agencies; our intent to present additional data regarding CRISPR-mediated knockout of each of two targets of interest, Ldha and Hao1, via the Company s proprietary LNP delivery technology, resulting in the reduction

of oxalate excretion, and other preclinical data during 2019 or thereafter our plans to commence manufacturing efforts in 2019; actions by government agencies; our growth as a company and the anticipated contribution of

the members of our board of directors and our executives to our operations and progress; the impact of our collaborations on our development programs; the potential timing of regulatory filings

regarding our development programs; our expectations regarding our uses of capital, expenses, future accumulated deficit and other financial results during the first quarter of 2019; and our ability to fund operations into the first half of

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; 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 THERAPEUTICS, INC.

CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED)

(Amounts in thousands, except per share data)

INTELLIA THERAPEUTICS, INC.

CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)

(Amounts in thousands)

Jennifer Mound Smoter

Senior Vice President

External Affairs & Communications