Intellia Therapeutics Corporate Overview August 2020 2 Intellia Therapeutics’ Legal Disclaimer This presentation contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia”, “we” or “our”) 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: receiving authorization to initiate clinical studies for NTLA-2001 for the treatment of transthyretin amyloidosis (“ATTR”) pursuant to its clinical trial application (“CTA”) or similar regulatory applications, and plans to dose the first patients with by year end 2020; plans to submit an investigational new drug (“IND”) application or similar clinical trial application for NTLA-5001, its first T cell receptor (“TCR”)-directed engineered cell therapy development candidate for its acute myeloid leukemia (“AML”) program in the first half of 2021; plans to submit an IND or similar clinical trial application for its hereditary angioedema (“HAE”) program in the second half of 2021; plans to advance and complete preclinical studies, including non-human primate studies for its HAE and other programs, and other animal studies supporting other in vivo and ex vivo programs, including its AML program; development of a proprietary LNP/AAV hybrid delivery system, as well as its modular platform to advance its complex genome editing capabilities, such as gene insertion; further development of its proprietary cell engineering process for multiple sequential editing; presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; ability to demonstrate its platform’s modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR, AML, and HAE programs, in any future studies, including human clinical trials; 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; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Novartis Institutes for BioMedical Research, Inc. (“Novartis”) or Regeneron Pharmaceuticals, Inc. (“Regeneron”), including its co-development programs for Hemophilia A and Hemophilia B; Regeneron’s ability to successfully co-develop products in the hemophilia A and B programs, and the potential timing and receipt of future milestones and royalties, or profits, as applicable, based on Intellia’s collaboration and co-development agreements with Regeneron and Novartis; and statements regarding the timing of regulatory filings and clinical trial execution, including dosing of patients, regarding its development programs; and the potential commercial opportunities, including value and market, for our product candidates; our expectations regarding our use of capital and other financial results during 2020; and our ability to fund operations at least through the next 24 months. Any forward-looking statements in this presentation 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 regulatory agencies’ evaluation of regulatory filings and other information related to its product candidates; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellia’s product candidates, including those that are co-developed, 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; 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 (“SEC”). All information in this presentation is as of the date of the presentation, and Intellia undertakes no duty to update this information unless required by law. 3 Our Mission Developing curative genome editing treatments that can positively transform the lives of people living with severe and life-threatening diseases 4 Modular Platform Drives Diversified Pipeline Full-Spectrum Genome Editing Company CORPORATE • Experienced management team • Well capitalized to drive pipeline forward PIPELINE • NTLA-2001 for ATTR: Submitted first CTA to initiate Phase 1 study; Intend to dose first patient by YE 2020 • NTLA-5001 for AML: Expect to submit IND in 1H 2021 for WT1-directed TCR T cell therapy • NTLA-2002 for HAE: Expect to submit IND in 2H 2021 PLATFORM • Rapid identification of development candidates • Precise knockout and/or insertion in vivo and ex vivo • Transient Cas9 expression via non-viral delivery ATTR: Transthyretin Amyloidosis AML: Acute Myeloid Leukemia HAE: Hereditary Angioedema 4 IND: Investigational New Drug or IND-equivalent WT1: Wilms’ Tumor 1 TCR: T Cell Receptor 5 Building a Full-Spectrum Genome Editing Company CRISPR creates the therapy CRISPR is the therapy Immuno-oncology Autoimmune diseases Genetic diseases Modular Platform LNP CELL RNA CRISPR/Cas9 In Vivo Ex Vivo LNP: Lipid Nanoparticle 6 CRISPR/Cas9 is an Effective Tool for Modifying the Genome KNOCKOUT Inactivation/deletion of disease-causing DNA sequence Insert new DNA sequence to manufacture therapeutic protein Correction of “misspelled” disease-driving DNA sequence REPAIR INSERT CRISPR/Cas9 7 Development Pipeline Fueled by Robust Research Engine * Lead development and commercial party ** Rights to certain in vivo targets *** Milestones & royalties CAR-T: Chimeric Antigen Receptor T cells HSC: Hematopoietic Stem Cells OSC: Ocular Stem Cells PROGRAM APPROACH Research Candidate Selection IND-Enabling Early-Stage Clinical PARTNER In Vivo: CRISPR is the therapy NTLA-2001: Transthyretin Amyloidosis Knockout NTLA-2002: Hereditary Angioedema Knockout Hemophilia A and B Insertion Research Programs Knockout, Insertion, Consecutive Edits Research Programs Various Ex Vivo: CRISPR creates the therapy OTQ923 / HIX763: Sickle Cell Disease HSC NTLA-5001: Acute Myeloid Leukemia WT1-TCR Solid Tumors WT1-TCR Undisclosed Programs Undisclosed Other Novartis Programs CAR-T, HSC, OSC UNDISCLOSED *** *** * ** * L E A D L E A D 8 LNP RNA CRISPR is the therapy In Vivo GENETIC DISEASES Strategic Advantages: Systemic non-viral delivery of CRISPR/Cas9 provides transient expression Potentially curative therapy from single course of treatment Permanent gain of function with targeted gene insertion 9 Modular Approach to Unlocking Treatment of Genetic Diseases PROPRIETARY LNP DELIVERY SYSTEM Transient expression Large cargo capacity Redosing capability ENABLES MULTIPLE EDITING STRATEGIES Restore Introduce functional DNA sequence INSERT CONSECUTIVE EDITING Any combination of knockout and insertion strategies + AAV Remove Knockout toxic or compensatory genes KNOCKOUT Remove / Restore + + 10 Modular In Vivo Genome Editing Approach Validated Across Multiple Targets Hem B: Circulating human FIX protein in NHPs at or above normal levels ATTR: >95% reduction of serum TTR sustained for a year in NHPs AATD: >98% reduction of disease-causing protein and sustained restoration of wild type AAT in serum to therapeutic levels in mice Restore Introduce functional DNA sequence INSERT CONSECUTIVE EDITING Any combination of knockout and insertion strategies + AAV Remove Knockout toxic or compensatory genes KNOCKOUT Remove / Restore + + AATD: Alpha-1 Antitrypsin Deficiency FIX: Factor IX Hem B: Hemophilia B NHP: Non-Human Primate 11 Transthyretin Amyloidosis (ATTR) Caused by accumulation of misfolded transthyretin (TTR) protein, which affects nerves, heart, kidneys and eyes NTLA-2001 in development for ATTR • Employs a knockout edit to reduce circulating TTR protein levels • Aims to address hATTR and wtATTR, both polyneuropathy and cardiomyopathy, with a single course of treatment 50,000 hATTR patients worldwide1 ~200-500K wtATTR patients worldwide2 2-15 years typical life expectancy from onset of symptoms1 Only chronic treatment options currently available 1 Ann Med. 2015; 47(8): 625–638. 2 Compiled from various sources hATTR: Hereditary ATTR wtATTR: Wild-Type ATTR 12 Control Lead LNP: Dose Level #1 (n=3) Lead LNP: Dose Level #2 (n=3) Therapeutically relevant serum TTR knockdown Single Dose ATTR: Sustained >95% Serum TTR Protein Reduction After a Single Dose in NHPs 13 ✓ Initiated IND-enabling toxicology studies for NTLA-2001 ✓ Commenced manufacturing for Phase 1 materials ✓ Submitted first CTA for NTLA-2001 to initiate Phase 1 study Dose first patient by YE 2020 ATTR: Advancing NTLA-2001 Toward the Clinic Achievements and Next Steps 14 Hereditary Angioedema <span >(NYSE:<a href='' title='Haemonetics Corporation'>HAE</a>)</span> Airway obstruction is particularly dangerous because it can cause death by asphyxiation Genetic disease characterized by overproduction of bradykinin, which leads to recurring, severe and unpredictable swelling in various parts of the body Attacks can occur every 7-14 days on average for untreated patients1 NTLA-2002 in development for HAE: • Employs a knockout edit of KLKB1 gene in hepatocytes • Aims to reduce plasma kallikrein activity to prevent excess bradykinin production leading to HAE attacks after a single course of treatment Only chronic treatment options currently available 1 in 50,000 HAE patients1 1 Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359:1027-1036 15 Reduced angioedema anticipated following knockout of KLKB1 Factor XII Factor XIIa • Kallikrein inhibitors are clinically validated in preventing HAE attacks • KLKB1 knockout is expected to be safe, as human nulls show no associated pathology* Knockout of KLKB1 Aims to Reduce Bradykinin Activity in People with HAE *Girolami B. et. al., Acta Haematol 2010;123:210–214 16 Achieved Sustained Therapeutically Relevant Kallikrein Activity Reduction After a Single Dose in NHPs *Banerji et al., NEJM, 2017 Kallikrein Activity Reduction Single Dose 0 5 10 15 20 25 30 35 40 45 50 0 20 40 60 80 100 120 140 160 180 Kallikrein Activity Reduction Time (Weeks) Plasma Kallikrein Activity (% of Basal) control Dose Level #1 (n=3) Dose Level #2 (n=3) Dose Level #3 (n=3) Control Dose Level #1 (n=3) Dose Level #2 (n=3) Dose Level #3 (n=3) Therapeutically relevant impact on attack rate* 17 LNP Delivery System: gRNA Reprograms Genetic Target Cas9 mRNA AAAA AAAA KLKB1 gRNA AAAA Target-specific gRNA TTR gRNA AAAA HAE: Rapid Path to NTLA-2002 Development Candidate Nomination HAE Program: Builds on ATTR program’s infrastructure, including modular LNP delivery system Applies insights gained from ATTR and other research programs to liver knockout target Platform advances expedite progression to NHP proof-of-concept gRNA: Guide RNA Expect to submit IND or IND-equivalent in 2H 2021 18 CRISPR creates the therapy Ex Vivo IMMUNO-ONCOLOGY / AUTOIMMUNE DISEASES Strategic Advantages: CRISPR/Cas9 enables precise genome engineering for creating cell therapies to treat IO and AI diseases Pursuing modalities, such as TCR, with broad potential in multiple indications Focused on recapitulating natural cell physiology CELL 19 TCR T Cell Modality Broadens Opportunity to Address Most Tumors • TCRs efficiently detect tumor antigens • Physiological signaling minimizes T cell exhaustion and immune toxicity • Healthy donor TCRs avoid reactivity against normal tissues • High-affinity TCRs can activate both cytotoxic and helper T cells Immunol Rev. 2019 Jul;290(1):127-147. Intracellular Tumor Antigens CAR-T: Limited to surface antigens TCRs: Recognizes both surface and intracellular antigens Selecting naturally-occurring, Total Addressable Tumor Targets high-affinity TCRs 20 CRISPR Engineering Overcomes Key Challenges of Traditional Approaches Key Challenges • Mutagenesis risk from random lentiviral insertion • Mixed expression of endogenous and tgTCR • Mispaired TCRs have unpredictable specificities and pose GvHD risk • Lower tgTCR expression per T cell leads to reduced efficacy Our Solution • Precise replacement of endogenous TCR with tgTCR • No insertional mutagenesis risk • Reduced risk of unwanted reactivity against normal tissues • High tgTCR expression per T cell leads to a more efficacious cell product Traditional tgTCR addition CRISPR/Cas9 tgTCR replacement Mixed TCRs tgTCRs only Heterogenous Cell Product Homogenous Cell Product tgTCR: transgenic therapeutic TCR 21 Based on FACs analysis High and Uniform Expression of tgTCR per T cell 100 80 60 40 20 0 Normalized tgTCR Expression (%) TCR A TCR B TCR C TCR D Removal of Endogenous TCR Prevents Mispairing 60 40 20 0 % Cells with mispaired TCRs TCR A TCR B TCR C TCR D ’s Approach (TRAC and TRBC KO + Insertion) TRBC KO only + Insertion Our Approach for TCR Replacement with Elimination of Endogenous TCRs Creates a Homogenous T Cell Product 22 Efficient multiplexed editing Higher T cell expansion Reduced translocations Untreated Standard Process Intel ia Process 0 20 40 60 80 100 %Editing Gene 1 Gene 2 Gene 3 Untreated Standard Process Intel ia Process 0 50 100 150 Fold Expansion Untreated Standard Process Intel ia Process 0 5 10 15 Cumulative Translocat oi n Events per 200 Cells Complex Translocations Reciprocal Translocations Translocations to other chromosomes Standard Process: Cas9/sgRNA RNP electroporation based on manufacturer’s instructions Proprietary Process Enables Multiple Sequential Edits With Minimal Translocations 23 Acute Myeloid Leukemia (AML) >21K new cases in the U.S. in 20191 >40K new cases in the 7MM2 in 20181 Cancer of the blood and bone marrow that is rapidly fatal without immediate treatment, and is the most common type of acute leukemia in adults1 <30% 5-year overall survival1 NTLA-5001 in development for AML • Engineer WT1-directed T cells capable of specifically killing AML blasts 1 NIH SEER Cancer Stat Facts: Leukemia – Acute Myeloid Leukemia (AML) 2 GlobalData EpiCast Report: Acute Myeloid Leukemia July 2017, 7MM: Seven Major Markets (includes U.S.) 24 Wilms’ Tumor 1 (WT1) is an Attractive Tumor Target WT1 is Overexpressed in >90% of AML Blasts • Independent of mutational status • Low normal tissue expression WT1 is Overexpressed in Variety of Solid Tumors • AML program provides foundation for expansion into solid tumors Normalized WT1 expression Normal expression Overexpression Cilloni et al., J Clin Oncol, 2009 Sugiyama et al., Jap J Clin Oncol, 2010 25 Engineered T Cells Capable of Specific and Potent Killing of WT1-Positive AML Blasts Lead WT1-Specific TCR Profile: Proprietary T Cell Engineering Process Yields: • Sourced from healthy donor T cells • HLA-A*02:01 restricted TCR • Displays high avidity for VLD* epitope ‒ VLD epitope is efficiently processed by tumor proteasome, and presented by AML blasts • Consistent high-level editing efficacy • High and homogeneous tgTCR expression • Cytotoxic and helper T cell response • No detectable bone marrow cell toxicity *VLD is the WT1(37-45) epitope VLDFAPPGA In collaboration with IRCCS Ospedale San Raffaele 26 ✓ Engineered WT1-specific T cells capable of specifically killing patient-derived AML blasts ✓ Nominated NTLA-5001 as development candidate Submit IND or IND-equivalent in 1H 2021 AML: Advancing NTLA-5001 Toward the Clinic Achievements and Next Steps 27 Multiple Workstreams to Advance Cell Therapy Efficacy in Solid Tumors Allogeneic Cell Source • Knock out MHC-I and MHC-II complexes • Address multiple surface protein signals • Achieve persistence in presence of natural killer cells Solid Tumor Efficacy • CRISPR screening to unravel targetable key regulators of T cell fitness in the tumor microenvironment Functional Modulation • Knock out and/or knock-in of key receptors, including checkpoint inhibitors, to modulate T cell functionality in multiple microenvironments 28 Upcoming Milestones: Driving Forward In Vivo and Ex Vivo Programs in 2020 o Submitted first CTA for NTLA-2001 to initiate a Phase 1 study ATTR o Dose first patient by YE 2020 AML o Presented preclinical data at scientific conference in 1Q 2020 o Submit IND or IND-equivalent for NTLA-5001 in 1H 2021 o Presented preclinical data at scientific conference in 1Q 2020 o Nominated NTLA-2002 as development candidate in 1H 2020 o Submit IND or IND-equivalent for NTLA-2002 in 2H 2021 In Vivo In Vivo Ex Vivo NTLA-2001 NTLA-5001 R&D Advancements o Present preclinical data at upcoming scientific conferences in 2020 ✓ ✓ HAE ✓ NTLA-2002 ✓Graphic Source: Intellia Therapeutics Inc.

Introduction: What is Intellia Therapeutics?

Intellia Therapeutics, Inc. (NASDAQ: NTLA) is a preclinical-stage genome editing company focusing on developing novel therapeutics using the genome-editing process known as CRISPR/Cas9, or Clustered, Regularly Interspaced Short Palindromic Repeats. Currently, Intellia is broadly focused on in-vivo, delivering directly to target cells in the body, and ex-vivo, developing a therapy from engineered human cells, with a global clinical focus (three countries in progress).

Founded in 2014, NTLA has since grown to revenues of $43 million (2019) with over 270 employees based in Cambridge, Mass. Although CRISPR/Cas9 genome editing technology is not yet clinically validated for human therapeutic use, the company is making strides in strategic markets to achieve the required approvals and is currently transitioning from pre-clinical to clinical in 2020-2021.

Products: Intellia has a pipeline of four therapies in the post-research phase with three realistically expected to show clinical aspirations before year-end 2021.

1. NTLA-2001 for transthyretin amyloidosis "ATTR": Submitted first CTA to initiate a Phase 1 study; Intend to dose first patient by YE 2020

2. NTLA-5001 for acute myeloid leukemia "AML": Expected to submit IND in 1H 2021 for WT1-directed TCR T-cell therapy

3. NTLA-2002 for hereditary angioedema "HAE": Expected to submit IND in 2H 2021

Customers/market: Currently, Intellia is pre-clinical, but the potential market for patients of each of the above products is as follows. For ATTR alone, the expected patient population is hard to estimate, but rival Akcea has found there to be an estimated 50,000 patients with hereditary ATTR amyloidosis worldwide and 200,000 patients with wild-type ATTR amyloidosis worldwide. Intellia additionally estimates there to be over 120 unique mutations from the traditional ATTR adding a layer of complexity to current therapies, but expanding the patient pool to 250,000-500,000. For AML globally, the expected afflicted population is very roughly estimated to be 450,000 with 20,000 new cases in the USA alone estimated for 2020. For HAE, there are roughly 150,000-250,000 people globally afflicted with this rare deficiency.

Management: As with most managerial teams in biotechnology, a key factor for differentiation is management's ability to not only master the science behind the development and testing of therapies but the ability to garner the right resources, partnerships, regulatory-approvals and broader connections for bringing therapies to market.

Chairman of the Board: Frank Verwiel, M.D. & MBA, brings 25 years of strategic, operational, and international experience in the pharma and biotech sector. He has served on the board of directors for AveXis Inc. (AVXS) until the Novartis acquisition in 2018 and on the board of Achillion Pharmaceuticals (ACHN) until the Alexion acquisition in 2020. He also led Aptalis Pharma Inc. as CEO until its 2014 acquisition by Forest Laboratories.

CEO: John Leonard, M.D., brings 30+ years of experience to Intellia, particularly from pharmaceutical R&D. His most recent position in 2013 was Chief Scientific Officer -CSO- and senior VP of R&D at AbbVie (ABBV) where he left to pursue his passion for turning the newly understood CRISPR/Cas9 technology into a therapeutic reality. He also serves on the Board of Directors for IQVIA (IQV), a health IT and clinical research firm, and IFM Therapeutics, a pharmaceutical company specializing in autoimmune diseases.

CSO: Laura Sepp-Lorenzino, Ph.D., brings 30 years of post Ph.D. experience having served for 14 years at Merck & Co (MRK) most recently as executive director and department head of RNA Therapeutics - Discovery Biology. She has also served as a VP at Alnylam Pharmaceuticals (ALNY) and Vertex (VRTX) and currently serves as a member of the Scientific Advisory Board at Thermo Fisher Scientific (TMO).

Financial position: Intellia in 2019 brought in $43.1 million (42% y/y) in collaboration revenue which includes upfront technology access payments for licenses, technology access fees, research funding, and milestone payments earned under the collaboration and license agreements with Novartis and Regeneron (partner since IPO). Intellia has not achieved profitability, of course, acting still in the pre-clinical phase with a promising pipeline. Gross profit loss in 2019 amounted to -$65 million with a total net loss of -$99 million (+16% y/y) after an R&D expense of -$108 million (+22% y/y). Although loss-making, the CEO did state in September at Baird’s 2020 Virtual Global Healthcare Conference that their 2Q 2020 cash position of $362 million is enough for 2+ years of operations.

Investment thesis: Intellia Therapeutics Inc. has been quiet for several years as they advanced the research needed for CRISPR/Cas9 applications and delivery. 2020 marks the first year that they will go clinical with a promising proprietary in vivo development candidate aiming to treat transthyretin amyloidosis (pat. pop. 250,000-500,000). By Dec. 2020, investors will know whether this candidate reaches Phase 1, and by Dec 21' investors will know whether Intellia's two other candidates remain on track for IND applications. Analysts have made their expectations clear for both a multiple expansion from 15.84x (Sep 2020) to 22.6x by Dec. 2020 and a revenue growth target of $61 million (+41% y/y). With the optimism of partner support from Regeneron and Novartis, Intellia is likely to perform well this year with an FYE price target of $30.55 (+30% upside).

Market/pipeline: Phase 1 for ATTR starting YE 2020

Although Intellia has a range of products and research opportunities, three are important for investors to understand in the short-term (pre-2022); however, the full pipeline is shown in the graphic below.Intellia Therapeutics Development Pipeline Fueled by Robust Research Engine 2020, 2021, 2022 Graphic Source: Intellia Therapeutics Inc. (Aug 2020)

NTLA-2001: This is Intellia's first proprietary in vivo development candidate aiming to treat transthyretin amyloidosis -ATTR-, a disease with a target population of both hereditary and wild-type ATTR roughly amounting to 250,000-500,000 globally. Intellia seeks to competitively treat ATTR by reducing the level of transthyretin protein production with a single course of treatment with life-long suppression, a promising first advancement with the use of CRISPR/Cas9 therapy for ATTR. Differentiation to other therapies is based out of significant patient treatment burden reduction over competitors currently only offering chronic treatment options, and potential economic advantages. Intellia is leading the co-development with Regeneron, a long-time Intellia partner.

Intellia announced in Aug 2020 that they submitted a strong first Clinical Trial Application -CTA- in the UK to initiate a small-patient population Phase 1 study with 1st patient dosing expected by Dec. 2020 with other agencies under consideration. Intellia has found good interest in the UK for gene-based medicine and is enthusiastic about clinical efficiency there. Intellia stated at the Baird Conference that they aim to particularly monitor off-target effects with CRISPR and acute toxicities that add a level of risk from the results of mouse/primate tests completed. Data is roughly expected in mid-to-late 2021.

Financially speaking, the global amyloidosis treatment market size (includes types: ATTR, AL, AA, Aβ2M, amongst others) was valued at $4.5 billion (2019) and expected to grow to $5.2 billion (2021) with an expected CAGR of 6.7% until 2025.

NTLA-2002: This is Intellia's development candidate for treating hereditary angioedema -HAE- and second in vivo knockout therapeutic candidate. HAE is a rare genetic disorder affecting roughly 150,000 to 250,000 people globally. Only chronic treatment options are currently available making a knockout edit of the KLKB1 gene particularly effective and competitive over current options. Additionally, NTLA-2002 builds on the ATTR program’s infrastructure, including the modular LNP delivery system.

Intellia stated in Aug. 2020 that they aim to submit an IND in 2H 2021.

Financially speaking, the global HAE market is estimated globally to be roughly $2.6 billion (2021) and is expected to grow with a CAGR of 8.2% until 2025.

NTLA-5001: This is Intellia's first ex vivo development candidate and Intellia seeks to treat acute myeloid leukemia -AML- by engineering autologous T-cell receptors -TCR- focusing on Wilm's Tumor 1 antigen which is often over-expressed in AML and other cancers (potential lateral therapeutic targets such as ovarian cancer, glioblastoma, lung cancer, and mesothelioma). This affects roughly 450,000 people globally with 20,000 new cases estimated for 2020 in the USA alone.

Intellia stated in Aug 2020 that they aim to submit an IND in 1H 2021.

Financially speaking, the global AML market was valued at $702 million (2018) and is expected to grow to $1.04 billion (2021) with a 14% CAGR until 2024.

Competition: Investors should be aware that several competitors do exist in the space including genome engineering companies focused on CRISPR/Cas9, such as Beam Therapeutics (BEAM), Caribou Biosciences, CRISPR Therapeutics (CRSP), Editas Medicine (EDIT), amongst others, companies focused on genome editing such as Allogene Therapeutics (ALLO), bluebird bio (BLUE), Cellectis S.A. (CLLS), amongst others and companies pursuing gene therapy in vivo and ex vivo including Asklepios Biopharmaceutical and Gilead Sciences(GILD), amongst others.

NTLA's Financial Position: Sufficient Cash & Good Partnership Revenues

Intellia Therapeutics 2020 Financial Position

Table Source: Self Created | Data Source: Seeking Alpha - NTLA

Revenue/cash flow: As seen above, Intellia's scientific foundation and collaboration revenue have grown significantly since its IPO with $0 revenue in 2014 to $43 million in revenue in 2019. 1H 20'/1H has shown a 36% increase in revenue due to advancements in Intellia's pipeline and expanding partnerships that are building on each therapy's infrastructure, primarily stemming from the foundational CRISPR/delivery work of the ATTR program with LNP delivery, a key advantage that Intellia is developing to maintain momentum. Cash flow from Operations -CFO- for the first time in the company's history has turned positive with 2Q 2020 bringing in $57.4 million in positive cash flow offsetting 1Q 20's -$38.5 million CFO.

Balance sheet composition: As seen below, Intellia has kept a stable balance sheet with a consistent cash buffer (>$270 million) managed mainly from equity offerings, as seen with the most recent June 2020 $115 million public common stock offering. Total debt is primarily (ca 70%) made up of capital leases and shows no serious risks. As stated above, although Intellia is loss-making which continually reduces cash-on-hand, the CEO did state in September that Intellia's 2Q 2020 cash position of $362 million is enough for 2+ years of operations which can relieve investor worries of a cash shortage or further share dilutions in 2020.

Balance Sheet NTLA - Intellia Therapeutics Inc.

Table Source: Self Created | Data Source: Seeking Alpha - NTLA

Valuation: Multiple Expansions & Revenue Growth

Evaluating Intellia must be done on a revenue basis, as all of their internal therapeutics (ex Novartis partnership) are pre-clinical with the outlying progress seen primarily in revenue adjustments from milestones and multiple expansions as progress through the 6-7 year clinical journey begins to take shape starting with Phase 1 dosing of NTLA-2001 beginning in December 2020.

Financial Projections and Valuation for Intellia therapeutics 2020 (FWD EV/Sales)

Table Source: Self Created | Data Source: Seeking Alpha - NTLA

Investors should understand that due to a large analyst following (9 in total), Intellia on average should preform relatively to mark with the above base-case assumptions (+30% upside) reflecting the average analyst expectations and current financial position.

Revenue is conservatively forecasted and multiples above 22x reflect slight optimism regarding staying on track for Phase 1 by Dec. 2020 without acute toxicities or large off-target effects from the resulting small population initial trial in the UK potentially published in mid-to-late 2021. The pessimistic case which outlines a no-growth 2020 (from Sep.), is based on a multiple not rewarded for progress towards initial Phase 1 which should encompass any slight delays less than 6-months (approximately), but an on-track basis for 2021 targets with NTLA-2002 and NTLA-5001. The optimistic case (+36% upside) reflects an expansion towards last June's sentiment on multiples primarily rewarded based on a revenue surprise +$6.74 million and an EPS improvement of $0.06 over analyst expectations.

ChartData by YCharts

Conclusion: Goldman Agrees, NTLA is a "Buy"

In conclusion, Intellia Therapeutics Inc. (NASDAQ: NTLA) is a pre-clinical biotech company going clinical this year with competitive therapeutics and sufficient support from long-standing partners such as Regeneron and Novartis. The company is sufficiently capitalized after the June 2020 capital raise of $115 million which is projected to enable NTLA-2001 into Phase 1 by Dec 20' and both NTLA-2002 and NTLA-5001 at IND status by Dec. 21'.

For long-term NTLA holders, this may be the time that the market begins to take notice of this relatively quiet biotech and for new investors, it may be time to watch trial results/regulatory progress in 2021. Either way, it seems not only is Goldman marking NTLA a "buy" at a $30 price target, but the broader market as a whole with the author expecting FYE 2020 to reach a price target of $30.55 (+30% upside).

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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