ReCode TherapeuticsCorporate Presentation39th JP Morgan Healthcare ConferenceJanuary 2021

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FIRST-IN-CLASS TARGETED, GENETIC MEDICINES FOR RESPIRATORY DISEASESPowered by Best-in-Class, Non-Viral LNP Delivery Platform

NON-VIRAL LNP PLATFORMBEST-IN-CLASS & NEW CLASS

4 Proprietary Classes of LNPs

• SORT LNPs – Best-in-Class with Tissue-Specific Delivery

• Engineered for Gene Delivery or Editing

• Broad Range of Therapeutic Cargos

Well Positioned to Execute

• Series A, $80M – March 2020• Strong Patent Protection (2035 plus)• Scalable Manufacturing

• Proven Management Team, Top Industry Investors and Advisors

CAPABILITIES

THERAPEUTIC PROGRAMS FOCUSED ON HIGH, UNMET NEEDS

FIRST-IN-CLASS

Genetic Respiratory Diseases

• Primary Ciliary Dyskinesia (PCD)

• Cystic Fibrosis (CF)

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Experienced Drug Development Leadership and Industry Advisors

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DAVID LOCKHART, PhDCEO & President

VLADIMIR KHARITONOV, PhDSenior Vice President, CMC

BRANDON WUSTMAN, PhDSenior Vice President of R&D

MICHAEL TORRES, PhDVice President, R&D, Texas Site Head

MANAGEMENT

BOARD OF DIRECTORS SCIENTIFIC ADVISORS and KOLs

DAVID LOCKHART,PhDReCode Therapeutics

MICHAEL TORRES, PhDReCode Therapeutics

HELEN S. KIM,MBAVida Ventures

PETER THOMPSON,MDOrbiMed

ED HURWITZ, JD, MBAMPM Capital

RA SESSION IITaysha Gene Therapies

ARTHUR JOHNSON,PhDProfessor Emeritus, Texas A&M

DANIEL SIEGWART,PhDAssociate Professor, UTSW

PHILIP THOMAS, PhDProfessor, UTSW

JULIE EASTLAND, MBACFO & COO

ROBERT BRIDGES, PhDProfessor, RFUM

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• RE-DOSABLE, TITRABLE

• NO AAV-RELATED SAFETY ISSUES

• LARGE PAYLOAD CAPACITY

• BROAD PAYLOAD DIVERSITY

• TARGETS SPECIFIC TISSUES AND CELLS

• SCALABLE MANUFACTURING

Best-in-Class and NEW Class of Non-Viral LNPs Customizable to Enable Targeted Delivery of Genetic Medicines

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ReCode’s LNPs enable precise gene replacement, silencing, and correction

ADVANTAGES DIFFERENTIATED

5-COMPONENT SORT TECHNOLOGY DELIVERS BEYOND THE LIVER

Lungs LiverSpleen

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MODALITY TARGET DELIVERY DISCOVERY PRECLINICAL PHASE 1/2 (SAD/MAD/OLE)

PCDPrimary Ciliary Dyskinesia

mRNA DNAI1 Inhaled

mRNA PCD gene 2 Inhaled

mRNA PCD gene 3 Inhaled

tRNA PCD nonsense Inhaled

CFCystic Fibrosis

tRNA CFTR Inhaled

mRNA CFTR Inhaled

Gene Correction

CFTR(F508del) IV; Lung-SORT

Platform Non-Viral LNPs Various

Organs/Tissues IV; Various

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Therapeutic Focus on Genetic Respiratory DiseasesAddress Underlying Genetic Cause in High Unmet Need Diseases with Potential to Transform Patient Lives

Primary endpoint (change in FEV1) with potential to demonstrate rapid improvement applies to current leads and future PCD and CF programs

IND in 2022

IND in 2022

Indications with established endpoints and clear path to human POC and approval

Lead Therapeutic ProgramsPCD and CF

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PCD: Addressing High Unmet Need with ReCode Solutions

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Targeted Delivery: Epithelial cells of the lower airways

Repeated delivery of DNAI1 mRNA to the epithelial cells of the conducting airways (e.g., trachea, bronchi, and bronchioles).

Cells targeted by LNP-formulated mRNA delivered as an aerosol using existing methods and devices.

DNAI1 is a component of the ciliary axoneme required for ciliary beating

DNAI1 mutations impair ciliary activity and result in loss of mucociliary clearance (MCC)

The SolutionThe Cause

Challenges

• Gene editing & gene therapy approaches not well-suited: ⎼ Frequent cell turnover (half-life of weeks to months)⎼ Basal (stem) cells difficult to access⎼ Need broad coverage with a single administration to

a diseased lung with accumulated mucus

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Rescued human DNAI1-deficient ciliated cells

Repeat administration well tolerated

Cellular uptake in the presence of mucus

Normal beat frequency recovered

Synchronized wave-like motion restored

Activity comparable to normal controls

Ciliary Function Restored in Human Cells and Delivered to the Lung as an Inhaled Aerosol

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Green: Acetylated alpha-tubulin (microtubules)

Red: HA tagged DNAI1

Human DNAI1 knock-out cells treated with a single dose of formulated DNAI1-HA and immuno-stained with anti-acetylated tubulin and anti-HA 72 hrs after dosing.

DNAI1 incorporated into cilia of human respiratory epithelial cells

Well Tolerated

Single administration, escalating dose well tolerated

Expression levels constant with repeat dosing

Histopathology Results Show No Findings

Whole-body images of mice treated with luciferase mRNA formulated in a ReCode LNP and nebulized using an

Aerogen mesh nebulizer

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PCD and CF Have Clear and Similar Paths for Clinical Development and Approval Endpoints

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Same primary endpoints, same clinical sites & PIs, similar paths to approval *Phase 1/2 study could be the basis of drug approval

Phase 1/2 Study*Indication / Inclusion criteria Primary Outcome Measures

− Safety and tolerability− Absolute change in percent predicted

FEV1

PCD Lead mRNA Program− Adults− Pathogenic mutation in DNAI1− FEV1 between 40% and 90%

Placebo Low Dose

High Dose

SAD ∙● MAD (6 mo.) ● OLE (6 mo.)

CF Lead tRNA Program− Adults− CFTR mutation R553X, R1162X, or G542X − FEV1 between 40% and 90%

− Safety and tolerability− Absolute change in percent predicted

FEV1

Placebo Low Dose

High Dose

SAD ∙● MAD (3 mo.) ● OLE (9 mo.)

Precedent for clinical path established by approved drugs for CF

CF Therapeutic ProgramFirst-in-Class tRNA Program Targets Subset of the CF Population with No Approved Therapy

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CF Patients with Nonsense Mutations Do Not Benefit FromApproved Therapies

Currently No approved drugs to treat patients with CFTR nonsense mutations

Global CF patients by mutation/genotype

WW Prevalence 70-100K*

*Patients identified and tracked through global CF registry (CFTR2.org database)

**Patient cells with R116X mutation not available; expected that NanoCorrector should work for all arginine nonsense mutations

Global Patient Population with Nonsense Mutations

Mutation *Number of patients Addressed by lead tRNA

G542X 3,475 Yes(demonstrated)

W1282X 1,552 No

R553X 1,298 Yes (demonstrated)

R1162X 612 Yes(expected**)

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Development Path

Development Path

Potential to be First-in-Class for targeted subset of patients

• tRNA program focuses on a subset of ~5% of patients who do not adequately respond to currently approved drugs

• Trial endpoints well established based on approved therapies

Prevalence

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Addressing High Unmet Need in CF

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tRNA NanoCorrectors Restore Full-length Functional CFTR ProteinCF patients with nonsense (premature stop) mutations have no therapeutic options (~5% of CF patients)

The SolutionThe Need

The Challenges

⎼ Delivery to the right cells in the airway epithelium ⎼ Specificity of read-through: consistent amino acid

insertion and recognition of true stops

Full-length functional protein

Gln Val Arg Val lle

NanoCorrector tRNAs “read through” the stop mutation and restore full-length functional protein

NormalGO

Truncated non-functional protein

Gln Val

Arg

Gln Val Arg Val lle

Wild-type mRNA

mRNA with nonsense mutation (C to U

resulting in “STOP”)

Restored full-length protein

LNP + tRNALead program (tRNA) supported by a $3.4M Therapeutic Development Award by the Cystic Fibrosis Foundation (since 2017)

Repeated delivery of tRNA to the epithelial cells of the conducting airways using existing inhaled aerosol delivery methods and devices

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tRNA Restores CFTR Function in Patient-derived Cells

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G542X/F508del hBEs(1) tRNA restores function in human bronchial cells (hBEs) derived from

patients with G542X and R553X nonsense mutations

Restores current in hBEs comparable to that seen with Lumacaftor (VX-809)

R553X/F508del hBEs

1 Heterozygote cell line from patients with G542X and F508del alleles: tRNA is affecting G542X while Lumacaftor is affecting F508del.

Non-Viral LNP Delivery Technology

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ZNPsZwitterionic amino lipid (ZAL)

lipid nanoparticles

CSALCationic quaternary ammonium

sulfonamide amino lipid

dLNPsDegradable and ionizable amino dendritic

lipid nanoparticles

Selective ORgan Targeting(SORT LNPs)

Utilizes organ targeting (SORT) molecules to redirect LNPs

• Hybrid 3-component LNPs; multi-organ tropism• Optimized for structured cargos such as tRNA and mRNA;

nebulized delivery to lungs

• Traditional 4-component LNPs with programmed degradability

• Optimized for RNA delivery and gene editing in the liver

• In vivo POC with repeated administrations shown for a broad range of targets

• Novel 5-component LNPs

• Tissue-specific delivery• Optimized for delivery of RNA,

gene editing agents, and protein delivery in the lungs and spleen (IV), muscle and brain (local).

Proprietary LNP Platform Based on Decades of Research and Deep Understanding of Lipid ChemistryPlatform Includes Thousands of Novel Lipids

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Delivery Differentiator: SORT LNPs Deliver a Broad Range of Payloads to Specific Tissues and Cell Types

16Cheng et al. Nature Nanotechnology 2020, 15, 313.

• Preferential delivery to lung cells• Rescue of CFTR chloride transport in hBEs• Delivery of CRISPR/Cas9 to endothelium and

epithelium via IV administration

• Delivery of CRISPR/Cas9 to the spleen• Editing in T cells, B cells, and macrophages• Editing observed in lymphatic tissue

• Gene editing in liver hepatocytes (>95%)• Complete knockout of PCSK9 (serum & tissue)• Delivery of Epo, Klotho and IL-10 mRNA

Exemplary Results (in vivo and in vitro)

Delivery of Luciferase mRNA

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Heart

Lungs

Liver

Spleen

Kidneys

PBSLung-specific

SORT LNPLiver-specific

SORT LNPSpleen-specific

SORT LNP

Delivery of CRISPR / Cas9 to tdTom Reporter Mice • Novel 5-component LNPs with unique

biodistribution and packaging properties

• Standard 4-component LNP technology limited to liver delivery

• SORT LNPs utilize novel in vivo mechanisms to access other tissues and cell types

• Applicable for gene editing by IV administration of Cas9 mRNA or protein (plus sgRNA and donor DNA)

Proprietary SORT Platform Enables Tissue-specific CRISPR/Cas9 Gene Editing

17Cheng et al. Nature Nanotechnology 2020, 15, 313.

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SORT unlocks the future of genetic medicinesImproved Lung Delivery: IV delivery to the lung epithelium bypasses mucus and may further improve delivery in patients with impaired lung functionOpportunities for Curative Therapies: Editing of long-lived stem cells (e.g., basal epithelial cells in the airways)

SORT Delivery - Potential to Transform Treatment of CF

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Long-term Mid-termNear-term

Initial focus on patients with nonsense mutations using a tRNA read-through therapy

CF tRNA for nonsense mutation population

Broaden CF franchise with mRNA platform

Potentially address the entire CF patient population with mutation-agnostic mRNA therapy

Enable durable CF gene correction with SORT

Transform CF landscape using Selective ORgan Targeting (SORT) delivery of gene editing components to progenitor (basal) cells for a long-lasting CFTR correction

NEAR-TERM FOCUS

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Differentiated Non-Viral LNP Technology Delivers Diverse Cargos Beyond the Liver

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ReCode Translate Bio Generation BioArbutus, Acuitas, Arcturus, BioNTech, CureVac, Ethris,

Genevant, Moderna

liver extrahepatic liver extrahepatic liver extrahepatic liver extrahepatic

RNA mRNA, siRNA, miRNA tRNA Gene editingco-delivery of Cas9 mRNA + sgRNA +/- donor DNA

Gene editingCas9 RNPs* +/- donor DNA Gene therapylarge DNA constructs Class of LNPs 3-, 4-component LNPs,5-component SORTs1,2 3-, 4-components LNPs

1 ligand conjugated LNPs for cell targeting 4-component LNPs

1Can be delivered as an inhaled aerosol2Thousands of proprietary lipids available

Opportunity and Milestones

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ReCode Integrated Platform

Broad Therapeutic and Delivery Platform Enables Partnering Opportunities Outside of Genetic Respiratory Disease

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Applicable Disease Areas

Genetic respiratory diseases

Chronic inflammatory lung disease, Pulmonary fibrosis

CNS (including gene editing)

Gene therapy targets requiring repeated administration

Immunodeficiency & autoimmune diseases

Immuno-oncology

In vivo CAR T-cell therapies

Infectious disease/vaccines

Liver fibrosis and cirrhosis

Metabolic disorders

Muscular dystrophies

Reactivation of tumor suppressors (oncology)

Diverse Cargo TypesCRISPR(RNA or

protein)/Cas

mRNA, tRNA, siRNA, miRNA

mABs, other proteins

Small molecules

Administration Route

IV

Inhaled aerosol

Intramuscular

Intrathecal

Target Organs/Tissue

Lungs

Spleen

Lymphatic Tissue

Liver

Brain / CNS

Muscle

LNP Classes3-component ZALs &

CSALs

4-component dendrimer (dLNPs)

5-component SORT

RECODE’s Focus

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Key Upcoming Milestones

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Primary Ciliary Dyskinesia - DNAI1 Therapeutic Program Estimated Timing

PCD – Formulation Selection in NHPs 1H 2021

PCD – Clinical Candidate Selection 2H 2021

PCD – IND-enabling Tox Studies 2H 2021 / 1H 2022

Platform Technology

SORT: Biodistribution and Tolerability Studies in NHPs 1H 2021

SORT: Delivery to Other Organs and Tissues; Gene Editing in Basal Cells Ongoing

Cystic Fibrosis Therapeutic Programs

CF – tRNA Formulation/Nebulization Optimization 2H 2021

CF – mRNA Formulation Selection 1H 2021

CF – Clinical Candidate Selection 2H 2021

CF – IND-enabling Tox Studies 2H 2021 / 1H 2022

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ReCode Delivering the Future of Genetic Medicines

CapabilitiesAbility to Deliver

• Drug Development Expertise

• Scalable Manufacturing

• Seasoned Management Team and Industry-leading Investors and Advisors

ProgramsFirst-in-Class

• Genetic Medicines for Patients with High Unmet Needs

• Drug Development and Approval Paths Clear

• Platform Technology to Expand Solutions for PCD and CF… and Beyond!

PlatformBest-in-Class, NEW Class

• Proprietary Non-viral LNP Technology [SORT]

• Targeted Delivery

• Diverse Cargos, Mixed Cargos

A Differentiated Therapeutic Company

Delivering the Future of Genetic Medicines

Thank You!

ReCode Therapeutics Corporate PresentationSlide Number 2Experienced Drug Development Leadership and Industry AdvisorsBest-in-Class and NEW Class of Non-Viral LNPs �Customizable to Enable Targeted Delivery of Genetic MedicinesTherapeutic Focus on Genetic Respiratory Diseases�Address Underlying Genetic Cause in High Unmet Need Diseases with Potential to Transform Patient Lives�Slide Number 6PCD: Addressing High Unmet Need with ReCode SolutionsCiliary Function Restored in Human Cells and Delivered to the Lung as an Inhaled AerosolPCD and CF Have Clear and Similar Paths for Clinical Development and Approval EndpointsSlide Number 10CF Patients with Nonsense Mutations Do Not Benefit From�Approved TherapiesAddressing High Unmet Need in CFtRNA Restores CFTR Function in Patient-derived Cells�Slide Number 14Proprietary LNP Platform �Based on Decades of Research and Deep Understanding of Lipid Chemistry�Platform Includes Thousands of Novel Lipids�Delivery Differentiator: SORT LNPs Deliver a Broad Range of Payloads to Specific Tissues and Cell Types�Proprietary SORT Platform Enables Tissue-specific CRISPR/Cas9 Gene Editing�SORT Delivery - Potential to Transform Treatment of CFDifferentiated Non-Viral LNP Technology Delivers Diverse Cargos Beyond the Liver Slide Number 20Broad Therapeutic and Delivery Platform Enables Partnering Opportunities Outside of Genetic Respiratory DiseaseKey Upcoming MilestonesSlide Number 23Slide Number 24Thank You!