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© 2020 ActoBio Therapeutics, Inc. All rights reserved. THE ACTOBIOTICS ® PLATFORM designing living microbe-based therapeutics engineered to treat a wide set of diseases Corporate presentation - Pieter Rottiers, PhD, Chief Executive Officer, Director - JANUARY 2020

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Page 1: THE ACTOBIOTICS PLATFORM designing living microbe-based ... · designing living microbe-based therapeutics engineered to treat a wide set of diseases ... with high unmet needs

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THE ACTOBIOTICS ® PLATFORMdesigning living microbe-based therapeutics

engineered to treat a wide set of diseases

Corporate presentation - Pieter Rottiers, PhD, Chief Executive Officer, Director - JANUARY 2020

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Some of the statements made in this presentation are forward-looking statements.These forward-looking statements are based upon our current expectations andprojections about future events and generally relate to our plans, objectives andexpectations for the development of our business.

Although management believes that the plans and objectives reflected in or suggestedby these forward-looking statements are reasonable, all forward-looking statementsinvolve risks and uncertainties and actual future results may be materially different fromthe plans, objectives and expectations expressed in this presentation. All informationin this presentation is as of the date marked on the cover page, and ActoBioTherapeutics™ undertakes no duty to update this information unless required by law.

© 2019 ActoBio Therapeutics, Inc. All rights reserved. ActoBio Therapeutics™ is sharing the following materials for informational purposes only. Such materials do not constitutean offer to sell or the solicitation of an offer to buy any securities of ActoBio Therapeutics™. Any offer and sale of Intrexon’s securities will be made, if at all, only upon theregistration and qualification of such securities under all applicable federal and state securities laws or pursuant to an exemption from such requirements. The attachedinformation has been prepared in good faith by ActoBio Therapeutics™. However, ActoBio Therapeutics™ makes no representations or warranties as to the completeness oraccuracy of any such information. Any representations or warranties as to ActoBio Therapeutics™ shall be limited exclusively to any agreements that may be entered into byActoBio Therapeutics™ and to such representations and warranties as may arise under law upon distribution of any prospectus or similar offering document by ActoBioTherapeutics™.

TrademarksIntrexon, ActoBio Therapeutics, ActoBiotics, Powering the Bioindustrial Revolution with Better DNA, and Better DNA are trademarks of Intrexon and/or its affiliates. Other names may be trademarks of their respective owners.

FORWARD-LOOKING STATEMENTS

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ACTOBIO THERAPEUTICS™ - KEY INVESTMENT HIGHLIGHTS

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• ActoBiotics® is a fully integrated, cost-effective & unique food microbe-based delivery platform for therapeutics, with potential for superior efficacy and safety through oral or local targeted delivery

• L. lactis is designed to perform specific biological interventions

• Accelerates development and validated regulatory path for new IND candidates: <2 yrs from inception to IND achieved with AG013 for oral mucositis

• Strong R&D engine and large collection of strains, with potential for further development

PLATFORMPRODUCTPORTFOLIO

EXPERIENCE

• Straightforward and reliable cGMP manufacturing process

• IP portfolio across the technology platform:• >250 granted patents • >50 pending patent applications

• Extensive regulatory acceptance of platform and applications

• Experienced leadership team with unique expertise in bacterial engineering and strong scientific, regulatory and clinical backgrounds

• Three clinical stage programs: One inPhase IIb and two programs in Phase Ib/IIa, with other programs close to clinical stage

• Broad therapeutic application across a wide set of diseases with strong growth dynamics

Topline datafrom 2 clinical programs

expected in 2020

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ACTOBIOTICS® - AN INTEGRATED MICROBIAL DRUG PLATFORM

L. lactis essential gene removed

Therapeutic gene inserted

¹ Steidler et al., 2003, Nature Biotechnology.

• L. lactis has a long history of safe use in human nutrition

• Non-colonizing, non-human commensal species allowing for control of dosage and timing of exposure

• No known pathogenicity• Oral delivery platform demonstrated safe

for use in humans

• Construction strategy allows for minimum genetic modifications

• Engineered through chromosomal integration of single or multiple genetic elements through precise, targeted double homologous recombination

• Host is engineered for environmental containment¹, preventing accumulation of bacteria outside the body

• Engineering process established in-house to allow for continual creation of new drug candidates

• Industrial microorganism used for large scale food production

• cGMP manufacturing process established

• Following fermentation, the modified bacteria (ActoBiotics™) are freeze-dried and packaged

GENETICENGINEERING

SCALABLEMANUFACTURING

SAFETY

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ACTOBIOTICS™

CLINICAL AND

DEVELOPMENT

PIPELINE

I M M U N O T H E R A P Y

INDICATION PRECLINICALDISCOVERY PHASE I PHASE IIEST 2019 GLOBAL

ADDRESSABLE POPULATION*

PRODUCTCANDIDATE

D E S E N S I T I Z A T I O N

pre-AGFood, seasonal, perennial and venom allergy

51 K

5The ActoBiotics® platform can be easily adapted to deliver a wide range of additional therapeutic candidates.

I N B O R N E R R O R S O F M E T A B O L I S M

pre-AG Phenylketonuria

ANTICIPATEDMILESTONES

pre-AG Skin Disease

pre-AG Immuno-oncology

AG018 > 4 MChronic

Rhinosinusitis

AG020 12.3 MInflammatoryBowel Disease

AG013 850 KOral Mucositis Interim results1H 2020

T O L E R A N C E I N D U C T I O N

Type I Diabetes 132 KAG019 Interim resultsQ3 2020

Celiac Disease 1.9 MAG017 Start Phase Ib/IIa2020

*Total for US, EU, Japan. Sources on final slide.

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KEY TEAM MEMBERS

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PIETER ROTTIERS, PhD - CEO, Director• Extensive experience in drug discovery and development using the ActoBiotics platform, including over thirteen years of leadership and

managerial experience in biopharmaceuticals• Held several R&D and executive management functions at ActoGeniX and ActoBio Therapeutics, and progressed four ActoBiotics

products from discovery research into clinical trials• PhD in Biotechnology, University of Ghent, Belgium

LOTHAR STEIDLER, PhD - CTO• Leader of ActoBiotics technology, published author in Science and Nature Biotechnology, co-founder of ActoGeniX• Extensive experience of genetic engineering of L. lactis• Led and continues to lead all molecular biology at ActoGeniX and ActoBio Therapeutics.• PhD in Biotechnology, University of Ghent, Belgium

LUC VAN FRAEYENHOVEN - CFO• Finance executive with more than twenty years of experience in international finance functions• Held various senior finance positions including venture capital investor, CFO of the Ghent Volvo plant, and CFO of Volvo Europe• Commercial engineer and Master in Financial Management

STEFAN HERDINIUS, MSc, MBA - VP and Head of Strategic Drug Development• Accomplished leader with more than twenty years of experience in global early to late stage drug development• Instrumental in the development of new drugs in various therapeutic areas, including autoimmune diseases, neurology, virology and more,

at UCB Biopharma, Tibotec and Merck Sharp & Dohme• MSc in Pharmacy, Uppsala University, Sweden and MBA, Vlerick Business School, Belgium

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ACTOBIO THERAPEUTICS™: WORLD’S LEADING FOOD-MICROBE BASED VENTURE

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Well defined regulatory path

Scalable manufacturing

process

Multiple candidates ready for clinical development

Global IP Position

Experienced leadership team

Pioneering a new class of food-microbe

based genetically engineered

therapeutic agent

More than 20 years of scientific,

regulatory, and clinical experience

Designed to be safe & cost-

effective

Streamlined R&D engine allowing

continuous creation of new candidates

Strong pipeline with disease-modifying

potential and clinical stage programs

Fast path to IND &

clinic (<2yrs)

Therapeutic applications across

major diseaseswith high unmet

needs

ActoBiotics®A Unique and Superior

Delivery & Development Platform

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DELIVERY OF ACTOBIOTICS™ THERAPEUTICS

1. ActoBiotics™ freeze dried and inserted into enteric coated capsule

2. Capsule opens and releases ActoBiotics™ directly at the disease target

3. ActoBiotics™ produces therapeutic agent locally at target site (e.g. in GI tract)

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THE ACTOBIOTICS® PLATFORM COMPARED TO EXISTING TREATMENTS

IndicationTreatment withBiologicals

ROA (Biological) ActoBiotics™ ROA

Oral Mucositis Palifermin (HSCT) Injected Buccal rinse

IBD Anti-TNF Injected Oral capsule

Type 1 DiabetesInsulin (Replacement) Injected

Oral capsule (Immunotherapy)

Chronic Rhinosinusitis

Anti-IL5 / anti-IL4R InjectedNasal spray

Celiac Disease - - Oral capsule

ActoBiotics™ are as effective as injected biologicals

even at 1/1000th

the dose

32

50.000

1 1.000

ActoBiotics

Purified(topical)

TFF1 dose (ng) delivered for efficacy

ActoBiotics™

• Can be delivered in an oral capsule or in a topical solution

• No known toxicity or immunogenicity observed

• Targeted delivery to mucosa

• Simultaneous delivery of multiple proteins

Classic Protein Biologics

• Intravenous administration

• Systemic toxicity, immunogenicity observed

• Limited tissue penetration (notably in the gut)

• Single protein administration

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A unique delivery platform precisely tailored for specific disease modification

Specifically designed to target disease areas with high unmet need

Rapid development of new candidates

Targeted delivery via oral capsule, oral rinse or topical solution

Potential for agents with superior safety and efficacy

Robust and scalable manufacturing process

ACTOBIOTICS™MICROBE-BASED

BIOPHARMACEUTICALSfor expression and local

delivery of therapeutics at disease sites including the intestine, the mouth and

the nasopharynx

Feel free to visit our website www.actobio.com

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the game changer

INDUSTRIEPARK 7C BUILDING D 9052 GHENT BELGIUM

PHONE +32 9 277 11 77 [email protected]

Janu

ary

20

, 20

20

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SOURCES AND REFERENCES

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Figures for total addressable population and peak sales are based on data from the following sources, for US, EU and Japan, with some assumptions applied internally.

Type 1 Diabeteshttps://www.cdc.gov/diabetes/pdfs/library/diabetesreportcard2014.pdf http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0011501 https://www.idf.org/e-library/epidemiology-research/diabetes-atlas/19-atlas-6th-edition.html https://link.springer.com/article/10.1007/s00125-006-0213-8#Tab1 https://data.worldbank.org/indicator/SP.POP.0014.TO.ZS https://data.oecd.org/pop/young-population.htm

Celiac disease http://www.cureceliacdisease.org/wp-content/uploads/FactSheet1_Overview-of-Celiac-Disease.pdf https://www.nature.com/ajg/journal/v108/n5/full/ajg201360a.html https://www.sciencedirect.com/science/article/abs/pii/S0198885998000081 http://www.thesuperallergycookbook.com/PDF/CeliacDiseaseFacts.pdf

Oral mucositishttp://oncolex.org/Head-and-Neck-cancer/Diagnoses/Oral-cavity/Background/Prognosis https://www.uptodate.com/contents/epidemiology-and-risk-factors-for-head-and-neck-cancer https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/cancer-treatment-and-survivorship-facts-and-figures/cancer-treatment-and-survivorship-facts-and-figures-2014-2015.pdf http://www.onclive.com/publications/oncology-live/2014/july-2014/study-finds-mouth-rinse-alleviates-oral-mucositis-symptoms-in-head-and-neck-cancers https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3662500/

Chronic Rhinosinusitishttps://www.cdc.gov/nchs/fastats/sinuses.htm https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4386276/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4702060/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4939220/ https://www.aaaai.org/global/latest-research-summaries/Current-JACI-Research/chronic-rhinosinusitis-US

Inflammatory Bowel Diseasehttps://www.cdc.gov/ibd/ibd-epidemiology.htm http://www.sciencedirect.com/science/article/pii/S1873994613000305 https://link.springer.com/article/10.1007/s00535-009-0057-3 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4863044/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4737871/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5127933/

Allergieshttps://web.archive.org/web/20160909210149/ http://www.niaid.nih.gov/topics/environmental-allergies/Pages/immunotherapy.aspxhttps://www.npr.org/sections/health-shots/2013/03/26/175348886/allergy-drops-under-the-tongue-may-be-fine-alternative-to-shots https://web.archive.org/web/20160909210149/http://www.niaid.nih.gov/topics/environmental-allergies/Pages/immunotherapy.aspx