MONSIELiving with RDEBJefferies 2019 Healthcare Conference

June 5, 2019

Safe Harbor Statement

This presentation contains certain statements that may be forward-looking within the meaning of Section 27a of theSecurities Act of 1933, as amended, including statements relating to the product portfolio and pipeline and clinicalprograms of the company, the market opportunities for all of the company’s products and product candidates, and thecompany’s goals and objectives. These statements are subject to numerous risks and uncertainties, including butnot limited to the risks detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2018,and other reports filed by the company with the Securities and Exchange Commission.

This presentation does not constitute an offer or invitation for the sale or purchase of securities or to engage in anyother transaction with Abeona Therapeutics or its affiliates. The information in this presentation is not targeted at theresidents of any particular country or jurisdiction and is not intended for distribution to, or use by, any person in anyjurisdiction or country where such distribution or use would be contrary to local laws or regulations. The Companyundertakes no obligations to make any revisions to the forward-looking statements contained in this presentation or toupdate them to reflect events or circumstances occurring after the date of this presentation, whether as a result ofnew information, future developments or otherwise.

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Abeona: a fully-Integrated gene & cell therapy company

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Key Milestones

• Pivotal Phase 3 for Recessive Dystrophic Epidermolysis Bullosa (RDEB) – start mid 2019

• Updates on enrollment and clinical data for Phase 1/2 study in MPS IIIA and MPS IIIB – 2H2019

• CLN1/Infantile Batten disease - IND “may proceed” received May 2019

• Abeona GMP gene and cell therapy facility established in Cleveland

- Seamless transition from concept-to-commercial across multiple modalities

- EB-101 manufacturing for upcoming Phase 3 study and commercialization

- Quality systems and staff in place to support EB and AAV programs

Robust Pipeline

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RDEB Clinical ProgramEB-101

Orphan Drug Designation (FDA)✓

Orphan Drug Designation (EU)✓

Rare Pediatric Disease Designation (FDA)✓

Breakthrough Therapy Designation (FDA)✓

Regenerative Medicine Advanced Therapy Designation (FDA)

✓

Group of devastating inherited connective tissue diseases; characterized by skin blisters and erosions

• Recessive Dystrophic Epidermolysis Bullosa (RDEB): Absence of the COL7A1 gene (encodes type VII collagen to anchor skin to the underlying stroma)

No FDA-approved therapy

Phase 1/2 clinical trial completed• Conducted at Stanford University School of Medicine• Primary endpoint: Safety, wound healing vs. baseline• Secondary endpoint: Expression of type VII collagen, pain, itching,

quality-of-life

Preparing for pivotal Phase 3

Supportive Natural History Study helps shape clinical program• Average duration of untreated chronic wounds >7 years• 128 RDEB Subjects - clinical trial readiness

EB-101: Breakthrough Therapy for Severe Form of EB

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UNTREATED CHRONIC WOUND: ARMBASELINE 3 MONTHS 6 MONTHS

Recurrent Wounds Over Time (N=25)

Chronic Wounds Over Time (N=25)

Time to heal (6 weeks)Time to re-blister (3 weeks)

baseline 2 months 5 months

baseline 2 months 3 months

RDEB Wounds Can Have Distinct Time CoursesChronic

Recurrent

≤ 19 cm2

20-39cm2

≥ 40 cm2

RecurrentWounds 39% 4% 57%

Chronic OpenWounds 33% 13% 55%

0%

10%

20%

30%

40%

50%

60%

Perc

ent o

f Wou

nds

Stanford Natural History Study: Size Distribution Of Wounds

N = 120

N = 104

These large wounds are the most urgent to treat as they carry the greatest burden:• Pain• Pruritis• Risk of infection

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EB-101: 26-days post growth

EB-101: ready for patients

EB-101: Ex-Vivo Autologous Gene-Corrected Cell Therapy

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Site Location Descript Estimated duration 3m 6m 12m 24m

A R lateral hand Erosion 3-5 yrs

B R medialhandScar tissue 3-5 yrs

C L ventral footErosion and scar 3-5 yrs

D L hand Scar tissue 3-5 yrs

E R foot Erosion and scar 3-5 yrs

Z L ventralfootInduced wound New

≥75% healed 50-70% healed < 50% healed

Baseline 3 months 6 months 12 months

Phase 1/2 Study: EB-101 restored collagen VII that forms anchoring fibrils

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Anti-COl7A1 NC2 MabHoechst 33342

Pre-Grafting 3 Month 6 Month 9 Month 12 Month

Pain at wound site (% reported yes) 58% 0% 17% 20% 0%

Itch at wound site (% reported yes) 67% 5% 17% 50% 25%

Lack of durability at wound site(% reported yes) 90% 0% 0% 0% 0%

Ease of blistering at wound site (% reported yes) 83% 0% 0% 0% 0%

EB-101 significantly improved patient-reported outcomes

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Phase 1/2 results: EB-101 provides durable healing of chronic wounds

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2 years 5 years

A R distal forearm >5 B L forearm >5C R proximal forearm >5D R shoulder >5 E L arm 5 B L shoulder >5C R forearm 5-MarD R posterior shoulder >5E Lower back >5 Z R upper chest InducedA R lateral hand 5-MarB R medial hand 5-MarC L ventral foot 5-MarD L hand 5-MarE R foot 5-MarZ L ventral foot InducedA L distal forearm >5 B L medial forearm >5 C L proximal forearm >5 D R lateral forearm >5E R distal forearm >5 Z R medial forearm InducedA L upper arm 16B L upper arm 16

5 C R upper arm 16D R upper arm 16E R upper arm 16F R upper arm 16A R lateral back upper 20B R middle back upper 20

6 C R medial back upper 20D R lateral. back lower 20E R middle back lower 20F R medial back lower 20A R back corner 20

7 B R lateral outer leg 20C R Back central 20D R Back medial 20E R foot front anterior 20F Back upper corner 20

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12 months 3 years 4 years

1

2

3 months 6 months

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Participant Site Location Wound Age (years)

R&D Day 2018

PatientSiteLocationDescription at BaselineWound Age1 month23 months6 months12 months3 years4 years

2 years5 years

RDEB Wound Healing

1AR distal forearmErosion>5 yrs>75%

BL forearmErosion>5 yrs>50%-75%

CR proximal forearmErosion>5 yrs5 yrs

EL armNew blister1 wk

ZR armInduced woundNew

2ACentral chestErosion>5 yrs

BL shoulderErosion and scar>5 yrs

CR forearmErosion and scar3-5 yrs

DR posterior shoulderInflamed erosion>5 yrs

ELower backErosion>5 yrs

ZR upper chestInduced woundNew

3AR lateral handErosion3-5 yrs

BR medial handScar tissue3-5 yrs

CL ventral footErosion and scar3-5 yrs

DL handScar tissue3-5 yrs

ER footErosion and scar3-5 yrs

ZL ventral foot Induced woundNewUnable to assess

4AL distal forearmInflamed erosion>5 yrs

BL medial forearmInflamed erosion>5 yrs

CL proximal forearmInflamed erosion>5 yrs

DR lateral forearmInflamed erosion>5 yrs

ER distal forearmInflamed erosion>5 yrs

ZR medial forearmInduced woundNew

AL upper armErosion16 yrs

BL upper armErosion16 yrs

5CR upper armErosion16 yrs

DR upper armErosion16 yrs

ER upper armErosion16 yrs

FR upper armErosion16 yrs

AR back axilla (lateral), upper rowErosion20 yrs

BR back axilla (middle), upper rowErosion20 yrs

6CR back axilla (medial) upper rawErosion20 yrs

DR back axilla (lateral) lower rowErosion20 yrs

ER back axilla (middle), lower rowErosion20 yrs

FR back axilla (medial) lower rowErosion20 yrs

AR back cornerErosion20 yrs

7BR lateral outer legErosion20 yrs

CR Back centralErosion20 yrs

DR Back medialErosion20 yrs

ER foot front anteriorErosion20 yrs

F Back upper cornerErosion20 yrs

ASGCT 2018

PatientPatientSiteLocationDescription at BaselineWound Age1 month3 months6 months12 months2 years

GT 011AR distal forearmRecurrent wound>5 yrs

BL forearmRecurrent wound>5 yrs

CR proximal forearmRecurrent wound>5 yrs

DR shoulderOpen wound>5 yrs

EL armNew blister1 wk

ZR armInduced woundNew

GT 032ACentral chestOpen wound>5 yrs

BL shoulderRecurrent wound>5 yrs

CR forearmRecurrent wound3-5 yrs

DR posterior shoulderOpen wound>5 yrs

ELower backOpen wound>5 yrs

ZR upper chestInduced woundNew

GT 043AR lateral handOpen wound3-5 yrs

BR medial handOpen wound3-5 yrs

CL ventral footRecurrent wound3-5 yrs

DL handRecurrent wound3-5 yrs

ER footRecurrent wound3-5 yrs

ZL ventral footInduced woundNew

GT054AL distal forearmOpen wound>5 yrs

BL medial forearmOpen wound>5 yrs

CL proximal forearmOpen wound>5 yrs

DR lateral forearmOpen wound>5 yrs

ER distal forearmOpen wound>5 yrs

ZR medial forearmInduced woundNew

Chronic untreatedR elbowOpen wound >5yrs

Chronic untreatedL elbowOpen wound>5yrs

GT075AL upper armOpen wound16 yrs

BL upper armOpen wound16 yrs

CR upper armOpen wound16 yrs

DR upper armOpen wound16 yrs

ER upper armOpen wound16 yrs

FR upper armOpen wound16 yrs

Chronic untreated Antecubital fossaOpen wound16 yrs

GT 086AR back axilla (lateral), upperOpen wound20 yrs

BR back axilla (middle), upperOpen wound20 yrs

CR back axilla (medial) upperOpen wound20 yrs

DR back axilla (lateral) lowerOpen wound20 yrs

ER back axilla (middle), lowerOpen wound20 yrs

FR back axilla (medial) lowerOpen wound20 yrs

Chronic untreated Lower back (5 matched wounds)Open wound20 yrs

GT097AR back cornerOpen wound20 yrs

BR lateral outer legRecurrent wound20 yrs

CR Back centralOpen wound20 yrs

DR Back medialOpen wound20 yrs

ER foot front anteriorRecurrent wound20 yrs

FBack upper cornerRecurrent wound20 yrs

Chronic untreated R dorsal foot (small)Recurrent wound20 yrs

Chronic untreatedL dorsal foot (small)Recurrent wound20 yrs

EB-101 summary and next steps

• Successful Phase 1/2 • Favorable safety profile with no product-related SAEs to date• Significant and durable wound healing, with up to 5 years of follow-up• Continuous type VII collagen expression 2+ years post treatment

• Established GMP manufacturing capability at Abeona• Manufacture both clinical and commercial product in Cleveland• Scalable capacity to support commercial launch

• VITAL: Phase 3 Trial• Final stages of regulatory CMC review near completion• First patient expected to enroll mid-2019

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MPS III Clinical ProgramABO-102* ABO-101

Orphan Drug Designation (FDA)✓Orphan Drug Designation (EU)✓Rare Pediatric Disease Designation (FDA)✓

Fast Track Designation (FDA)✓Regenerative Medicine Advanced Therapy Designation* (FDA)

✓

Inherited monogenic disorders causing lysosomal enzyme deficiency

• Two most common forms categorized by deficient enzymes:

• MPS IIIA (SGSH), MPS IIIB (NAGLU)

• Abnormal accumulation of glycosaminoglycans (GAGs; heparan sulfate (HS))

• Loss of speech/language, cognitive decline, behavioral abnormalities, seizures,

sleep disturbances

• 70% of children with MPS III do not reach age 18 years

Estimated incidence of 1 in 70,000 births

Two ongoing global clinical trials

• ABO-102 (AAV-SGSH) for MPS IIIA: USA, EU, Australia clinical sites

• ABO-101 (AAV-NAGLU) for MPS IIIB: USA and EU clinical sites

No approved treatments available

Cell withlysosome deficiency

Normal cell

Ongoing Phase 1/2 clinical trials for Sanfilippo Syndrome (MPS III)

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MPS IIIA Natural History: Cognitive & Developmental Assessments

Chronological Age (mo)

Cog

nitiv

e A

ge E

quiv

alen

t (m

o)

Shapiro et al. 2016

0 3 6 9 12 15 18 21 24 27 30 33 36 39 42 45 48 51 54 57 60 63 66 69 72 75 78 81 84 87 90 93 96 99 102

105

108

111

114

0

3

6

9

12

15

18

21

24

27

30

33

36

39

42

45

Chronological Age (Month)

p

g (

)

DQ 1

00

Chronological Age (mo)

Dev

elop

men

tal A

ge (m

o)

Truxal, K.V. et al. 2016)

36m age equivalent

36m age equivalent

A Phase 1/2 Clinical Trial (ABT-001) for MPS IIIA with AAV9-SGSH

Intravenous Dosing

• Cohort 1: 5 x 1012 vg/kg (n=3)

• Cohort 2: 1 x 1013 vg/kg (n=3)

• Cohort 3: 3 x 1013 vg/kg (9-12) 8 subjects treated

Primary Endpoint

• Safety

Secondary Endpoints

• Cerebrospinal Fluid (CSF) and/or urinary HS and/or GAGs• CSF and serum SGSH enzyme activity• Liver, spleen and brain volume by MRI• Neurocognitive function as measured by Leiter International Performance Scale and the Mullen

Scales of Early Learning

• Adaptive functioning, by Vineland Adaptive Behavior Scale (caregiver report)

2-Year, Open-label, dose-escalation clinical trial

ClinicalTrials.gov: NCT02716246; Study Sponsor Abeona Therapeutics Inc

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Reductions in CSF and Urine Heparan Sulfate Post Treatment

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Lower Limit Quantitation

Normal Liver Volume

Reduction in Liver Volume Post Treatment vs Natural History

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1. (Truxal et. al., 2016, Mol. Genet. Metab.)

*Gray lines indicate natural history study patients1

Change in Mullen Developmental Age Post Treatment vs. Natural History

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1. (Truxal et. al., 2016, Mol. Genet. Metab.)

*Gray lines indicate natural history study patients1DQ= Developmental Quotient

* DQ100 Approximate typical development trajectory (Eapen et al. BMC Pediatrics 2013)

Summary of MPS IIIA ABO-102 Phase 1/2 Study Data

• N=14 as of November 2018- Length of follow up in cohort 3: 6 months (N=6 of 8 total), 12 months (N=3 of 8 total)

• Clear dose-response, and sustained improvement in biomarkers (HS CSF, Liver Volume)• Encouraging neurocognitive signals seen in younger, higher functioning patients in cohort 3

- Caregiver observations include:- Reports of improved attention, interaction with siblings/schoolmates/environment

- Improved sleep

- Improved speech in young patients

• Enrolled at age 2.3 years: “putting adjectives and adverbs into complete sentences”

• Safety: ABO-102 has been well tolerated to date- No serious drug related adverse events

- SGSH ELISpot negative

- Length of Follow up as of November 2018:• Cohort 1: 27-30 months; Cohort 2: 19-21 months; Cohort 3: 1-16 months

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2-year, Open-label, dose-escalation global clinical trial

IntravenousDosing

• Cohort 1: 2 x 1013 vg/kg (n=2 subjects*) 2 patients treated

• Cohort 2: 5 x 1013 vg/kg (n=3-6 subjects planned) 1 patient treated

Primary Endpoint • Safety

Secondary Endpoints

• CSF and/or urinary HS and/or GAG• CSF and serum NAGLU enzyme activity levels• Liver, spleen and brain volume (MRI)• Neurocognitive function as measured by Leiter International Performance Scale and the Mullen

Scales of Early Learning

• Adaptive functioning, by Vineland Adaptive Behavior Scale (caregiver report)

A Phase 1/2 Clinical Trial (ABT-002) for MPS IIIB with AAV9-NAGLU

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*Clinical study protocol states 3 subjects in Cohort 1; however, due to exceptional circumstances and following robust safety profile and positive review from DSMB, trial was cleared in Europe to advance to Cohort 2 dose

https://clinicaltrials.gov/ct2/show/NCT03315182?term=abeona&cond=MPS+IIIB&rank=1

Liver Volume CSF and Urine HS

Reduction in Liver Volume, CSF and Urine Heparan Sulfate Post Treatment vs. Natural History

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1. (Truxal et. al., 2016, Mol. Genet. Metab.)

*Gray lines indicate natural history study patients1

Change in Mullen Developmental Age Post Treatment vs. Natural History

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1. (Whitley et al., 2018)

*Gray lines indicate natural history study patients1

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CLN1 (Infantile Batten) DiseaseABO-202

Orphan Drug Designation (FDA)✓Orphan Drug Designation (EU)✓Rare Pediatric Disease Designation (FDA)✓

ABO-202: scAAV9 for Treatment of Infantile Batten Disease – CLN1

• Infantile Neuronal Ceroid Lipofuscinosis (INCL): - Severe neurodegenerative lysosomal storage disease, with no approved treatment- Caused by mutations in the CLN1 gene, encoding the soluble lysosomal enzyme palmitoyl-protein

thioesterase-1 (PPT1)

• Exclusive, worldwide license secured for AAV9 for treatment of CLN1

• Onset of symptoms between 6 to 24 months of age - Progressive visual failure, cognitive decline, loss of fine and gross motor skills develop by 2-3 years- By 5 years of age there is loss of light perception, complete loss of motor skills and social interaction;

myoclonus and seizures can appear eventually

- Death usually occurs by 7 years of age

• Therapeutic approach is a scAAV9 vector with codon-optimized CLN1 transgene

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ABO-202 Therapeutic Approach: combination IV & IT dosing

• Demonstrated efficacy in CLN1 mice support rationale for clinical dosing by both intrathecal (IT) and intravenous (IV) simultaneously

• IT: Intrathecal (lumbar puncture)

• IV: Intravenous (tail vein)

• Potential Benefits of IT/IV dosing include:

• Combination dosing with both a high intrathecal and high intravenous doses showed significant efficacy in CLN1 mice

• Combination dosing overcomes IT dose limitations due to volume constraints and bolsters IV doses, especially in older patients

• “Double exposure” to the CNS enables broad and distributed coverage

Steven Gray, Ph.D. - Batten Researcher

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ABO-202: Phase 1/2 clinical trial readiness

• IND-enabling toxicology and efficacy studies completed ⎻ Strong safety, with no significant toxicology findings in the combination dose escalation study⎻ Data demonstrated normalized survival, muscle function and cognition, especially with early treatment

• FDA IND clearance received Q2 2019

• Potential Benefits of IT/IV dosing include:- Overcoming IT dose limitations due to volume constraints and bolsters IV doses, especially in older patients- “Double exposure” to the CNS enables broad and distributed coverage

• Trial outcomes supported by large, multi-year Natural History Study

• World-leading investigators and clinical sites for CLN1⎻ University of Rochester and University of Hamburg

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Next Generation AIMTM AAV Platform

AIM™: Next Generation AAV Vector Platform

• AIM™ Vector Platform: AAV viral vector platform selected to target CNS, lung, skin, muscle, liver and other tissues

• Key Advantages of AIM™• First generation demonstrated increased gene delivery efficiency

to specific tissues• Second and third generations have increased tissue tropisms

• Over 100 capsids under evaluation

• Potential for redosing previously treated AAV subjects

• Cystic Fibrosis and Ocular programs demonstrate proof-of-concept to support pre-clinical studies

DNASAL FRAGMENTATION

ASSEMBLY AND AMPLIFICATION

CHIMERIC CAPSID LIBRARY

WILD TYPE AAVS

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AAV GMP MANUFACTURING

Abeona’s Gene Therapy Manufacturing and Quality Capabilities

Advantages of internal cGMP manufacturing• Control of supply chain, including timelines and cost• Internal Abeona quality systems and personnel

Multi-use production platform yields flexibility & risk reduction• Manufacturing suspension and adherent upstream mammalian and insect platforms• High quality, high titer material supporting clinical development & commercial scalability

Abeona’s large-scale cGMP capacity and deep expertise• 26,000 sq. ft facility in Cleveland– stage 1of 2 completed, stage 2 underway• State-of-the-art laboratories to support CMC development for process and analytics

Commercial readiness of Abeona’s facility• Designed for seamless transition from concept-to-commercial• Conversion of GMP to commercial scalability

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• FDA Division of Manufacturing and Product Quality (DMPQ) supports clinical or single use commercial facility

• 46 highly trained staff members in instrumentation and equipment Quality, technical operations, process development, assay development, management of contract manufacturing

• AAV processing GMP facility supportive of clinical translation• Separate Upstream and Downstream Suites• Capable of Clinical and Commercial Production

• Cell processing facility• Commercially viable GMP suites• Dedicated to the production of EB-101• Capacity for Commercial launch and scalability

Abeona’s Gene Therapy Manufacturing and Quality Capabilities

Hyperstacks18,000 cm2

Eppendorf BiobluScalable to 40L

CellSTACKS6,320 cm2

iCellis Nano40,000 cm2

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João Siffert, M.D. Chief Executive Officer, Head of R&D, Chief Medical Officer

Timothy J. Miller, Ph.D.President, Chief Scientific Officer

Christine SilversteinChief Financial Officer

Management Team

Max ColaoChief Commercial Officer, Head of Business Development

Edward CarrChief Accounting Officer

Maria Escolar, M.D. Pittsburgh Children’s Hospital

John Cooper, Ph.D.University of California, San Diego

Scientific AdvisorsJonathan Mink, M.D., Ph.D. University of Rochester

Erika Augustine, M.D.University of Rochester

Steven Gray, Ph.D.University of Texas Southwestern

Kevin Flanigan, M.D.Nationwide Children’s Hospital

Juan Ruiz, MD, Ph.D.Head of European Medical Affairs

Neena Patil, J.D.SVP, General Counsel

Jay BircherSVP, Quality & Technical Operations

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Slide Number 1Safe Harbor Statement Abeona: a fully-Integrated gene & cell therapy companyRobust PipelineSlide Number 5EB-101: Breakthrough Therapy for Severe Form of EBRDEB Wounds Can Have Distinct Time CoursesStanford Natural History Study: Size Distribution Of WoundsEB-101: Ex-Vivo Autologous Gene-Corrected Cell TherapyPhase 1/2 Study: EB-101 restored collagen VII that forms anchoring fibrils EB-101 significantly improved patient-reported outcomesPhase 1/2 results: EB-101 provides durable healing of chronic woundsEB-101 summary and next stepsSlide Number 15Ongoing Phase 1/2 clinical trials for Sanfilippo Syndrome (MPS III) MPS IIIA Natural History: Cognitive & Developmental AssessmentsA Phase 1/2 Clinical Trial (ABT-001) for MPS IIIA with AAV9-SGSHReductions in CSF and Urine Heparan Sulfate Post TreatmentReduction in Liver Volume Post Treatment vs Natural HistoryChange in Mullen Developmental Age Post Treatment vs. Natural HistorySummary of MPS IIIA ABO-102 Phase 1/2 Study DataA Phase 1/2 Clinical Trial (ABT-002) for MPS IIIB with AAV9-NAGLU Reduction in Liver Volume, CSF and Urine Heparan Sulfate Post Treatment vs. Natural HistoryChange in Mullen Developmental Age Post Treatment vs. Natural History Slide Number 26ABO-202: scAAV9 for Treatment of Infantile Batten Disease – CLN1 ABO-202 Therapeutic Approach: combination IV & IT dosingABO-202: Phase 1/2 clinical trial readinessSlide Number 30AIM™: Next Generation AAV Vector PlatformSlide Number 32Abeona’s Gene Therapy Manufacturing and Quality CapabilitiesSlide Number 34Slide Number 35