Delivering gene therapy to patients

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FORWARD-LOOKING STATEMENTS

This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this presentation, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. The forward-looking statements contained in this presentation reflect uniQure’s current views with respect to future events, and uniQure assumes no obligation to update any forward-looking statements except as required by applicable law.

These forward-looking statements include, but are not limited to, statements regarding the risk of cessation or delay of any of the ongoing or planned clinical studies and/or development of our product candidates, the risk of delay or failure to successfully commercialize or obtain further regulatory approval of Glybera, and the risk that our collaborations with Chiesi or our other collaboration partners will not continue or will not be successful. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors described under the heading “Risk Factors” in uniQure’s form 20-F and the prospectus dated February 5, 2014, both documents filed with the Securities and Exchange Commission. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, except as required by law.

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Glybera - 1st Approved Gene Therapy in the EU Glybera for Lipoprotein Lipase Deficiency

4

Gene Therapy and uniQure:

Changing the Future of Medicine

Gene

therapy

– a real

opportunity

uniQure has

all the

building

blocks

A new

chapter in

the history

of medicine

1 time interventions

Long term effect

Ultimate patient benefit

Highly disruptive*

Cutting edge science

Manufacturing leadership

Modular portfolio development

Regulatory expertise

Commercialization experience

Strong management team

*) MIT Review / Fierce 15:

uniQure among 50 most

disruptive companies in 2013

Establish Gene Therapy Leadership

Build a Portfolio

Milestones

6

Glybera – Gene Therapy for an Orphan Disease Single intervention to restore lipoprotein lipase enzyme

Lack of lipoprotein lipase

Prevents metabolism of post-prandial

TG/chylomicron complexes

Leads to Accumulation of

post-prandial chylomicrons in plasma

Provokes recurrent pancreatitis

7

Glybera Trial Results over 8 Years in 27 Patients

Glybera, uniQure Platform

Considered Safe

by EMA

Restoration of LPL Activity

Leads to

Improved Lipid Clearance

1 Yr Post a Single

Intervention

ZERO ICU Stays

in 6 Years

Post a Single Intervention

in the Label Population

ZERO Severe Pancreatitis

in 6 Years

Post a Single Intervention

in the Label Population

8

EU Post-approval Commitments

Foundation for US Filing

EU approval under

exceptional circumstances

2012 2013 2014 2015 2016 2017

Post-approval commitments

Clinical trials

leading to

EU Glybera

approval

Registry (incl. natural history)

Post-approval study

US filing based on

EU-post approval

commitments

9

uniQure Retains Rights in Attractive Key Markets

Retained key markets include

North America and Japan

Commercialization

in North America

by uniQure

Distribution agreements in

other geographies

> Israel agreement signed

> 5 more planned for 2014

Chiesi have European

commercialization rights

> EUR 31 m down payment

> 20 – 30% royalty from Chiesi

uniQure Territories

Chiesi Territories

10

Glybera EU Launch Preparations Led by Chiesi Chiesi confident for a launch in mid-2014

Finalizing 1st wave countries Germany, UK, NL, Sweden, Austria, Italy > Product released for patient treatment

> Centers of Excellence prepared

Site preparations including Registry set-up finalized

Health care staff training on track

Patients identified, consent procedures ongoing

> European pricing strategy and implementation plan finalized

List prices in several countries soon to be known

> Germany: NUB (in-patient) and AMNOG (out-patient) processes started – Charité, Berlin

> UK list price submission soon - Manchester

> Sweden – List price negotiation hospital/County – Karolinska, Gothenborg

> Other countries to follow soon

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Gene Therapy Platform Building Blocks

Administration

GENE/VECTOR

COPIES INTO

THE CELLS

Manu-

facturing

GENE /

VECTOR

COPIES

Vector

DELIVERY

VEHICLE

Gene

“Blueprint”

THERAPEUTIC

“Protein Factory”

in the body

PROTEIN

FACTORY

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natural AAV DIRECTED EVOLUTION optimized AAV

uniQure’s Vector Platform Strategy

TROPISM

AAV1 Exclusive

for LPLD in Muscle

AAV2

AAV5 Exclusivity for

LIVER / CNS

AAV6

AAV8

AAV9

Synthetic

AAV Mutants Synthetic

AAV Mutants Synthetic

AAV Mutants Synthetic

AAV Mutants Synthetic

AAV Mutants Synthetic

AAV Mutants Synthetic

AAV Mutants Synthetic

AAV Mutants Synthetic

AAV Mutants Synthetic

AAV Mutants Synthetic AAV

Super Mutants

4D Therapeutics

13

uniQure

Baculo-based

Plasmid-based

adherent cultures

Plasmid-based

Suspension cultures Herpes-based Adenovirus-based

Suspension

Cell Culture ++ - ++ + ++

Efficiency

DNA Transfection ++ + - ++ ++

Scalability ++ -- - + ++

Safety ++ - - concerns concerns

Low COGs ++ - - + +

EU Regulatory Approved ++ - - - -

FTO / IP Protection ++ - - complex complex

uniQure’s Leading Insect Cell Manufacturing Platform

Mammalian Cells Insect Cells

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World-leading Manufacturing Capabilities in EU and US Building the world’s largest dedicated AAV manufacturing unit in the US

Amsterdam, NL > 2 x 50 L

> EMA approved facility

Lexington, MA, USA

> 2 x 500 L

(expansion to 2 x 2000 L possible)

> Copy/scale of existing technology

> Estimated time of completion for

test batches end of 2014

> Site opened for employees

on May 1, 2014

Establish Gene Therapy Leadership

Build a Portfolio

Milestones

16

Pipeline 2014

GLYBERA EU

GLYBERA US

HEMOPHILIA B

ACUTE INTERMITTENT PORPHYRIA

SANFILIPPO B

PARKINSON’S DISEASE

HEMOPHILIA A, Huntington’s miRNA, etc ….

PRECLINICAL I II III MARKET

uniQure sponsored investigator led

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Hemophilia B Spontaneous bleeds, joint damage, compliance

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AAV8/FIX Single Intervention Using uniQure Gene

Cassette Reduces Need for Prophylactic Treatment(1)

1) Third party trial conducted by St. Jude’s Children Research Hospital and UC London

Patient 1 2 3 4 5 6 7 Patient 8 Severe

Moderate

Mild

Disease

Severity Low-dose Mid-dose High-dose

Oral Presentation Presented in New England Journal of Medicine 12/2011

12

11

10

9

8

7

6

5

4

3

2

1

% Expression

of normal

St Jude/UCL trial started 4 yrs. ago demonstrated sustained, dose dependent effect:

after single treatment 6/8 patients off prophylaxis, 2/8 significantly lower frequency

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AAV5/FIX Phase I/II Dose Escalation Study in 2014

Population

> ≦ 1% of normal plasma

factor IX levels

> On prophylactic therapy

Objectives

> Assess safety/tolerability

> Define optimal therapeutic dose

Key efficacy assessments

> Factor IX plasma levels

> Need for FIX replacement

therapy

> Incidence of spontaneous

bleeding

> Health related quality of life

High (2.0 × 1013)

Do

se

(gc/k

g)

Mid (2.0 × 1012)

Low (2.0 × 1011)

ST Jude

ST Jude

ST Jude

uniQure

uniQure

AAV5 insect cell

production

AAV8 mammalian

cell-based

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AAV5/FIX - Efficacy in Primates

Human AAV5/FIX expression

levels in macaques similar to

those achieved in humans from

current AAV8 clinical study

(UCL/St Jude’s)

Linear dose response levels

of human AAV5/FIX

in dose escalating GLP tox study

Preclinical data suggests

AAV5 works as well as AAV8

Porphyria

Porphyria

Key

AMT-060 in Rhesus Monkeys

Porphyria

AMT-060 in Cynomolgus Monkeys

0 5 0 1 0 0 1 5 0

0 .1

1

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D a y s a fte r v e c to r in fu s io n

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(% o

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1

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D a y s a fte r v e c to r in fu s io n

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els

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0 5 0 1 0 0 1 5 0

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1

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D a y s a fte r v e c to r in fu s io n

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)

5 e 1 1 g c /k g

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9 .3 e 1 3 g c /k g

5 e 1 2 g c /k g

21

Competitors:

Spark, Baxter, Dimension,

Biomarin

AAV5/FIX – uniQure’s Competitive Position Timeline puts AA5-FIX as first to market –

no other gene therapy competitor has full FTO or validated manufacturing

Gene cassette

exclusive from

St. Jude

AAV5 vector

exclusive from NIH

Proprietary, validated,

scalable, manufacturing

22

Patients Well Controlled – A Myth! Enzyme Replacement Therapy Standard of Care Cost per Patient

per Lifetime

> US$ 10 m

Hospitalization Days

per Patient per Lifetime

> 1.500 2 Interventions

per Patient per Week

> 6,000 Treatments

over 6 Decades

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Strong Savings Potential at Profitable GT Pricing Lifetime cost for a severe hemophiliac – prophylaxis: > US$ 10 m

Pricing will depend upon duration of gene

therapy effect

> Proven expression in animals > 10 years

> Proven expression in humans > 4 years

Performance based annuity payments possible

> Ease of measurement of FIX in plasma

US$ 9 m

US$ 1.5 m

Drug

Related

Cost

Non-Drug

Related

Prophylaxis

US$ 10 m

US$ ? m

US$ ? m

Gene

Therapy

Refs. Carlsson KS et al. On-demand vs. Prophylactic treatment forsevere haemophilia in Norway and Sweden:

differences in treatment characteristics and outcome. Haemophilia 2003; 9:555-566

Henrad s et al. The health and economic burden of haemophlia in Belgium: a rare, expensive and challenging disease.

Orphanet Journal of Rare diseases 2014: 9:39

Refs. Carlsson KS et al. On-demand vs. Prophylactic treatment forsevere haemophilia in Norway and Sweden:

differences in treatment characteristics and outcome. Haemophilia 2003; 9:555-566

Henrad s et al. The health and economic burden of haemophlia in Belgium: a rare, expensive and challenging disease.

Orphanet Journal of Rare diseases 2014: 9:39

Refs. Carlsson KS et al. On-demand vs. Prophylactic treatment forsevere haemophilia in Norway and Sweden:

differences in treatment characteristics and outcome. Haemophilia 2003; 9:555-566

Henrad s et al. The health and economic burden of haemophlia in Belgium: a rare, expensive and challenging disease.

Orphanet Journal of Rare diseases 2014: 9:39

Refs. Carlsson KS et al. On-demand vs. Prophylactic treatment forsevere haemophilia in Norway and Sweden:

differences in treatment characteristics and outcome. Haemophilia 2003; 9:555-566

Henrad s et al. The health and economic burden of haemophlia in Belgium: a rare, expensive and challenging disease.

Orphanet Journal of Rare diseases 2014: 9:39

Carlsson, et al., Henrad et al., company estimates

24

Market Volume (in US$ bn)

Hemophilia A

Hemophilia B

Hemophilia Enzyme Replacement Market Big market volume to protect for current ERT competitors

Market:

US$ 8 bn

Growth

6% p.a. Sources: Morningstar Estimates

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Pipeline 2014

GLYBERA EU

GLYBERA US

HEMOPHILIA B

ACUTE INTERMITTENT PORPHYRIA

SANFILIPPO B

PARKINSON’S DISEASE

6 HEMOPHILIA A, Huntington’s miRNA, etc ….

PRECLINICAL I II III MARKET

uniQure sponsored investigator led

✔

✔

✔

✔

8/8 pts., safety established,

DNA/RNA expressed, data

H2/14

4/4 pts. dosed, data H2/15

3/24 pts. dosed, data 2015

Establish Gene Therapy Leadership

Build a Portfolio

Milestones

27

Cash position of EUR 77.5 m / QU1 2014 to reach

multiple clinical milestones

2014 2015 2016

H1 H2 H1 H2 H1 H2

Glybera – EU launch

Porphyria results

Hemophilia B results

Sanfilippo B results

Glybera Data to Support

U.S. Filing

28

Gene therapy and uniQure:

Changing the future of medicine

Gene

therapy

– a real

opportunity

uniQure has

all the

building

blocks

A new

chapter in

the history

of medicine

1 time interventions

Long term benefit

Highest possible patient benefit

Highly disruptive*

Cutting edge science

Manufacturing leadership

Modular portfolio development

Regulatory expertise

Commercialization experience

Strong management team

*) MIT Review / Fierce 15:

uniQure among 50 most

disruptive companies in 2013

The Leader in Gene Therapy