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OIS@ASRSAugust 8, 2016

Visionary Science

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Forward Looking StatementsToday’s presentation includes forward-looking statements intended to qualify for the Safe Harbor from liability established by the Private Securities Litigation Reform Act of 1995. These forward-looking statements, including statements regarding our planned pre-clinical and clinical studies, timing or ability to close partnerships, regulatory approval process and demand for our product candidates, are subject to risks, uncertainties and other factors that could cause actual results to differ materially from those suggested by our forward-looking statements.

These factors include, but are not limited to, the following:

• We have incurred significant losses since inception and anticipate that we will continue to incur significant losses for the foreseeable future.

• Our ability to generate revenue from product sales is highly uncertain.

• We may need to raise additional funding in the future, which may not be available on acceptable terms, or at all.

• No gene therapy products have been approved in the United States, and we may not be able to obtain regulatory approvals for our product candidates.

• We have encountered and may continue to encounter substantial delays in our clinical trials or fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities.

• We rely on third parties to conduct, supervise and monitor our clinical trials and to conduct certain aspects of our product manufacturing and protocol development.

• The insurance coverage and reimbursement status of our product candidates is uncertain.

• Negative public opinion and increased regulatory scrutiny of gene therapy and genetic research may adversely affect public perception of our product candidates and prospects for our business.

• If we are unable to obtain and maintain adequate patent protection for our technology and products our competitors could develop and commercialize technology and products similar or identical to ours.

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Company Highlights

Broad collaboration with Biogen to develop gene therapies in ophthalmology & other indicationsKey Partnership

AGTC is developing genetic therapies to treat patients with inherited diseases. Treatments are precisely designed to meet the needs of each specific genetic disorder. AGTC’s most advanced gene therapy programs are designed to restore visual function in patients with rare blinding diseases.

Multiple opportunities to provide long-term value to patientsBroad Pipeline

>100 patents and patent applications protecting candidate genes, vector capsids, manufacturing and delivery

Extensive IP Portfolio

Deep Expertise In vector selection, design, manufacturing and delivery

Become the leader in ophthalmology and otology gene therapyClear Vision

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AAV Gene Therapy – Potential For Long Lasting EffectA

B

E

C

D

F

GH

Based on figure from Bartlett, et al., J Virol 2000

ITR ITRpromoter cDNA

AAV vector

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Why Ophthalmology

• No current treatments for diseases targeted by AGTC product candidates

• 200+ genetic disease of the eye• Many people fear blindness more than death• Blinding eye diseases hinder the lives of patients

every day, making it impossible to perform tasks such as driving, riding a bike, and even playing outside.

Significant Unmet Medical Need

Growing Scientific Support

• Extensive preclinical data­ Highly predictive animal models­ Well characterized diseases

• Well-defined clinical endpoints simplify trial design

• Preliminary evidence of safe targeted delivery in human trials

Rod diseases

PupilIris Cornea

Lens

Retina

Fovea

Conediseases

Rod diseases

AMD

Optic nerve

Vitreoushumour

The Retina - a highly organized tissue

Retinal pigmentepithelial cell

Rod cellCone cell

Mueller cell

Horizontal cellBipolar cellAmacrine cell

Ganglion cell

Light enters retina

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Lead Program: X-linked Retinoschisis (XLRS)

Disease• Missing structural protein results

in poor vision not correctable with eyeglasses

• No current treatments

Normal OCT XLRS OCT

Impact• Poor vision (20/100) detected by

school age• Difficulty reading, driving, or

recognizing faces• 30% chance of retinal detachment

or vitreous hemorrhage at any age

Positioned for Success • Robust animal models and an

understanding of human disease phenotype

• Favorable regulatory environment• Defined clinical development plan• Strong IP position

AAV2tYF-GFP AAV2tYF-GFP AAV2tYF-RS1myc

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XLRS Endpoints – For Approval

Visual Acuity• ETDRS chart

– Visual acuity is measured as number of letters read

– More letters read = better visual acuity (85 ETDRS letters = 20/20 vision)

By Patient (Example from AGTC LCA trial)

Blue = treated eye

Visual Field• Static perimetry (spot by spot, with

each spot getting its own visual sensitivity value)

• Kinetic perimetry (from outside in, defining zones of visual sensitivity along the way)

Static PerimetryHill of Vision

Red = untreated eye

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XLRS Endpoints – For Additional Support

Retinal Function• Electroretinography (ERG) to quantify

electrical activities among retinal cell types

– a-waves: photoreceptors– b-waves: bipolar cells and

Müller cells

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3302432/

Retinal Layers• Optical coherence tomography (OCT)

to identify and quantify anatomic features

- retinal thickness - thickness of individual layers within retina

(e.g. ellipsoid zone)- total cyst volume

Horizontal (mm)

Ver

tical

(mm

)

Cyst S ize (micron)

-3 -2 -1 0 1 2 3

-3

-2

-1

0

1

2

3 0

50

100

150

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350

400

450

500

Horizontal (mm)

Ver

tical

(m

m)

Cyst Size (micron)

-3 -2 -1 0 1 2 3

-3

-2

-1

0

1

2

3 0

50

100

150

200

250

300

350

400

450

500

Horizontal (mm)

Ver

tical

(mm

)

Cyst S ize (micron)

-3 -2 -1 0 1 2 3

-3

-2

-1

0

1

2

3 0

50

100

150

200

250

300

350

400

450

500

3.330 mm3

2.154 mm3

2.494 mm3

Day 1

Day 90

Day 91A-wave implicit time = B-wave latency

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XLRS * RS1 Clinical data 2016

ACHM CNGB3 Wholly Owned Clinical data

2016

CNGA3 Wholly Owned File IND 2016

XLRP * RPGR File IND 2017

AMDTarget-1 Wholly Owned

Target announcement

2016

Target-2 Wholly OwnedTarget

announcement 2016

* AGTC has the option to co-promote 2nd approved product and the right on both programs to opt into a 50/50 cost sharing and profit sharing relationship.

Pipeline: Multiple Near Term Shots on Goal

Program Gene Proof –of-Concept

IND-Enabling Phase 1 Pivotal Partnering Next Key

Milestones

Research on-going for additional indications

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Visionary Science

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