Presentation by AGTC at OIS@ASRS 2016. Participant: Stephen Potter, Chief Business Officer - AGTC Powered by: Healthegy For more ophthalmology innovation Visit us at www.ois.net

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OIS@ASRS
August 8, 2016
Visionary Science

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Forward Looking Statements
Today’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 indications
Key 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 Effect
A
B
C
D
F
G
H
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
Pupil
Iris Cornea
Lens
Retina
Fovea
Cone
diseases
Rod
diseases
AMD
Optic
nerve
Vitreous
humour
The Retina - a highly organized tissue
Retinal pigment
epithelial cell
Rod cell
Cone cell
Mueller cell
Horizontal cell
Bipolar cell
Amacrine 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 Perimetry
Hill 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)
Vertical(mm)
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)
Vertical(mm)
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)
Vertical(mm)
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
3.330
mm3
2.154
mm3
2.494
mm3
Day 1
Day 90
Day 91
A-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
AMD
Target-1 Wholly Owned
Target
announcement
2016
Target-2 Wholly Owned
Target
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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