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Visionary Science for Life Changing Cures

2016 – 2017

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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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XLRS * RS1 Clinical data

ACHM CNGB3 Wholly Owned Clinical data

CNGA3 Wholly Owned File IND

XLRP * RPGR File IND

AMDTarget-1 Wholly Owned Target

announcement

Target-2 Wholly Owned Target announcement

Pipeline: Multiple Shots on Goal

Program Gene Proof –of-Concept

IND-Enabling Phase 1 Pivotal Partnering Next Key

Milestones

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AGTC’s Flexible Platform…Genetic therapies are complex with interdependent

components that must work in harmony

…and Robust IP Portfolio

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Why Ophthalmology• No current treatments for diseases targeted

by AGTC product candidates• 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.

Restoring SightSignificant Unmet Medical Need

Child with achromatopsia outdoors

Same child indoors in dim light

Day blindness 20/200

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

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The EyeThe Retina - a highly organized tissue

Pupil

IrisCornea

Lens

Retina

Fovea

Conediseases

Rod diseases

AMD

Optic nerve

Retinal pigmentepithelial cell

Rod cellCone cell

Mueller cell

Horizontal cell

Bipolar cell

Amacrine cell

Ganglion cell

Vitreoushumour

Rod diseases

Light enters retina

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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 – Dose Escalation Phase 1/2 Trial

• Primary Endpoint is Safety̶O Multiple secondary endpoints; visual acuity and visual fields most relevant

• Eight Patients Dosed̶O Mild to moderate inflammation; overall well tolerated̶O Expanded number of sites; multiple patients in screening

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AchromatopsiaDisease• 70% of Achromatopsia is caused by mutations in

the A3 and B3 genes. AGTC is currently working on these two genetics mutations that result in severely impaired vision and day blindness

Impact • Best-corrected visual acuity about 20/200

(legally blind)• Extreme light sensitivity resulting in daytime

blindness• Reduced or complete loss of color discrimination

Positioned for Success• Robust animal models and an understanding of

human disease phenotype • Favorable regulatory environment• Defined clinical development plan• Strong IP position

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ACHM – Dose Escalation Phase 1/2 Trial

• Primary Endpoint is Safety̶O Multiple secondary endpoints; visual acuity and color vision most relevant

• Two Patients Dosed̶O Multiple eligible patients identified for trial

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X-linked Retinitis Pigmentosa (XLRP)Disease• Missing protein results in night blindness and

progressive constriction of visual fields; legal blindness by age 45

Proof-of-Concept• Dog model with same genetic defect

AAV Delivery to Primate Retina• Subretinal delivery to rods and cones

Treated area

Untreated area

Photoreceptor Preservation

Visual Field Constriction

Photoreceptornuclei

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Management

Sue Washer Chief Executive Officer

Jeff Chulay, M.D. Chief Medical Officer

Rabia Ozden, M.D. VP Clinical Development

Matt Feinsod, M.D. Product Development Officer

Stephen Potter Chief Business Officer

Larry Bullock Chief Financial Officer

Mark Shearman, Ph.D. Chief Scientific Officer

Experienced Team

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Financial SummaryStrong Balance Sheet

$173 MillionCash & Investment as of 6/30/2016

$100-120 Million Fiscal YE 6/30/2017 Cash Expected

Sufficient Cash to: Complete enrollment and analysis

of full data set from the ongoing Phase 1/2 human clinical trials for XLRS and both of the planned ACHM Phase 1/2 human clinical trials

Initiate and analyzeinitial data from the Phase 1/2 human clinical trial for XLRP

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Visionary Science for Life Changing Cures

2016 – 2017

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