The guthy-jackson Charitable Foundation was established in 2008 to help solve the orphan autoimmune disease, Neuromyelitis Optica (NMO). In just over five years, tangible advances have been achieved to this end, and our resolve to find a cure grows ever stronger. Back then, we knew that to reach our goals we needed to venture beyond traditional science models and reignite not only research, but the entire process – all while catalyzing partnerships and pathways leading to the clinic. We work at an accelerated pace, navigating the latest scientific technologies, fostering improved clinical paradigms and engaging movers and shakers. All of this energy and dedication is laser-beam focused on our sole mission: solve NMO. And because every moment makes a difference in the life of a patient facing NMO, we seek real-time solutions with a sense of urgency.

To date we have invested nearly $35 million in heartfelt resources on behalf of the NMO patient community. Thus far, our strategy has been to support basic and clinical research to advance discoveries, proof-of-concept clinical studies and industry-sponsored clinical trials. In an unbiased way, we work to accelerate academic, healthcare, industry and regulatory partnerships that reduce barriers and build bridges between the laboratory and the clinic.

Clinical trials depend on a seamless coordination of effort. Most important among these is informing and empowering patients interested in participating in the evaluation of new treatments. As a one-stop-shop for patient information and advocacy, in 2013 we rolled out our online NMO Patient gateway: NMOtion

(“in motion”). This interactive and multi-media resource offers news and tools, including the latest public information on NMO clinical trials. This gateway also affords opportunities for NMO patients and caregivers to register to receive updates on clinical trials, and volunteer blood and clinical data for research. While our Foundation does not sponsor, conduct or endorse any clinical trial for approval, making information available is intended to promote informed decision-making by each individual member of the NMO community.

Since its inception, our Foundation mantra has been about making connections – linking stakeholders – from patients to researchers to caregivers and beyond. Sharing information and ideas is the key, and our media reaches around the world to unite a global effort to solve NMO. Last year we announced our collaboration with Laboratory Corporation of America (LabCorp) to broaden our research biospecimen collection network. This new partnership grows the number of sites in the United States at which patients and relatives may donate blood and clinical data for research.

We are excited that promising treatments are entering clinical trials with hopes of benefitting NMO patients. Never before have the NMO scientific and clinical altimeters reached these heights – and we continue to aim even higher. But rather than reflect on advances made – we focus with even greater energy on the work that remains if we are to solve NMO together. In 2014, we begin a program which focuses on innovative cures, to lead us into bold new territory to seek cures for NMO.

We will stand and advocate for patients and families facing this disease. We will continue to steward even more effective connections among academia, industry and regulatory stakeholders. We will push the conventional envelope on the journey to discover cures. We do this for our daughter – and all the daughters and mothers, sons and fathers, families and friends who live with NMO. – With hope and gratitude, Victoria Jackson & Bill Guthy Founders

2013 Annual ReportFrom the Founders...

As we close the chapter on an exciting fifth year, our path begins

to lead us into bold new territory to seek cures for NMO...

www.guthyjacksonFoundation.org

6th AnnualNMO Roundtable Conference

In 2008 we hosted our first NMO Roundtable Conference. In five years’ time, a growing number of international researchers have actively engaged in NMO research

and clinical studies. At this year’s sixth International NMO Roundtable Conference, 39 researchers shared noteworthy progress in their studies – all of which were centered on finding solutions for NMO.

COUNTR

IES

COUNTRY GROwTh

18 ———————————————————

16 ———————————————————

14 ———————————————————

12 ———————————————————

10 ———————————————————

8 ———————————————————

6 ———————————————————

4 ———————————————————

2 ———————————————————

0 ———————————————————

2008 2009 2010 2011 2012 2013

2

4

7

1112

17

Thanks for making the NMO meeting such a great event to attend. Clearly

Victoria has assembled a talented group of dedicated individuals who make BIG

things happen. — Tom Tedder

Noel Rose, Roundtable Keynote Speaker

2008NMO Roundtable Conference

20136th Annual NMO Roundtable ConferenceNovember 4 - 5, 2013 • Los Angeles, CAContinuing Medical Education Credits16 AMA PRA Category 1 Credits

ATT

ENdEES

ROUNDTABLE CONFERENCE ATTENDEE GROwTh

140 ———————————————————— 120 ————————————————————

100 ————————————————————

80 ————————————————————

60 ————————————————————

40 ————————————————————

20 ————————————————————

0 ————————————————————

2008 2009 2010 2011 2012 2013

64

36

7686

107

128

www.guthyjacksonFoundation.org2013 Annual Report

NMO Patient DayNovember 6, 2013 Los Angeles, CA

2013 Patient Day Group Photo

NMO Patient Day

200 ————————————————————

175 ————————————————————

150 ————————————————————

125 ————————————————————

100 ————————————————————

75 ————————————————————

50 ————————————————————

25 ————————————————————

0 ————————————————————

2009 2010 2011 2012 2013

NMO PATiENT DAY BLOOD DRAw sAMPLEs

18 25

62

117

193

SAmplE

S

When we hosted our first NMO Patient Day five years ago, we didn’t know what to expect – and neither did the audience which consisted of just 56 NMO patients and caregivers.

There were a lot of unanswered questions that everyone had, including us. That November day was the birth of a long-awaited moment for the NMO community. Since that day, we have been sharing and learning from each other about this rare orphan disease. We’re learning that more and more people are diagnosed with NMO after having first been misdiagnosed with multiple sclerosis. We’re learning that there are many more people living with NMO than we were told. Most importantly, we’re learning that we’re not alone, and working together with the scientific, research and pharmaceutical communities is the only way NMO will be cured.

I am so excited about all the tools and

encouragement I received after meeting

so many folks dealing with NMO!

My promise is to pay it forward with

everything that I have to educate, bring

awareness and hopefully fundraise

towards the end goal which is a cure.

— HeaTHer SHeppard, Nmo advocaTe

400 ————————————————————

350 ————————————————————

300 ————————————————————

250 ————————————————————

200 ————————————————————

150 ————————————————————

100 ————————————————————

50 ————————————————————

0 ————————————————————

2009 2010 2011 2012 2013

NMOPATiENT DAY GROwTh

88

144

187

296

370

ATT

ENdEES

Other Attendees

20%

2013 NMO PATiENT DAY ATTENDEEs

Caregivers/Family & Friends

37%

Patients

43%

www.guthyjacksonFoundation.org2013 Annual Report

Kathryn KienholzKathy CooksonKelly AltusKelly UreKelsey MahoneyKim goldmanKris StracenerKullgren Family TrustLaura KalbLaurey TomlinsonLee LeachLee NeibartLinus KarlssonLoreen FeserLuanne grayLuis TormentaMargaret WardMaria TandrediMarie BertolamiMarilyn SteinMark TannerMartin ReichbachMary DuncanMedical Solution ParntersMichael CherryMichael EdwardsMichele LuterbachMichelle FinnertyMichelle ZelekowtizMildred PierceNancy OlesenNathaniel SzkilNick AdamsOrene BumbalesPamela AyersParkview School DistrictPatricia Corron

$5,000 - $10,000Bertolami FamilyBruce FogelElaine VernonNancy Reimer

$2,000 - $4,999Christina BaynesLisa TranMichelle RosenbergSterne Kessler goldstein FoxTanya Spencer

$1,000 - $1,999Annette PluttAntique TroveCelstle Karasgerard DiNomeHoneywell-jeffrey LeclercIBM Employee Contribution

Campaignjacinta BehneKate KarasKelly StyneLojo FoundationMarilynne FranksMichael YeamanWilliam Dumont

$500 - $999Anthony LoweryC. SpielbergerChad LoomisCharles CarsonDaoDiane PiersonElaine RobertsFrank Bertolami jrjack Suzarjoseph KrigNancy Perry-Crotty

$250 - $499Alejandro EspinoAngie SimonDaniel HonigDarlene SmithDenis VaillancourtFrances Churnowjames Rajchel

2013 Donor List

Dona

tion F

acts

$74,293 TOTAL DONATiONs

2013 DONATiONs

$50,000 sPONsORs ONE EUREKA GRANT

DiD YOU KNOw?

Contact us to sponsor your own Eureka Grant

janet RoubianLinda MayPatricia CiffoneProstmanSteve KrigTerri DiCamilloWayneWells Fargo Community

Support Campaign

UNDER $250Alexander greeneAlison CollierAlison jumpAllen PartinAmy HeumannAnastasia gianakasAndy KleinAnne Van DruffAnonymousAtlantic Polymers CorpBarbara glassoverBarbara MatthewsBarbara NicholsBarboro MetzBenjamin HudsonBeverly FoyseBonnie MillerBreyfogle FamilyBridget FloersBuricaterCameron RiverCari MentenCarla MortensenCarol AdamsCarol DavisCathy WilkieCharlesCharles BumbalesClare BlaserCornCynthia WeinfeldDale CarterDaniel BuonagurioDaniel ClarkDavacoolDavid AntonsonDavid FantineDavid MillerDavid WenDebbie Edwarda

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Patricia DiggsPaul PipertzisPaula goettlPaula HendersonPauline RussellPeggy MichelmanPeter OaknePoshe SalonRaif SarchichRamie LavelleRaymond jensenRebecca HooverRichard LagroonRobert HuertaRobin TrambleRomanelli Dental OfficesRosemary BertolamiSarah CampbellSarah FordSarah RoseShellySherry FryStefanie TuckerStephanie gianakeasStephen KooThomas NyboTina PatelTricia IdlerValerie LauroVince DelfiniVirginia HarrisonWanda HarveyWarren FeldmanWells Fargo BankWilliam MatthewsWoltmanZaro Bake Shop

The guthy-jackson Charitable Foundation

13 FUNDED iNsTiTUTiONs 22 PRiNCiPAL

iNvEsTiGATORs28 TOTAL PROjECTs (11 NEw iN 2013)

qUiCK sTATs

Key PersonnelJacinta Behne, M.A.Executive Vice PresidentAdministration and Operations

Katja Van Herle, M.D., M.S.P.H.Executive Vice PresidentClinical Translation and Public Health Initiatives

Michael Yeaman, Ph.D., AdvisorChair, Basic and Translational Sciences - gjCFProfessor of Medicine, David geffen School of

Medicine at UCLAChief, Division of Molecular MedicineVice Chair, Department of Medicine

Harbor-UCLA Medical Center

Medical AdvisorsAmit Bar-Or, M.D., FRCP(C)Associate Professor, Neurology and NeurosurgeryDepartment of Neurology and NeurosurgeryMontreal Neurological Institute

Carrolee Barlow, M.D., Ph.D.AdvisorDrug Development

Terrence F. Blaschke, M.D.Senior Program Officerglobal Health Discovery and Translational SciencesBill and Melinda gates FoundationProfessor of Medicine and Molecular Pharmacology

(Emeritus)Stanford University School of Medicine

Michael Clare-Salzler, M.D.Professor; Chairman; DirectorCenter for Immunology and TransplantationUniversity of Florida

Terry J. Smith, M.D.Frederick G.L. Huetwell Professor of Opthalmology

and Visual SciencesUniversity of Michigan Kellog Eye CenterProfessor, Internal MedicineDivision of Metabolism, Endocrinology and Diabetes

Michael Sofroniew, M.D., Ph.D.Professor, Department of Neurology and Brain

Research InstituteDavid geffen School of MedicineUniversity of California, Los Angeles

Andre Van Herle, M.D.Professor of Medicine (Active Emeritus)University of California, Los Angeles

Howard Weiner, M.D.Professor of Medicine, Center for Neurologic DiseasesBrigham and Women’s HospitalDirector, Multiple Sclerosis ProgramPartners MS Center in Boston, MA

International Clinical Consortium DirectorValerie Pasquetto, Ph.D., PMPProgram DirectorInternational Clinical Consortium

Research Recap

Funding PartnershipsgjCF and the National MS Society awarded Principal Investigator Ilya Kister a joint grant for his NMO research project Cerebral ultra-high field MR imaging in NMO: US-German longitudinal study.

Brigham and Women’s Hospital, Harvard Vijay Kuchroo, D.V.M., Ph.D.

Cleveland Clinic Richard Ransohoff, M.D.

Johns Hopkins University Michael Levy, M.D., Ph.D.

Massachusetts General Hospital Tanuja Chitnis, M.D. Eric Klawiter, M.D.

Mayo ClinicCharles Howe, Ph.D.Claudia Lucchinetti, M.D. Sean Pittock, M.D. Brian Weinshenker, M.D. Dean Wingerchuk, M.D.

Mount Sinai gareth john, Ph.D. Ilana Katz-Sand, M.D.

New York University Ilya Kister, M.D.

Oxford Universityjacqueline Palace, BM (hons), FRCP, DM

St. George’s University of LondonMarios Papadopoulos, M.D., FRCS (SN)

Stanford UniversityMay Han, M.D. Lawrence Steinman, M.D.

University of California, San Francisco Alan Verkman, M.D., Ph.D.Scott Zamvil, M.D., Ph.D.

University of Colorado, Denverjeffrey Bennett, M.D., Ph.D.

University of UtahLawrence Cook, Ph.D. john Rose, M.D.

gjCF Industry CouncilLaunched in 2013, the gjCF Industry Council comprised of industry-based, clinical development and Foundation leaders, focuses on minimizing barriers to develop and approve agents and strategies for improved prevention, treatment and an eventual cure for NMO. Leaders in the pharmaceutical industry are invited to join the Foundation’s Industry Council.

2013 Industry Council Members

International Clinical Consortium members work toward developing and delivering improved medicines, therapeutic strategies and diagnostic tools for NMO.

COLLABORATivE CROssCURRENTs 42165

COUNTRiEs

wORKiNG GROUPs

REsEARChERs & CLiNiCiANs

2013 NMOBiOsAMPLE sETs

503 NMO PATiENT sETs

163 CONTROLs

www.guthyjacksonFoundation.org

Fund Sources and Uses

2013 Annual Reportwww.guthyjacksonFoundation.org

sOURCEs OF FUNDs AvAiLABLE FOR 2013CoNtRibutioNs by: AMouNt:

William R. Guthy $5,421,000

industry Council $49,980

General Public $74,293

Funds Carried Over from 2012 $378,958

TOTAL OF FUNDs AvAiLABLE TO ThE FOUNDATiON iN 2013 $5,924,231

2013 FUNDs UsED BY ThE FOUNDATiON

Funds Carried Over to 2014 $47,725

TOTAL FUNDs UsED FOR 2013 OR CARRiED OvER TO 2014 $5,924,231

Research Grants $5,536,345

Management Grants $299,621

Management and General $40,540 (paid directly by The Guthy-Jackson charitable Foundation)

$5,536,345

TOTAL MANAGEMENT AND GENERAL ExPENsEs $5,876,506

Management Grants $299,621

Management and General $40,540 (paid directly by The Guthy-Jackson charitable Foundation)

REsEARCh GRANTs

Management & General

5.79%$340,161

ResearchGrants

94.21% $5,536,345

www.guthyjacksonFoundation.org