the monaciano symposium - Fondo Studenti Italiani

Transcription

the monaciano symposium - Fondo Studenti Italiani
THE MONACIANO SYMPOSIUM
PONTE A BOZZONE
SIENA, ITALY
OCTOBER 1–4, 2013
THE MONACIANO SYMPOSIUM
A Strategic Planning Retreat to Advance the Treatment of Hereditary Retinal Degeneration
Dear Participants,
On behalf of Celeste and Bruno Piperno, the symposium organizing committee would like to welcome
you to Tenuta di Monaciano. The beautiful villa, houses, and grounds of this estate are the site
for our meeting of experts focused on the development of treatments for retinal dystrophies and
degenerations. The past ten years have seen tremendous progress toward our shared goals. Genereplacement therapy, and to some extent stem-cell therapy, have been transformed from a dream
into an evolving reality. As a result, the field has reached a critical juncture, with many challenges
and opportunities ahead. The Monaciano Symposium is aimed at defining these challenges, and at
identifying the strategies and solutions needed to move the field forward in the next ten years.
The agenda for our meeting was developed to reflect the priorities indicated by your responses to
the survey conducted earlier this year. By now meeting together, we hope to further delineate these
priorities and to build the consensus needed to solve the critical problems that confront us. A planned
outcome of our meeting is a “white paper” that will report our recommendations to the international
vision community. In addition, we hope that new opportunities for collaboration will be identified, and
that existing collaborations will be deepened as a result of our deliberations.
It is thus with sincere pleasure that we welcome this distinguished group to what promises to be a
rich and productive meeting where we will create a blueprint for our work in the years ahead.
Sincerely,
The Symposium Organizing Committee
Robin R. Ali, Ph.D. William W. Hauswirth, Ph.D.
John R. Heckenlively, M.D. Paul P. Lee, M.D., J.D. Paul R. Lichter, M.D.
Alessandro Iannaccone, M.D., M.S.
Debra A. Thompson, Ph.D.
David N. Zacks, M.D., Ph.D.
THE HOSTS OF THE MONACIANO SYMPOSIUM
Celeste and Bruno Piperno
It has long been a dream of Celeste and Bruno Piperno to host a meeting at their beloved
Tenuta di Monaciano that would advance the treatment of retinal degenerative diseases.
As steadfast advocates for patients in Rome affected by these diseases, they are known for
their personal commitment and dedication to making a difference in the lives of many individuals
in their community.
Through their close ties and friendships throughout Europe and the United States, Celeste and
Bruno have come to understand the importance of international collaborative efforts in advancing
translational studies. They identified the goal of bringing together leaders in retinal dystrophy
research in an ideal location in which the existing challenges and shared goals needed to move
the field forward could be defined.
Tenuta di Monaciano is that location, a jewel in the Tuscan countryside complete with working
vineyards and an elegant villa that has been in the Piperno family since early in the 20th century.
Celeste and Bruno have opened the doors of their villa for our meetings, and are providing meals
and accommodations for the participants and guests in the houses of the estate.
We owe our deep thanks to Celeste and Bruno for imagining and making the Monaciano
Symposium a reality. The most meaningful thanks we can offer will be our progress toward
making another of their long-held dreams come true — the successful treatment of retinal
dystrophy patients.
AGENDA
TUESDAY, OCTOBER 1
Reception
3:00 p.m. – 4:00 p.m.
Session A 4:00 p.m. – 6:00 p.m.
Welcome
Participants and guests
Alessandro Iannaccone
Presentation of the meeting charge
Introductory lectures: Progress and challenges
John Heckenlively, Debra Thompson,
Paul Lichter
Dinner
at Monaciano
7:00 – 9:00 p.m.
Robin Ali, William Hauswirth
WEDNESDAY, OCTOBER 2
Session B
8:30 a.m. – 12:30 p.m.
Topic 1: Identifying the most compelling therapeutic targets
Discussion leaders:
Robert Molday, Thomas Reh
8:30 a.m. – 9:00 a.m.
Introduction by discussion leaders
9:00 a.m. – 9:50 a.m.
Small group discussions
Coffee Break
9:50 a.m. – 10:10 a.m.
Topic 2: Refining and expanding Discussion leaders:
therapeutic strategies
John Flannery, David Gamm
10:10 a.m. – 10:40 a.m.
Introduction by discussion leaders
10:40 a.m. – 11:30 a.m.
Small group discussions
11:30 a.m. – 12:30 p.m.
Solution reporting by small group
leaders; large group discussion
Small group leaders Topics 1 & 2:
Lunch
12:30 p.m. – 1:30 pm
Isabelle Audo, Alberto Auricchio,
Eyal Banin, Deniz Dalkara, Henry Klassen,
Enrica Strettoi, Debra Thompson,
Richard Weleber
Session C
1:30 p.m. – 4:00 p.m.
Topic 3: Streamlining regulatory processes and solving infrastructure needs
1:30 p.m. – 2:20 p.m.
2:20 p.m. – 3:00 p.m.
Coffee Break 3:00 p.m. – 3:20 p.m.
3:20 p.m. – 4:00 p.m.
Panel discussion
Leader: David Zacks
Members: Robin Ali, William Hauswirth, Naheed Khan, Bart Leroy, Stuart Naylor
Panel discussion and Q & A
Small group discussions
Solution reporting and large group discussion
Small group leaders Topic 3:
Stephen Daiger, Elise Heon,
Thiran Jayasundera,
Henry Klassen, András Komáromy,
Michel Michaelides,
Simon Petersen-Jones, Paul Sieving
Dinner/Tour
at Castello di Brolio (travel by coach)
5:00 p.m. – 9:30 p.m. THURSDAY, OCTOBER 3
Session D
8:30 a.m. – 12:00 noon
Topic 4: Identifying the patients who could benefit from treatment
Discussion leaders:
Kari Branham, Thiran Jayasundera,
Richard Weleber
8:30 a.m. – 9:00 a.m. Introduction by discussion leaders
9:00 a.m. – 9:50 a.m. Small group discussions
Coffee Break
9:50 a.m. – 10:10 a.m.
THURSDAY, OCTOBER 3 (cont)
Topic 5: Formalizing outcome measures Discussion leaders:
John Heckenlively, Mark Pennesi,
Paul Sieving
10:10 a.m. – 10:40 a.m.
Introduction by discussion leaders
10:40 a.m. – 11:30 a.m.
Small group discussions
11:30 a.m. – 12:30 p.m.
Solution reporting by small group leaders;
large group discussion
Small group leaders Topics 4 & 5:
Eyal Banin, Deniz Dalkara,
Alessandro Iannaccone, Naheed Khan,
András Komáromy, Bart Leroy,
Michel Michaelides, Benedetto Falsini
Lunch
12:30 p.m. – 1:30 p.m.
Session
E
1:30 p.m. – 4:00 p.m.
Topic 6: Optimizing collective efforts: Enhancing funding, increasing collaboration, reducing redundancy Panel discussion
Leader: Eyal Banin
Members: Alberto Auricchio,
Stephen Daiger, John Flannery, Elise Heon
1:30 p.m. – 2:20 p.m.
2:20 p.m. – 2:40 p.m.
Coffee Break 2:40 p.m. – 3:00 p.m.
3:00 p.m. – 4:00 p.m.
Panel discussion Q & A
Small group leaders Topic 6: Robin Ali, William Hauswirth, David Gamm, Robert Molday, Simon Petersen-Jones,
Rajesh Rao, Thomas Reh, David Zacks
Dinner at Monaciano
7:00 – 9:00 p.m. Small group discussions
Solution reporting and
large group discussion
FRIDAY, OCTOBER 4
Session F
8:30 a.m. – 10:30 a.m.
Topic 7: Recommendations
report planning
8:30 a.m. – 9:00 a.m
Topic 1 & 2 small group leaders
Discussion leaders:
John Heckenlively,
Alessandro Iannaccone, Paul Lichter
Therapeutic targets and strategies
9:00 a.m. – 9:30 a.m.
Topic 3 small group leaders
Advancing regulatory and
treatment issues
9:30 a.m. – 10:00 a.m.
Topic 4 & 5 small group leaders
Patient identification/
characterization and outcomes
analysis
10:00 a.m. – 10:30 a.m.
Topic 6 small group leaders
Increasing collaboration and
shared funding
Departure
Airport shuttle available by request
10:30 a.m. – 11:00 a.m.
WEDNESDAY AM —Session B (Topics 1 & 2)
Red Group
Blue Group
Green Group
Yellow Group
Audo (Leader)
Auricchio (Leader)
Banin (Leader)
Dalkara (Leader)
Klassen (Leader)
Strettoi (Leader)
Thompson (Leader)
Weleber (Leader)
Branham
Daiger
Jayasundera
Ali
Flannery
Gamm
Michaelides
Falsini
Hauswirth
Khan
Sieving
Heckenlively
Heon
Leroy
Rao
Iannaccone
Komáromy
Petersen-Jones
Reh
Molday
Pennesi
Zacks
Naylor
WEDNESDAY PM —Session C (Topic 3)
Red Group
Blue Group
Green Group
Yellow Group
Daiger (Leader)
Klassen (Leader)
Petersen-Jones (Leader)
Komáromy (Leader)
Jayasundera (Leader)
Sieving (Leader)
Heon (Leader)
Michaelides (Leader)
Audo
Banin
Auricchio
Ali
Branham
Flannery
Leroy
Dalkara
Falsini
Gamm
Naylor
Heckenlively
Hauswirth
Khan
Pennesi
Molday
Iannaccone
Rao
Reh
Zacks
Weleber
Strettoi
Thompson
THURSDAY AM —Session D (Topics 4 & 5)
Red Group
Blue Group
Green Group
Yellow Group
Khan (Leader)
Banin (Leader)
Iannaccone (Leader)
Dalkara (Leader)
Michaelides (Leader)
Leroy (Leader)
Komáromy (Leader)
Falsini (Leader)
Ali
Auricchio
Audo
Gamm
Branham
Hauswirth
Daiger
Heckenlively
Flannery
Jayasundera
Klassen
Heon
Pennesi
Petersen-Jones
Thompson
Molday
Reh
Rao
Weleber
Naylor
Sieving
Zacks
Strettoi
THURSDAY PM —Session E (Topic 6)
Red Group
Blue Group
Green Group
Yellow Group
Ali (Leader)
Rao (Leader)
Hauswirth (Leader)
Gamm (Leader)
Molday (Leader)
Petersen-Jones (Leader)
Reh (Leader)
Zacks (Leader)
Dalkara
Branham
Banin
Audo
Falsini
Flannery
Khan
Auricchio
Heon
Iannaccone
Komáromy
Daiger
Heckenlively
Jayasundera
Klassen
Leroy
Weleber
Michaelides
Pennesi
Naylor
Sieving
Thompson
Strettoi
PRE-MEETING SURVEY RESPONSES
Therapeutic Intervention in Inherited Retinal Degeneration:
Strategies, Opportunities, Challenges, Needs
1.
the
most
compelling
therapeutic
strategies
1. IIdentifying
den2fying the most compelling therapeu2c strategies 0.0% 10.0% 20.0% 30.0% 40.0% 50.0% 60.0% Which biological mechanisms and pathways are the most accessible targets? 63.0% Which aspects of pathology can be delayed or reversed? 63.0% Is cell therapy the treatment of choice for rare forms of re2nal dystrophy? What are the most promising strategies for improving delivery of therapeu2c agents? 55.6% 55.6% What are the treatment op2ons for early-­‐ vs. advanced-­‐stage disease? 48.1% What can be done to prevent or delay cone photoreceptor cell death in rod-­‐cone degenera2ons? 40.7% What informa2on can be obtained that could help match the 2ming of treatment to the therapeu2c window of opportunity? 25.9% Which small molecule therapeu2cs are the best candidates for future development? Which currently available tradi2onal forms of therapy should be more widely employed to improve quality of life and/or prolong the therapeu2c window of opportunity? 14.8% 14.8% 70.0% 2. Maximizing the identification of patients likely to benefit from treatment
0.00% 10.00% 20.00% 30.00% 40.00% 50.00% 60.00% 70.00% 80.00% What criteria should be used to select the subset of pa>ents who would benefit most from a given therapy (e.g., muta>on, gene, effects)? What can be done to facilitate the iden>fica>on of eligible pa>ents who meet study criteria for inclusion in specific clinical trials? How can we establish a shared database of pa>ent muta>ons that could be used to enhance recruitment of eligible pa>ents for inclusion in specific clinical trials? What strategies are needed to provide newly approved therapies to large numbers of pa>ents, including those in underdeveloped countries? 73.10% 69.20% 46.20% 46.20% How can more eligible pa>ents gain access to currently available tradi>onal forms of therapy that could reduce or delay their symptoms? Are rigorous studies of disease natural history needed to improve our ability to evaluate the effec>veness of targeted interven>ons? What resources are available for studies of disease natural history? 23.10% 19.20% 11.50% 3. Formalizing outcome measures and facilitating regulatory approval
0.00% 10.00% 20.00% 30.00% 40.00% 50.00% 60.00% 70.00% 80.00% What is the reproducibility and reliability of currently available outcome measures for detecAng therapeuAc benefit? 73.10% Which funcAonal tests are essenAal for opAmizing the evaluaAon of outcomes in clinical trials, while at the same Ame reducing the burden on paAents? 61.50% What addiAonal detecAon technologies or paradigms need to be developed to more quickly and meaningfully measure therapeuAc outcomes? 46.20% How should controls be designed for studies evaluaAng the effects of systemically delivered therapeuAcs, including supplements? 38.50% What technologies and tesAng protocols should be set for child paAents who cannot reliably perform tests that adults can? 34.60% What are the possibiliAes for streamlining regulatory requirements as the field develops? 30.80% 4. Enhancing collaboration and reducing redundancy of effort
0.00% 10.00% 20.00% 30.00% 40.00% 50.00% 60.00% 70.00% 80.00% What kinds of collabora=ve interac=ons would be needed to accelerate progress and reduce costs? 70.40% What are the typical road blocks to collabora=on for which solu=ons can be iden=fied and widely applied? 44.40% What mechanisms can be put in place to enhance resource sharing and coordinate project planning? What criteria and mechanisms should be used to priori=ze the selec=on of target genes? 40.70% 37.00% Should common protocols and FDA paperwork be prepared so that each center does not have to duplicate the same work? 33.30% What are the advantages and disadvantages of centralized produc=on of therapeu=c agents? 29.60% What infrastructure requirements will be needed to support large-­‐scale treatment of pa=ents? 18.50% 5. Enhancing funding & fundraising processes to optimize collective efforts
0.00% 10.00% 20.00% 30.00% 40.00% 50.00% 60.00% How can the field a9ract disease-­‐neutral donors to fund research development and clinical trials? What can be done to reduce the cost of treaFng paFents? 65.40% 61.50% Is there a financial model that could distribute funding across mulFple centers that have the capacity to contribute to progress on specified projects? 57.70% What financial model could be developed to provide therapeuFc coverage for the largest number of genes and paFents? 42.30% How can we use the Internet to raise funding for joint efforts targeted to specified projects in underdeveloped countries? 19.20% How can we tap into exisFng medical infrastructure and resources for joint efforts targeted to specified projects in underdeveloped countries? 7.70% 70.00% 6. Are there additional questions you would like to include, and/or an issue that you feel is
essential for the meeting to address?
Reappraising the role of secondary autoimmune complications in hereditary retinal degenerations -- there
is ample evidence that this is a true and often very important issue and therapeutic target.
Does early intervention slow or prevent disease progression in a genetic photoreceptor disease?
What are the disease and/or genetic features that make one therapeutic strategy (e.g. comparison of
different gene therapy vectors, cell therapies and therapeutic proteins or small molecules) preferable to
others?
What is the role and importance of large animal models? Considering the cost, shall resources be
invested in the development of new large animal models?
Are combination therapies worth pursuing at this time?
How can we lobby national governments to help finance gene therapy, stem cell therapy and subretinal
implant technology to aid our patients?
How do we get inexpensive yet accurate genetic testing on patients?
How much will new treatments cost? What cost/benefit can be justified. Discussion about QALYs
We should explore the potential of multi-center grants from the NIH, such as the R24 mechanism for
developing new collaborative trials.
How to optimize the identification, development and use of animal models for preclinical testing.
What are the outcomes that we can reasonably expect, and how do we manage expectations/avoid
hype?
Are there ways to streamline the IND process?
Security protected website to help with collaborations, sharing information, and agreed upon data
storage.
How can we define the optimal window of opportunity for therapeutic intervention?
What are the most important environmental factors influencing the disease progression, and how can we
modulate them? (For instance: nutritional aspects, exposure to oxidative damage, etc.)
7. What are some key outcomes you would like the meeting to achieve?
Ways to accelerate and facilitate clinical application of promising novel therapeutic interventions.
Identify priorities for the years to come and define strategies to achieve them.
Find ways to enhance collaboration between investigators to maximize access to patients with rare
conditions.
Publish the outcome of our meeting to impact the course of research in the field for the next decade or
so.
A realistic perspective of the relative strengths and difficulties in implementing various approaches to the
treatment of retinal disease.
1. Identify ways to enhance collaboration and reduce redundancy. 2. Identify ways to develop a patient
database. 3. Identify novel outcome measures that reflect clinically relevant improvements.
Recommendations about specific role that comparative ophthalmology group can plan in advancing the
overall mission of the field.
More interactions and collaborations; more streamlined procedures to get potential therapeutic
treatments to clinical trials.
To help establish how we shall be able to fund further development of gene therapy, stem cell therapy
and treatment with subretinal implants to help more patients see.
Develop collaborations, enhance mechanisms for resource and information sharing.
Develop a more collaborative approach to investigating these diseases.
Agreement over stringent therapeutic outcome measures.
People’s views on current trials. People’s views on best emerging therapeutic approaches (for given
diseases). Scope and feasibility of collaborative ideas.
Guidelines for best testing methods in different patient populations (based on age or disease).
More effective collaboration between groups.
Identify tangible mechanisms of collaboration to move the field forward in a defined manner.
Consensus statements on as many of the issues raised in 1-8 as possible.
Some new collaborative efforts, and agreement on sharing some resources.
Promotion of a collaborative spirit and identification of complementary expertise.
8. Are there any key papers that you think everyone should be aware of?
Adamus G, Wang S, Kyger M, Worley A, Lu B, Burrows GG. Systemic immunotherapy delays photoreceptor cell loss and
prevents vascular pathology in Royal College of Surgeons rats. Mol Vis. 2012;18:2323-37. http://www.molvis.org/molvis/v18/
a246.
Berger W, Kloeckener-Gruissem B, Neidhardt J. The molecular basis of human retinal and vitreoretinal diseases. Prog. Ret.
Eye Res. 2010;29:335-375.
http://dx.doi.org/10.1016/j.preteyeres.2010.03.004.
Birch DG, Locke KG, Wen Y, Locke KI, Hoffman DR, Hood DC. Spectral-Domain Optical Coherence Tomography Measures of
Outer Segment Layer Progression in Patients With X-Linked Retinitis Pigmentosa. JAMA Ophthalmol. 2013;
http://dx.doi.org/10.1001/jamaophthalmol.2013.4160.
Cideciyan AV, Jacobson SG, Beltran WA, Sumaroka A, Swider M, Iwabe S, Roman AJ, Olivares MB, Schwartz SB,
Komáromy AM, Hauswirth WW, Aguirre GD. Human retinal gene therapy for Leber congenital amaurosis shows advancing
retinal degeneration despite enduring visual improvement. Proc Natl Acad Sci U S A 2013; 110:517-525.
http://dx.doi.org/10.1073/pnas.1218933110.
Csaky KG, Richman EA and Ferris FL, 3rd. Report from the NEI/FDA Ophthalmic Clinical Trial Design and Endpoints
Symposium. Invest Ophthalmol Vis Sci 2008;49:479-89.
http://dx.doi.org/10.1167/iovs.07-1132.
Eiraku M, Takata N, Ishibashi H, Kawada M, Sakakura E, Okuda S, Sekiguchi K, Adachi T, Sasai Y. Self-organizing optic-cup
morphogenesis in three-dimensional culture. Nature. 2011;472:51-6.
http://dx.doi.org/10.1038/nature09941.
Kyger M, Worley A, Adamus G. Autoimmune responses against photoreceptor antigens during retinal degeneration and
their role in macrophage recruitment into retinas of RCS rats. J Neuroimmunol. 2013;254:91-100.
http://dx.doi.org/10.1016/j.jneuroim.2012.10.007.
Pearson RA, Barber AC, Rizzi M, Hippert C, Xue T, West EL, Duran Y, Smith AJ, Chuang JZ, Azam SA, Luhmann UF, Benucci
A, Sung CH, Bainbridge JW, Carandini M, Yau KW, Sowden JC, Ali RR. Restoration of vision after transplantation of
photoreceptors. Nature. 2012;485:99-103.
http://dx.doi.org/10.1038/nature10997.
Ratnam K, Carroll J, Porco TC, Duncan JL, Roorda A. Relationship between Foveal Cone Structure and Clinical Measures of
Visual Function in Patients with Inherited Retinal Degenerations. Invest Ophthalmol Vis Sci. 2013;54:5836-47.
http://dx.doi.org/10.1167/iovs.13-12557.
Schachar IH, Zahid S, Comer GM, Stem M, Schachar AG, Saxe SJ, Gardner TW, Elner VM, Jayasundera T. Quantification
of Fundus Autofluorescence to Detect Disease Severity in Nonexudative Age-Related Macular Degeneration. JAMA
Ophthalmol. 2013;131:1009-15.
http://dx.doi.org/10.1001/jamaophthalmol.2013.4014.
Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA,
Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau
P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D,
Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski
J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Translating the genomics
revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther. 2013;21:266-8.
http://dx.doi.org/10.1038/mt.2013.4.
Zahid S, Khan N, Branham K, Othman M, Karoukis AJ, Sharma N, Moncrief A, Mahmood MN, Sieving PA, Swaroop A,
Heckenlively JR, Jayasundera T. Phenotypic Conservation in Patients With X-Linked Retinitis Pigmentosa Caused by RPGR
Mutations. JAMA Ophthalmol. 2013;131:1016-25.
http://dx.doi.org/10.1001/jamaophthalmol.2013.120.
PARTICIPANTS
Robin R. Ali, Ph.D.
Div of Molecular Therapy
UCL Institute of Ophthalmology
11-43 Bath Street
London, England EC1V 9EL United Kingdom
442076086817
r.ali@ucl.ac.uk
Deniz Dalkara, Ph.D.
Therapeutics
Institut de la Vision/INSERM
17 rue Moreau
Paris, 75012 France
5106420209
deniz.dalkara@inserm.fr
Isabelle S. Audo, M.D., Ph.D.
Department of Genetics
Institut de la Vision /INSERM
/UPMC/CNRS/CHNO
17 Rue Moreau
Paris, 75012 France
33153462540
isabelle.audo@inserm.fr
Benedetto Falsini, M.D.
Ophthalmology
Catholic University
Lgo Vito 1
Rome, 00168 Italy
390630154929
bfalsini@rm.unicatt.it
Alberto Auricchio, M.D.
Tigem Fondazione Telethon
Via Carlo Spinola 16
Rome, 00154 Italy
39-081-6132228
auricchio@tigem.it
John Gerard Flannery, Ph.D.
Helen Wills Neuroscience Institute
University of California-Berkeley
132 Barker Hall MC3190
Berkeley, CA 94720-3190 United States
(510)642-0178
flannery@berkeley.edu
Eyal Banin, M.D., Ph.D.
Ophthalmology
Hadassah-Hebrew University Med Ctr
Ein-Karem PO Box 12000
Jerusalem, 91120 Israel
(972)2-6776585
banine@cc.huji.ac.il
David M. Gamm, M.D., Ph.D.
Ophthalmology and Visual Sciences
University of Wisconsin-Madison
1500 Highland Ave, Rm 621
Madison, WI 53705-2280 United States
(608)261-1516
dgamm@wisc.edu
Kari E. Branham, M.S.
Ophthalmology and Visual Sciences
University of Michigan Medical School
Kellogg Eye Center
1000 Wall St
Ann Arbor, MI 48105 United States
(734)936-8638
haag@umich.edu
William W. Hauswirth, Ph.D.
Department of Ophthalmology
University of Florida College of Medicine
Box 100284 JHMHC
Gainesville, FL 32610-0284 United States
(352)392-0679
hauswrth@eye.ufl.edu
Stephen P. Daiger, Ph.D.
Human Genetics Center, School Pub Hlth
University of Texas Hlth Sci Ctr Houston
1200 Herman Pressler Drive
Houston, TX 77030 United States
(713)500-9829
stephen.p.daiger@uth.tmc.edu
John R. Heckenlively, M.D.
Ophthalmology and Visual Sciences
University of Michigan Medical School
Kellogg Eye Center
1000 Wall St, Rm 326
Ann Arbor, MI 48105 United States
(734)763-2280
jrheck@umich.edu
Elise Heon, M.D.
Ophthalmology and Vision Sciences
Hospital for Sick Children
555 University Ave
Toronto, ON M5G 1X8 Canada
(416)8138606
elise.heon@sickkids.ca
Alessandro Iannaccone, M.D., M.S.
Ophthal/Hamilton Eye Institute
University of Tennessee Health Sci Ctr
930 Madison Ave Ste 731
Memphis, TN 38163 United States
(901)448-7831
iannacca@uthsc.edu
Thiran Jayasundera, M.D., FRCSC FRANZCO
Ophthalmology and Visual Sciences
University of Michigan Medical School
Kellogg Eye Center
1000 Wall Street
Ann Arbor, MI 48105 United States
(734)7302453
thiran@umich.edu
András Komáromy, DrMetVet, Ph.D.
Small Animal Clinical Studies
Michigan State University
College of Veterinary Medicine
D-208 Veterinary Med Center
East Lansing, MI 48824-1314 United States
(517)353-5420
komaromy@cvm.msu.edu
Bart Peter Leroy, M.D., Ph.D.
Dept Ophthalmology & Ctr Med Genetics
Ghent University Hospital & University
De Pintelaan 185
Ghent, 9000 Belgium
3293322311
bart.leroy@ugent.be
Paul R. Lichter, M.D.
Ophthalmology and Visual Sciences
University of Michigan Medical School
Kellogg Eye Center
1000 Wall St
Ann Arbor, MI 48105 United States
(734)764-6468
plichter@umich.edu
Naheed W. Khan, Ph.D.
Ophthalmology and Visual Sciences
University of Michigan Medical School
Kellogg Eye Center
1000 Wall Street, Rm 346
Ann Arbor, MI 48105 United States
(734)763-0691
nwkhan@umich.edu
Michel Michaelides, M.D., FRCOphth
Medical Retina
Moorfields Eye Hospital
162 City Road
London, England EC1V 2PD United Kingdom
44 207 608 6885 (6866)
michel.michaelides@ucl.ac.uk
Henry J. Klassen, M.D., Ph.D.
Ophthalmology
University of California-Irvine
843 Hlth Sci Rd Hewitt Hall, Rm 2014
Irvine, CA 92697-4390 United States
(949)824-7750
hklassen@uci.edu
Robert S. Molday, Ph.D.
Biochemistry/Molecular Biology
University of British Columbia
2350 Health Sciences Mall
Vancouver, BC V6T 1Z3 Canada
(604)822-6173
molday@mail.ubc.ca
Stuart Naylor, Ph.D.
London, England
United Kingdom
44(0)7501 011680
stun.naylor@gmail.com
Mark E. Pennesi, M.D., Ph.D.
Ophthalmology
Casey Eye Institute
Oregon Health Science Center
3375 SW Terwilliger Blvd
Portland, OR 97239 United States
(415)676-1721
pennesim@ohsu.edu
Debra A. Thompson, Ph.D.
Ophthalmology and Visual Sciences
University of Michigan Medical School
Kellogg Eye Center
1000 Wall St
Ann Arbor, MI 48105-0714 United States
(734)936-9504
dathom@umich.edu
Simon M. Petersen-Jones, DVetMed, Ph.D.
Small Animal Clinical Sciences
Michigan State University
College of Veterinary Medicine
736 Wilson Rd, D-208
East Lansing, MI 48824-1314 United States
(517)353-3278
peter315@cvm.msu.edu
Richard G. Weleber, M.D.
Ophth & Molecular Med Genetics
Casey Eye Institute
Oregon Health Science Center
3375 SW Terwilliger Blvd
Portland, OR 97221 United States
(503)494-3016
weleberr@ohsu.edu
Rajesh Rao, M.D.
Ophthalmology and Visual Sciences
University of Michigan Medical School
Kellogg Eye Center
1000 Wall St
Ann Arbor, MI 48105-0714 United States
(734)763-8122
rajeshr@med.umich.edu
David N. Zacks, M.D., Ph.D.
Ophthalmology and Visual Sciences
University of Michigan Medical School
Kellogg Eye Center
1000 Wall St
Ann Arbor, MI 48105 United States
(734)763-7711
davzacks@umich.edu
Thomas A. Reh, Ph.D.
Department of Biological Structure
University of Washington
I-516 Hlth Sci Bldg; 1959 NE Pacific St
Seattle, WA 98195 United States
(206)543-5069
tomreh@u.washington.edu
Paul A. Sieving, M.D., Ph.D.
National Eye Institute NIH
31 Center Dr, MS 2510
Bethesda, MD 20892-0001 United States
(301)496-2234
pas@nei.nih.gov
Enrica Strettoi, Ph.D.
CNR Neuroscience Institute
Area della Ricerca CNR, Via G Moruzzi 1
Pisa, 56100 Italy
390503153213
MEETING STAFF
MargaretAnn Cross
Scribe/Writer
University of Michigan Health System
Ann Arbor, MI 48105 United States
(734)546-3364
margcros@med.umich.edu
Sofia Piperno
Rome, Italy
Cameron Strong
Ann Arbor, MI USA
ACKNOWLEDGMENTS
Support for the Monaciano Symposium was provided by:
Celeste and Bruno Piperno
University of Michigan Department of Ophthalmology and Visual Sciences
University of Tennessee Health Science Center, Hamilton Eye Institute
University of Michigan Office of the Vice President for Research
Lions International of Tuscany
Paul P. Lee, M.D., J.D.
A special thanks to Dierdre Jeske for providing outstanding administrative assistance,
to Lisa Burkhart for developing the meeting website and survey, and to Nancy Mariani and
Pina Vuocolo Giuseppina for handling logistics at the estate.