LCA Community Conference
About the Conference
The Hope in Focus LCA Community Conference is a gathering dedicated to connection, learning, and shared hope within the LCA community. Each gathering, we bring together families, researchers, clinicians, advocates, and individuals living with LCA to exchange knowledge, share lived experiences, and strengthen the global community working toward understanding and treating Leber congenital amaurosis (LCA).
Our goal is to create a space where science and lived experience meet—where progress is shared openly, and where every story contributes to a stronger future.
We’re also excited for what’s ahead—stay tuned for updates and announcements about our next LCA Community Conference in 2027 as we continue growing this important community together.
Past Conferences
Over the years, Hope in Focus has hosted impactful conferences that have grown in both reach and depth. Each event has built on the last, expanding conversations around research, care, and community support.
LCA Family Conference 2025
June 20, 2025 – June 21, 2025 • Minneapolis, MN
This conference was centered on advancing understanding and accelerating treatments for inherited retinal diseases (IRDs), while strengthening the community of patients, families, clinicians, and researchers working together toward a future of restored vision and improved quality of life.
| PANELS: | SPEAKERS: |
| Living with an IRD We’re jumping right into it with a panel about mother-son and father-daughter relationships! Moderated by Hope in Focus co-founder and Board Chair Laura Manfre, our panelists include parents of children with an IRD and two young adults living with an IRD. Read the recap about this panel here. | Laura Manfre, Moderator • Lee St. Arnaud, LCA Parent • Elle St. Arnaud, LCA IQCB1/NPHP5, 2025 High School graduate • Leslie Morris, IRD Parent • Jack Morris, RP CRB1, 2025 Brown University graduate |
| Participating in a Clinical Trial This insightful panel will explore what it means to participate in a clinical trial. Hear directly from a clinician overseeing trials and a person living with LCA who has participated in one. Together, they’ll discuss the decision making process, what to expect when participating, and the personal and scientific impact of clinical research. Whether you’re considering a trial or simply curious, this session provides valuable perspectives from both the medical and patient perspectives. Read the recap about this panel here. | Ben Shaberman, Foundation Fighting Blindness, Moderator • Tomas Aleman, MD, University of Pennsylvania • Sarah McCabe, LCA2 RPE65 |
| Gene Therapies for LCA We’ll cover an overview of gene therapy, including the progress of current LCA gene therapies in the pipeline, as well as information about gene therapies that are transitioning from the clinic to approved treatments. Read the recap about this panel here. | Ben Shaberman, Moderator • Kenji Fujita, Atsena Therapeutics • Sarah Tuller, Opus Genetics • Dr. Bikash Pattnaik, University of Wisconsin-Madison |
| Emerging Therapies on the Horizon: Optogenetics, Cell Therapies & ASOs This panel will explore the development of cutting-edge treatments for inherited retinal diseases. Experts will break down the science behind optogenetics, cell therapies, and antisense oligonucleotides (ASOs), highlighting how these innovative approaches aim to preserve or restore vision. Read the recap about this panel here. | Amy Laster, Moderator • Dr. Raj Agrawal, Ray Therapeutics • Michael Schwartz, Sepul Bio • Ben Shaberman, Foundation Fighting Blindness |
| Bringing it All Together Hope in Focus will discuss how you can be involved in the active development of treatments, advocacy, and policy work, as we build an educated and empowered community. | • Courtney Coates, Hope in Focus • Laura Manfre, Hope in Focus |
During the panels Saturday, we provided the children and our chaperones to go to:
The Mall of America; The Crayola Experience & SEA LIFE Aquarium
Notable moments included:
- Our fun raffle on Saturday night
- Optional Field Trip to the Como Park Zoo & Conservatory
Find more pictures from this conference on our Facebook Album
LCA Family Conference 2023
June 23, 2023 – June 24, 2023 • Indianapolis, IN
This conference was focused on the latest advances in research, deepen your understanding of roles played by various organizations in developing treatments, and gain insight into ways an active patient community can support and accelerate treatment.
| PANELS: | SPEAKERS: |
| Advocating for Treatments What does it mean when industry, FDA, and others talk about the “voice of the patient”? What role can we, as a patient community, play in advancing treatments? Why is it critically important that those living with the disease are included in the drug development process? This session will answer these questions and more! Read the recap about this panel here. | Jonathan Stokes, Abbvie, Moderator Jacose Bell, Spark Therapeutics Wiley Chambers, Food and Drug Administration Jill Dolgin, Beacon Therapeutics Laura Manfre, Hope in Focus Emily Place, Mass Eye and Ear |
| Living with LCA Hear first-hand from those living with LCA. This panel will be moderated by a father, his two high school-aged children with vision loss due to LCA, a young professional, one of the first brave persons to go through a clinical trial for gene therapy, and a Teacher of the Visually Impaired (TVI). They will discuss and share their experiences on key topics, and they will include time for you to ask questions. Read the recap about this panel here. | Lee St. Arnaud, Moderator Beth Borysewicz, Educational Consultant State of Connecticut Department of Aging and Disability Services with Bureau of Education Services for the Blind Mohamed Farid, Founder and CEO, MKF Continuity Tami Morehouse, RPE65, volunteered for the first clinical trial for gene therapy Patrick St. Arnaud, IQCB1 / NPHP5 Mirielle St. Arnaud, IQCB1 / NPHP5 |
| LCA & Drug Development: Setting the Stage Our partners at the Foundation Fighting Blindness will talk about the background of LCA, what makes it unique, and how that affects therapy approaches followed by an overview of the drug development process explaining what it takes to turn research into treatments. Read the recap about this panel here. | Chad Jackson, Foundation Fighting Blindness Ben Shaberman, Foundation Fighting Blindness |
| Gene Agnostic Therapies During this session we will learn about therapies in development that can benefit multiple gene mutations and its potential impact for the LCA community. Read the recap about this panel here. | Chad Jackson, Moderator Huma Qamar, Ocugen Eric Daniels, Kiora Pharmaceuticals Ben Shaberman, Foundation Fighting Blindness |
| Gene Therapies for LCA This session will provide an overview of gene therapy, the current progress of gene therapies in the pipeline for LCA, and information about how gene therapies translate to the clinic as an approved treatment. Read the recap about this panel here. | Ben Shaberman, Moderator Shannon Boye, Atsena Therapeutics Ash Jayagopal, Opus Genetics Thomas Mendel, The Ohio State University Bikash Pattnaik, University of Wisconsin |
During the panels Saturday, we provided the children and our chaperones to go to:
The Children’s Museum
Notable moments included:
- “Party Like a Local” Dinner
- Optional Field Trip to the Indianapolis Zoo
Find more pictures from this conference on our Facebook Album
LCA Family Conference 2019
June 26, 2019 – June 28, 2019 • Philadelphia, PA
The Hope in Focus (formally Sofia Sees Hope) LCA Family Conference brought together families, researchers, clinicians, and advocates to share the latest advances in LCA and inherited retinal disease (IRD) research. Through educational panels, personal stories, and community discussions, the conference fostered connection, encouraged collaboration, and empowered families to play an active role in advancing treatments and advocacy.
| PANELS: | SPEAKERS: |
| Foundation Fighting Blindness Hear what our partners at the Foundation Fighting Blindness have been up to – from their approach to tackling tough problems to fight blindness, to exciting research updates for retinal disease. Read the recap about this panel here. | Ben Yerxa, CEO Foundation Fighting Blindness |
| The Long and Winding Road of the Human Genome Project and Gene Therapy Read the recap about this panel here. | Dr. Katherine High |
| Your Voice Matters! Hear from the US Food & Drug Administration, industry representatives, and rare disease organizations how patients and caregivers can make a real difference in moving treatments forward. Moderated by Annette Tonti, Executive Director of Sofia Sees Hope. Read the recap about this panel here. | Jamie Ring, Head, Patient Advocacy at Spark Therapeutics, Inc. Jill Dolgin, Head of Patient Advocacy at AGTC Emily Milligan, Executive Director at the Barth Syndrome Foundation Terri Booker, Advocate for Sickle Cell Anemia |
| One Disease, Many Approaches From gene augmentation, to gene editing, to oligonucleaotidetherapy, there’s more than one way to approach genetic therapy! The experts will walk you through these very different approaches to genetic treatment. Read the recap about this panel here. | Moderated by Brian Mansfield, Executive Vice President Research, interim Chief Scientific Officer at Foundation Fighting Blindness. Jean Bennett, MD, PhD Director, Center for Advanced Retinal and Ocular Therapeutics (CAROT), University of Pennsylvania Perelman School of Medicine; Professor, Ophthalmology and Cell & Developmental Biology Tomas Aleman, MD, University of Pennsylvania Perelman School of Medicine Mike Schwartz, M.S., MBA, ProQR Pam Stetkiewicz, VP Program Management, Editas Medicine |
| All About Clinical Trials What’s involved in getting a treatment approved for clinical trial? What’s it like to be in a trial? How will I know if it’s safe, or if it’s the right thing to do for me and my family? In this session, you’ll hear from experts about the safeguards in place for your protection, and from patients who have been in a trial and will share their personal experiences. | Moderated by Ben Shaberman, Senior Director, Scientific Outreach & Community Engagement at Foundation Fighting Blindness. Dr. Wiley Chambers, Supervisory Medical Officer in the Office of New Drugs at the FDA Tami & Michael Moorehouse, Phase 1 RPE65 Trial Subject LCA/RPE65 genetic therapy trial Michel Michaelides, MD, Head of Clinical Ophthalmology, MeiraGTX |
| Living with an IRD Breakout Groups We’ll set up these sessions based on interest. When you register, let us know which sessions are most interesting to you, or if there’s another topic you would like to hear about! | Living with an IRD: The Early Years Living with an IRD: The Teen Years Living with an IRD: The Adult Years Genetic testing & counseling Empowering your school-aged child through self-advocacy Connecting Online: The Good, The Bad, The OMG I’m Blocking You |
During the panels Saturday, we provided the children and our chaperones to go to:
LEGOLAND® Discovery Center
Notable moments included:
- “Party Like a Local” Dinner
- Cookies & Cocktails
- Sunday Morning Excursion
Find more pictures from this conference on our Facebook Album
LCA Family Conference 2018
October 5 – October 6, 2018 • Groton, CT
The 2018 Hope in Focus (formerly Sofia Sees Hope) Family Conference marked the organization’s first gathering dedicated to connecting individuals and families affected by LCA and inherited retinal diseases (IRDs). Bringing together patients, caregivers, researchers, and industry leaders, the conference focused on advances in research, the development of emerging treatments, and the important role the patient community plays in accelerating progress through advocacy, education, and collaboration.
| PANELS: | SPEAKERS: |
| IRD Milestones: Reasons to Be Excited At no time in history has there been more promising research applied to genetically inherited eye disease. Hear from our partners at the Foundation Fighting Blindness about how much easier it is today to access genetic testing and genetic counseling and the exciting research and trials that are underway. Read the recap about this panel here and here. | Dr. Brian Mansfield, Senior Vice President of Research, Foundation Fighting Blindness |
| The Road to Treatment: Understanding How Therapies Are Developed From research to federal approval, what does it take to develop and approve a new treatment for rare disease? We’ll explore the regulatory, clinical, and industry aspects so you have a deeper understanding of what is involved in developing treatments, including how rare disease is different. Read the recap about this panel here and here. | Moderator: JEFFREY FINMAN, Jupiter Point Pharma Consulting, LLC, Board Member, Sofia Sees Hope Panelists: • DR. WILEY CHAMBERS, Supervisory Medical Officer in the Office of New Drugs at the FDA • JENNIFER HUNT, Vice President of Clinical Operations, Editas Medicine • TAMI MOREHOUSE, Phase 1 RPE65 Trial Subject RPE65 genetic therapy trial |
Thank You to these speakers and organizations for their participation!
- Kristen Angell, Associate Director, Advocacy, National Organization for Rare Disorders (NORD)
- Beth Borysewicz, TVI for State of Connecticut and Sofia Sees Hope Board Director
- Dr. Wiley Chambers, Supervisory Medical Officer in the Office of New Drugs at the U.S. Food and Drug Administration
- Michael & Sela Cornell, Father and daughter from Chicago
- Jill Dolgin, Head of Patient Advocacy, AGTC
- Jeffrey Finman, Jupiter Point Pharma Consulting, LLC and Sofia Sees Hope Board Member
- Jennifer Hunt, Vice President of Clinical Operations, Editas Medicine
- Dr. Brian Mansfield, Senior Vice President of Research, Foundation Fighting Blindness
- Tami Morehouse, Phase 1 RPE65 Trial Subject RPE65 genetic therapy trial
- Jamie Ring, Head of Patient Advocacy, Spark Therapeutics
