Hope in Focus is pleased to bring our web series “Let’s Chat About …” to the Leber congenital amaurosis and rare inherited retinal disease communities. We’ve developed the series with those living with LCA and IRDs in mind, but we invite all members of our community, including those in research, industry, and the regulatory communities to join any of the sessions, as we look ahead to a common goal of advancing treatments for rare retinal disease.
Watch and Listen to Past Let’s Chat About Sessions
Co-Founder & President
Let’s Chat About … Opus Genetics
Ben Yerxa, PhD, Co-Founder & President of Opus Genetics speaks about the Opus Genetics’ business model, pipeline, manufacturing process, and their focus on tried and true methods.. Founded in 2021, Opus Genetics is a patient-first, science-driven gene therapy company tackling manufacturing obstacles standing in the way of treatments for ultra-rare blinding conditions.
State of Connecticut Department of Aging and Disability Services with Bureau of Education Services for the Blind
Let’s Chat About … Self-Advocacy and Supporting Your Child’s Education
Beth Borysewicz, Hope in Focus Board Director and Educational Consultant, State of Connecticut Department of Aging and Disability Services with Bureau of Education Services for the Blind. Beth found her passion working with the blind and visually impaired community when 3-year Sofia joined her preschool classroom. Beth now has more than 16 years of experience working with students across Connecticut. Beth discusses how to leverage the expanded core curriculum for independent living skills, supporting your child’s transition to adulthood, and sharing best practices for self-advocacy in the classroom and beyond.
Daniel de Boer
Let’s Chat About … ProQR’s work in treatments for inherited retinal disease
Daniel de Boer is Founder and Chief Executive Officer of ProQR, which was incorporated in 2012. Daniel is a serial entrepreneur and passionate advocate for rare disease patients. After one of his children was diagnosed with a rare disease, he started ProQR to develop RNA therapies for rare diseases. Under Daniel’s leadership, ProQR developed a platform that yielded a diversified pipeline of potential treatments for rare diseases and raised more than $400M in funding, including an IPO on Nasdaq. Before founding ProQR, Daniel was founder and Chief Executive Officer of several technology companies. Daniel is also co-funder and strategic advisor to Amylon Therapeutics and Wings Therapeutics, strategic advisor at Frame Therapeutics, Meatable, Algramo and a member of the advisory board at the Termeer Foundation. In 2018 Daniel was named “Emerging Entrepreneur of the Year” by EY. In 2019 Daniel was selected for the Young Global Leader program at the World Economic Forum.
Michel Michaelides, MD
Head of Clinical Ophthalmology
Let’s Chat About … gene therapies for LCA
Dr. Michaelides is Head of Clinical Ophthalmology at MeiraGTX. Mike is a founding member of MeiraGTx and a Professor of Ophthalmology at the University College London Institute of Ophthalmology in the Dept. of Genetics. He is a Consultant Ophthalmologist at Moorfields Eye Hospital (MEH) in the Depts. Of Inherited Eye Disease, Medical Retina and Paediatric Ophthalmology.
He is currently the principal investigator of three active interventional clinical trials and has 10 on-going ethically approved studies, including six in-house natural history studies and a multi-centre international natural history study.
Mike was awarded a Medical Retina and Genetics clinical fellowship at MEH (2008-2009) and a combined Ophthalmic Genetics and Paediatric Ophthalmology Clinical & Research fellowship at Casey Eye Institute, USA (2009-2010). He is a recipient of a career development award from the Foundation Fighting Blindness (USA) – an award which is rarely given to non-US applicants; and has gained membership of the highly prestigious Macular Society and Retina Society in the USA.
Edmond Chen, MD
Vice President, Clinical Development-Ophthalmology and Hematology
Let’s Chat About … CRISPR and gene editing technology
Dr. Chen is Vice President, Head of Clinical Development at Editas Medicine, overseeing an exciting and emerging portfolio that spans the therapeutic areas of hematology, oncology, ophthalmology, and neuroscience. Under his leadership, Editas has advanced EDIT-301 into the clinic for Sickle Cell Disease and Beta-Thalassemia. As a physician executive with 20+ years of combined clinical and industry experience, he has a track of success at companies, including Merck and Bayer. His therapeutic area and drug development expertise is deep and diverse, from rare disease and indications such as bronchiectasis, vasculitis, and pulmonary hypertension to large cardiovascular areas including congestive heart failure, thrombosis, and therapeutics for primary and secondary cardiovascular prevention.
He earned his M.D. at the University of California, San Francisco School of Medicine where he subsequently trained and practiced in internal medicine and cardiology. He holds a BA with Honors in Molecular and Cell Biology, Neurobiology, from the University of California, Berkeley.
Jill Dolgin, PharmaD
Executive Director, Global Patient Advocacy
Let’s Chat About … The importance of the patient voice in rare disease
Jill Dolgin, PharmD is the Head of Patient Advocacy at Applied Genetic Technologies Corporation (AGTC) which is a clinical-stage biotechnology company developing transformational genetic therapies for inherited retinal diseases. Dr. Dolgin leads the patient and professional engagement strategy to drive disease awareness and clinical trial recruitment efforts for the AGTC pipeline. She is responsible for incorporating the voice of the patient throughout drug development and corporate culture. She has over 20 years of global biopharmaceutical experience in Medical Affairs, Corporate Communications, Patient and Professional Advocacy, and Public Policy.
Her role within the company is to ensure that the needs of the patients are considered and incorporated into every aspect of drug development. Externally, Jill works with patient advocacy groups to educate patients and families about the importance of participating in clinical trials, gene therapy and the importance of “getting your voices heard” to help consumers, healthcare professionals, and policy makers understand your challenges in living with your disease. Dr. Dolgin earned a Doctorate in Clinical Pharmacy from the University of the Sciences at Philadelphia and a Bachelor of Science degree in Pharmacy from The Ohio State University.
Hope in Focus
Let’s Chat About … the importance of self-advocacy
In the world of rare disease, self-advocacy can be so important. But it is also overwhelming, intimidating, and scary. Tami Morehouse and Jack McCormick both have Leber congenital amaurosis due to mutations in their RPE65 gene. They joined us for a conversation about their own self-advocacy journeys.
Tami made research history when, at age 44, she participated in a clinical trial for gene therapy for LCA2 (RPE65). Ultimately, Spark Therapeutics developed the drug that was marketed as LUXTURNA® following Federal Drug Administration approval in December 2017. Tami volunteers in Hope in Focus’ Family Connections program, which connects people within the LCA community to share information and provide support. She works as an information and referral specialist for 211 of Ashtabula County, Ohio.
Jack graduated from Wilfrid Laurier University in Waterloo, Ontario, in 2018. He is a passionate advocate for inclusion and accessibility on all fronts. He initially tried to hide his blindness but that all ended when he got Jake, his beloved guide dog. In college he founded Eye To Eye, a student club whose goal is to illuminate stigma associated with vision impairments. He volunteers with Fighting Blindness Canada, including working on the 2018 Young Leaders Summit. He works in human resources for a major software company.
Vice President, Clinical & Outcomes Research
Foundation Fighting Blindness
Let’s Chat About … the importance of patient registries and My Retina Tracker
Todd Durham is the Vice President, Clinical & Outcomes Research at the Foundation Fighting Blindness, a national non-profit that funds research to treat and cure inherited retinal diseases. In his current role, Todd is responsible for directing the Foundation’s Clinical Consortium of retinal experts, developing strategies to enhance product development, partnering with industry, and providing technical input on partnered programs and investment decisions. Todd has over 25 years of drug development experience. Prior to his current position, he contributed to research on numerous marketed products as Director of Biostatistics with IQVIA’s Real World Evidence Solutions, was a doctoral fellow with Bristol Myers Squibb, and worked in various statistical and leadership roles for Novan, Inspire Pharmaceuticals, Quintiles, and as a self-employed consultant. Todd earned a BSPH and MS in biostatistics and a PhD in health policy and management (Decision Science and Outcomes Research) from the UNC Gillings School of Global Public Health.
Director, Patient-Centered Outcomes Research
Let’s Chat About … why natural history and patient outcome studies matter.
Jonathan Stokes has a devoted interest in understanding and bringing to light the patient voice and perspective, with over 16 years of research study design and implementation experience. His experience is primarily in health outcomes research; specifically, the development and evaluation of clinical outcomes of assessment (COAs) for use in clinical trials intended to substantiate product labeling goals, as well as use of COAs in real world clinical practice. Areas of focus include the evaluation of cardinal signs and symptoms of disease, health-related quality of life, improvements and activation in treatment adherence, understanding unmet need, and exploring the burden of disease. Jonathan holds an MBA from Northeastern University.
Licensed Genetic Counselor and Research Study Coordinator
Massachusetts Eye and Ear
Let’s Chat About … the ins and outs of genetic testing.
Emily Place graduated from the University of St. Thomas with a Bachelor of Arts in Biology. She went on to earn her master’s degree in Human Genetics from Sarah Lawrence College. Prior to working at the Ocular Genomics Institute, she worked as a pediatric genetic counselor at The Children’s Hospital of Philadelphia.
Emily joined the OGI in 2011, where she provides risk assessment and genetic counseling services to families with inherited retinal dystrophies. She also serves as the study coordinator for the research study of molecular genetics of inherited retinal dystrophies.
Wiley A. Chambers, MD
Supervisory Physician, Division of Ophthalmology
U.S. Food and Drug Administration
Let’s Chat About … What it takes to receive approval for a new treatment for rare disease.
Wiley A. Chambers, MD, is Supervisory Physician in the Division of Ophthalmology at the Food and Drug Administration (FDA). After receiving an undergraduate degree from Colgate University, Dr. Chambers completed medical school and a residency in Ophthalmology at The George Washington University School of Medicine and Health Sciences in Washington, DC. He is currently a Clinical Professor of Ophthalmology and Adjunct Assistant Professor of Computer Medicine at The George Washington University. He joined the FDA in 1987, as a primary reviewer for ophthalmic drug products and in 1990 became a Supervisory Medical Officer for Ophthalmologic Drug Products. In this capacity, Dr. Chambers has supervisory responsibility for the clinical review of ophthalmologic drug products and ophthalmic therapeutic biologic products submitted to the Center for Drug Evaluation and Research. Additionally, Dr. Chambers is the recipient of numerous Public Health Service, FDA and Center for Drug Evaluation and Research awards for his work with the FDA and he has served as the American Academy of Ophthalmology’s Delegate to past United States Pharmacopeia Conventions.
Senior Director, Scientific Outreach & Community Engagement
Foundation Fighting Blindness
Let’s Chat About … What’s in the therapy pipeline for Leber congenital amaurosis.
For 16 years, Ben has been reporting on retinal research for all of FFB’s electronic and print publications. In addition, he presents the latest scientific advancements at local and national events for patients and families, and conducts various training activities for staff and constituents. He enjoys working with constituents one-on-one to help them understand their retinal disease and the research underway that may benefit them. Ben also leads the company’s outreach to eye care professionals throughout the United States.
Ben has written three books – Retina Boy, Jerry’s Vegan Women, and The Vegan Monologues – all published by Loyola University (Maryland). He earned a Master of Arts in writing from Johns Hopkins University, a Master of Science in systems management from the University of Maryland, and a Bachelor of Science in computer information science from Cleveland State University.