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Protection, Correction, Regeneration: Are combination therapies in the future for Inherited Retinal Degenerations?

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AuthorBlock: Randy M. Wheelock1, Paul A. Sieving8, Tomas S. Aleman2,3, Jacque L. Duncan7, Ian M. MacDonald4, Mark E. Pennesi9, Stephen H. Tsang5,6
1The Choroideremia Research Foundation, Inc., Johnson City, Tennessee, United States; 2Scheie Eye Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, United States; 3Center for Advanced Retinal and Ocular Therapeutics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, United States; 4Opthalmology and Visual Sciences, The University of Alberta, Edmonton, Alberta, Canada; 5Opthalmology, Columbia University, New York, New York, United States; 6Pathology and Cell Biology, Columbia University, New York, New York, United States; 7Ophthalmology, University of California San Francisco, San Francisco, California, United States; 8Office of the Director, National Eye Institute/National Institutes of Health, Bethesda, Maryland, United States; 9Casey Eye Institute, Oregon Health and Science University, Portland, Oregon, United States;

DisclosureBlock: Randy M. Wheelock, 4D Molecular Therapeutics, Inc. Code I (Personal Financial Interest) , Paul A. Sieving, None; Tomas S. Aleman, None; Jacque L. Duncan, Neurotech USA Inc. Code F (Financial Support), Allergan Code F (Financial Support), Nightstar Therapeutics Code F (Financial Support), Second Sight Medical Products Code F (Financial Support), AGTC, Inc. Code C (Consultant), Editas Medicine, Inc. Code C (Consultant), ImagineEyes Code C (Consultant), ProQR Therapeutics Code C (Consultant), Spark Therapeutics Code C (Consultant), Sparing Vision Code C (Consultant), ProQR Therapeutics Code R (Recipient), Sparing Vision Code R (Recipient), Second Sight Medical Products Code R (Recipient), Ian M. MacDonald, None; Mark E. Pennesi, IONIS Pharmaceuticals Code C (Consultant), Editas Code C (Consultant), ProQR Pharmaceuticals Code C (Consultant), Biogen Code C (Consultant), Nacuity Pharmaceuticals Code C (Consultant), RegenexBio Code C (Consultant), Astellas Pharmaceuticals Code C (Consultant), Genesight Code C (Consultant), Horama Code C (Consultant), Opthotech Code C (Consultant), Spark Therapeutics Code C (Consultant), Eyevensys Code C (Consultant), AGTC Code C (Consultant), AGTC Code S (Non-remunerative), Sanofi Code C (Consultant), Sanofi Code S (Non-remunerative), Foundation Fighting Blindness Code S (Non-remunerative), Nightstar Therapeutics Code C (Consultant), Nightstar Therapeutics Code S (Non-remunerative), Stephen H. Tsang, None;

SIG Description (Limit 1200 Characters)
There are continuing advances in treatment approaches for inherited retinal degenerations (IRDs) with multiple clinical trials for a variety of treatment approachs including gene therapies, cell therapies, drug, neuroprotective and prosthetic devices. We are now seeing combinations of therapies being used in rare and common disease with gene correction combined with cell therapy being one example. In the field of IRD therapy development there are opportunities for gene mutation specific therapies as well as non gene specific approaches that could benefit multiple IRD genotypes. This Special Interest Group Session will bring together a panel and audience of experts in the field to identify and debate the various avenues for treatment of IRDs and the potential for those to be used in combination therapies to arrest and possibly reverse vision loss. Inherited retinal degenerative disease genotypes and phenotypes are highly heterogeneous, however commonalities exist between some of these unique diseases that could benefit from similar treatment approaches. In other rare, and non-rare diseases combination therapies are becoming standards of care.