Please join Dr. Kumar-Singh, Tufts University, as he presents, “Anti-Complement Gene Therapy for Dry and Wet Age-Related Macular Degeneration- from conception to clinical trials”” The lecture will take place on February 19, 2019 as part of the Research Lecture Series in Lecture Hall 2. All are welcome to attend. One hour of Mass CE will be awarded. You are invited to join us for a light reception in Conference Room 1 following the lecture.

ABSTRACT: Age-related macular degeneration (AMD) is the most common cause of blindness among the elderly.  There is currently no FDA-approved treatment for ~90% of AMD patients that suffer from the ‘dry’ form of this disease.  Genetic and immunohistochemical evidence suggests that an arm of the immune system known as complement plays a significant role in the pathophysiology associated with AMD.  The terminal step in activation of complement results in the formation of a pore on the surface of cells known as the membrane attack complex (MAC).  At high levels, MAC kills cells.  MAC has been found to be elevated in AMD patients.  The inability to efficiently form MAC through a naturally occurring mutation reduces the risks of developing AMD.  We have developed an inhibitor of MAC known as sCD59 that can be delivered to the retina using a recombinant adeno-associated virus (AAV).  In cell culture and in animals, sCD59 expressing AAV can inhibit the formation of MAC.  In humans, AAV expressing sCD59 was found to be safe in a Phase I clinical trial for ‘dry’ AMD.  A phase II clinical trial is planned in 2019.

Approximately 10% of AMD patients suffer from the ‘wet’ form of the disease. These patients are currently treated with frequent intraocular injections of anti-VEGF therapies.  Although highly effective, the burden associated with these injections leads to reduced patient compliance, ultimately resulting in up to 50% of ‘wet’ AMD patients continuing to lose vision in the long term.  Approximately 30% of ‘wet’ AMD patients do not respond to anti-VEGF therapy.  We have initiated a Phase I gene therapy clinical trial for ‘wet’ AMD that addresses both of these clinical unmet needs of ‘wet’ AMD patients.


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