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Gene Therapy Trial for Dry AMD

Oct 16 2019

Posted In:

20/20 Blog

Palo Alto, CA — A gene therapy clinical trial for Dry Age Related Macular Degeneration (AMD) is beginning at the Byers Eye Institute under the direction of Vinit Mahajan M.D., Ph.D., associate professor and Vice Chair for Research in Ophthalmology. The trial is in partnership with Gyroscope, an ophthalmology company developing genetically-defined therapies to treat eye disease. 

AMD is the leading cause of blindness in people over fifty. Patients suffer from progressive, decreased central vision that affects reading, driving, and other daily activities. Current treatments are limited to vitamin supplements that aim to slow disease progression, since there is no cure at this time. However, gene therapies may revolutionize the treatment of genetic diseases, giving patients hope for a cure for the first time.

Mahajan said, “With a focus on precision health, researchers have made enormous strides in genome sequencing and analysis, gene delivery, and genome surgery as we narrow in on the molecular causes of disease. It is exciting to be working with Gyroscope to move the field forward and possibly bring life altering therapies to patients.”

The first step in the new trial is to identify patients with specific gene mutations that put them at risk for dry AMD. Genetic testing is performed with Dr. Mahajan at the Byers Eye Institute after a clinical exam and specialized retinal imaging confirms the diagnosis and disease stage. 

Dr. Mahajan has expertise in gene therapy and genetic diseases of the eye. He was a surgeon for the human Leber’s Congenital Amaurosis (LCA) gene therapy trial that improved functional vision in blind patients. This was the first gene therapy approved by the FDA.  In his laboratory, researchers develop and study gene therapy to refine, improve, and fix genes that cause blindness in patients.

To be considered for this study, patients can send an email to clinical research coordinator, Teja Chemudupati, at

Please visit the links below to learn more about the Mahajan Lab’s work with gene therapy:

Gene Therapy Lab

Key Proteins will Guide Personalized Health

Gene Therapy and Eye Disease

Patient Guide to Dry Macular Degeneration

Patient Guide to Genetic Testing

A Molecular Approach to Clinical Trials

Patients Needed for Clinical Trials and Research

Molecular Analysis Plays Important Role in Orphan Disease

Bay Area Genetic Retinal Disease Case Conference

CNN: Experimental gene therapy for blindness considered by FDA