Patients and Caregivers

Looking for information on our trials?

Research and Clinical Trials

AGTC is currently conducting clinical studies for the treatment of several genetic blinding conditions. This page will be updated as new clinical studies begin.

View our resource on Gene Therapy Clinical Studies for Achromatopsia (ACHM) and X-Linked Retinoschisis (XLRS) for more information about:

         Gene Therapy
         Inherited Retinal Disorders
         AGTC’s XLRS Clinical Trial
         AGTC’s ACHM (CNGB3) Clinical Trial

If you have additional questions about any of our studies, please contact us at advocacy@agtc.com.

X-linked Retinoschisis (XLRS)

AGTC is currently conducting a clinical study to evaluate the safety and efficacy of an investigational gene therapy in patients with XLRS.

This study is currently recruiting.

For more information, and to see whether you or someone you know qualifies to join the study, please click here to view the study page on ClinicalTrials.gov.  

Achromatopsia (ACHM B3)

AGTC is currently conducting a clinical study to evaluate the safety and efficacy of an investigational gene therapy in patients with ACHM caused by mutations in the CNGB3 gene (also known as ACHM B3).

This study is currently recruiting.

For more information, and to see whether you or someone you know qualifies to join the study, please click here to view the study page on ClinicalTrials.gov.

Achromatopsia (ACHM A3)

AGTC is currently conducting a clinical study to evaluate the safety and efficacy of an investigational gene therapy in patients with ACHM caused by mutations in the CNGA3 gene (also known as ACHM A3).

This study is currently recruiting.

For more information, and to see whether you or someone you know qualifies to join the study,  please click here to view the study page on ClinicalTrials.gov.

X-linked Retinitis Pigmentosa (XLRP)

In August 2017, AGTC announced the filing of an Investigational New Drug application with the U.S. Food and Drug Administration to conduct a Phase 1/2 clinical trial of the company's gene therapy product candidate for the treatment of XLRP caused by mutations in the RPGR gene.

AGTC plans to initiate a clinical study evaluating the safety and efficacy of its proprietary XLRP gene therapy candidate in the United States in the coming months.

For information about AGTC’s XLRP program and/or the upcoming clinical trial, please contact us at advocacy@agtc.com.

Approximately 50 percent of RP cases are isolated and have no previous family history; the cause of these cases cannot be explained. Other cases of RP, where family history has been determined, fall into three main categories: autosomal recessive, autosomal dominant, and X-linked. 

Advocacy & Education

Patients and caregivers interested in learning more about rare and blinding diseases may find valuable information and resources on the following websites:

Patients and Caregivers

Contact Us

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Cambridge, MA 02139
617.843.5728
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