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AGTC is on the front lines of researching the exciting potential of gene therapy to treat a range of rare inherited conditions.  As a leader in gene therapy, we are focused on leveraging our best-in-class technology platform to discover treatments that are life-changing for patients and families.  Learn more about the promise of gene therapy and how it works

We are currently focused in three key areas: ocular disease, neurodegenerative diseases and otology.


  • X-linked Retinitis Pigmentosa
  • Achromatopsia
  • Optogenetics (BionicSight partnership)
  • Age-related Macular Degeneration (Dry AMD)


  • Frontotemporal Dementia (FTD)
  • Amyotrophic Lateral Sclerosis (ALS)


  • Non-syndromic hearing loss

Learn more about our pipeline.

We are now conducting clinical trials to help answer the questions about the safety and effectiveness of potential treatments for X-linked Retinitis Pigmentosa and Achromatopsia.  In addition, AGTC is partnering with BionicSight in the development of an optogenetic therapy.


Why enroll in a clinical trial?


Clinical trials are crucial to the research and development of new therapies. However, many of these potential new therapies are slow to reach patients, often due to challenges in getting patients to enroll in clinical trials. The problem is worse in rare conditions, due to the low number of people with the condition, lack of awareness about active studies, and other barriers such as out of pocket costs and travel.

Choosing to enroll in a clinical trial is an important decision with many potential benefits. By participating in a clinical trial for affected individuals, patients are provided access to the latest promising therapies, while contributing to overall research and understanding of the condition.


What to expect if I am considering a clinical trial


Each person’s experience in a clinical trial, including those evaluating the potential benefit of a new therapy for a rare condition, is different.


What should I think about before joining a clinical trial?


Before joining a clinical trial, it is important to learn as much as possible. Discuss your questions and concerns with members of your health care team to determine if the trial is a good option based on your current condition and options available. Before making a decision to enter a trial, it’s important to understand:

  • What happens during the trial
  • The type of health care you will receive
  • Any related costs once you are enrolled in the trial
  • The benefits and risks associated with participating

To learn more about clinical trials, please visit the FDA website


Visionary science for life changing cures.

AGTC is now pre-screening for a phase 2/3 clinical trial of an investigational gene therapy in people with X-Linked Retinitis Pigmentosa.

Learn more about the clinical trial or how to qualify for pre-screening: