For Patients
Compassionate Use and Expanded Access Policy
Compassionate Use and Expanded Access Policy

Bringing patients into focus
with ocular gene therapy

Atsena Therapeutics is evaluating investigational gene therapies in clinical trials for patients with inherited retinal diseases who have limited or no treatment options.

We are committed to developing safe and effective therapies and to upholding the highest standards of ethics, transparency, and patient safety. While we fully understand the urgency and desire for access to our promising therapies and recognize the significance of compassionate use or expanded access programs, our current priority is to advance our clinical trials as efficiently as possible to ultimately benefit the greatest number of patients.

Therefore, we wish to emphasize that participation in Atsena Therapeutics’ clinical trials is currently the only pathway for patients to gain access to our investigational therapies, and we do not offer compassionate use nor expanded access programs at this time. The safety and well-being of patients are of paramount importance to us. The investigational nature of our treatments requires stringent adherence to clinical trial protocols, which have been designed to carefully assess safety and efficacy. We believe this approach will lead to broader patient access in the future.
It is important to note that our investigational therapies have not yet received approval from the U.S. Food and Drug Administration (FDA) or other regulatory agencies. As with any investigational treatment, there are inherent uncertainties, and the therapy’s effectiveness in treating a specific condition cannot be guaranteed. Additionally, patients must be aware that using investigational therapies may carry unexpected serious side effects. As we continue to progress, we will assess the feasibility of expanded access programs and other avenues to broaden patient access to our therapies.

For more information from the FDA about expanded access in the United States, visit:

For further inquiries or additional information about this policy, we encourage you to reach out to us at [email protected]. We anticipate acknowledging receipt of requests sent to this email within five business days.

For more information on Atsena’s clinical trials, please visit:

Leber Congenital Amaurosis Type 1 (LCA1):
X-Linked Retinoschisis (XLRS):

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