Compassionate Use Policy

Lysogene is committed to developing and commercializing safe and effective gene therapy products for patients living with serious, life-threatening, rare diseases. We appreciate the responsibility of bringing these innovative products to patients, and this commitment drives our work each day. We believe in the importance of collaborating closely with patient, medical and regulatory communities to help achieve this goal.

The primary preferred mechanism for access to investigational therapies is by participation in a clinical study. Clinical studies collect the information necessary to understand the safety and efficacy of the potential therapy allowing for regulatory authority review and potential approval.  Ultimately, we believe that approval by regulatory authorities gives the opportunity for the broadest access by allowing physicians to prescribe the therapy to any patient who might benefit.

We also understand that patients and families are interested in accessing our investigational therapies prior to regulatory approval and outside of the clinical trial setting, through compassionate use* programs, sometimes called “early access” or in the United States, Expanded Access. Compassionate use must balance the urgency of receiving investigational treatment with many other factors.

After much consideration, we have determined that, at this point in time, we are unable to offer a compassionate use program that would be in the best interest of the patient community. We will continue to evaluate the possibility of compassionate use and we will update the community if any such programs can be conducted in a fair and sustainable way without compromising clinical development and potential regulatory approval.

For information on ongoing Lysogene clinical studies:

  • Visit and search registered clinical studies for the disease.
    • If you locate a clinical study with a participating site that you are interested in, you or your physician may contact that site directly for more information.
  • For additional information, contact the Lysogene Patient Access group.
  • Compassionate use programs include requests for access under section 561(b) of the Federal Food, Drug and Cosmetic Act, as well as ‘Right to Try’ legislation.


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