Patient Early Access Policy Information

AVROBIO is committed to developing gene therapies that have the potential to transform the treatment of rare diseases. To accomplish this, we depend collaborate with many researchers, patients, patient groups, physicians, and the guidance of regulatory agencies.

Clinical trials serve a vital role in treatment development. They are carefully regulated, monitored and conducted in specific populations to assess safety and efficacy.

Currently, access to AVROBIO investigational therapies is only possible through participation in clinical trials. Completing the necessary clinical trials and obtaining the data needed for review and approval by regulatory agencies is the best way to work towards making AVROBIO’s gene therapies available to the greatest number of patients in the future.

AVROBIO bases its position on Patient Early Access on these key considerations:

  • Whether sufficient evidence that the potential benefits to the patient(s) outweighs the potential risks, based on available safety and efficacy data.
  • Whether it will compromise or delay ongoing clinical trials or future access to the broader community.
  • Whether there is adequate supply of the investigational gene therapy to complete the ongoing and planned clinical trials.

If you have additional questions, please speak with your physician or contact We will acknowledge receipt and respond to your inquiry within 7 days.

As more information and clinical data become available, AVROBIO may revise this policy. If you would like to receive regular updates about AVROBIO please email