PATIENTS AND FAMILIES Patient Early Access Policy

Patient Early Access Policy

AVROBIO is committed to developing gene therapies that have the potential to transform the treatment of rare diseases. To accomplish this, we collaborate with many researchers, individuals living with genetic disorders, patient groups and physicians and we work closely with regulatory agencies.

Clinical trials serve a vital role in development of new therapeutic options. 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 fastest way towards making AVROBIO’s gene therapies potentially available to the greatest number of individuals in the future.

AVROBIO bases its position on Patient Early Access on these key considerations:
  • Whether based on available safety and efficacy data, sufficient evidence is established that the potential benefits to the patient(s) outweighs the potential risks.
  • Whether there is adequate supply of the investigational gene therapy to complete the ongoing and planned clinical trials.
  • Whether it will compromise or delay ongoing clinical trials or future access for the broader community.

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


Explore more

Illustration person

Patient advocacy

Connect with our patient advocacy and engagement team.

about us illustration

About us

Read about our mission and vision.

illustration graph

Our pipeline

Learn more about our pipeline of investigational gene therapies.