Expanded Access Policy
At LEXEO Therapeutics, we are dedicated to developing gene therapies that will transform the lives of patients with serious debilitating and/or life-threatening diseases. Our goal is to provide these treatments as quickly as possible to patients, once rigorous testing demonstrates our gene therapies are safe and effective for patients, and regulatory authorities, like the US Food and Drug Administration (FDA), have approved these therapies.
Evaluation of investigational treatments in clinical trials plays a major part in achieving this important goal, and we apply the strongest scientific, legal, and ethical principles to the evaluation of our investigational gene therapies. Patients, families, caregivers, healthcare professionals, and patient advocacy organizations may find information about our clinical studies on www.clinicaltrials.gov, consistent with FDA regulations, and ex-US clinical trial registries for our global trials which include clinical sites outside the US.
Under certain conditions, the FDA may allow possible access to investigational treatments by patients and their healthcare provider and outside of clinical trials through an Expanded Access Program (also referred to as “compassionate use” or “early access”) while the investigational product is still under development.
It is important to understand an Expanded Access Program involves an investigational treatment whose safety and effectiveness has not been established for the patient’s specific condition. As such, these investigational treatments have not been approved as safe and effective by the FDA. Expanded Access Programs are regulated by the US FDA under Title 21 CFR Part 312 Subpart I, but do not take the place of a rigorously conducted clinical trial for an investigational product, as described above.
At this time LEXEO Therapeutics does not have an Expanded Access program for our programs under development. LEXEO believes that the most appropriate way to access our investigational therapies is through participation in one of our clinical trials. We encourage patients and their healthcare providers to discuss if participation in one of our planned or ongoing clinical trials is appropriate. Please contact us if you have any questions about our clinical trial programs or individual clinical trials. We do not anticipate providing Expanded Access to investigational therapies until we have results from clinical trials that indicate a therapy may provide benefit for patients with acceptable safety.
As a part of our strong commitment to patients and our focus on developing transformative gene therapies, LEXEO Therapeutics will regularly review this Expanded Access Policy and make revisions as appropriate, in accordance with the 21st Century Cures Act of 2016.