American Gene Technologies
U.S. Expanded Access Policy
Overview
American Gene Technologies (AGT) is developing AGT103‑T, a first‑in‑class autologous lentiviral vector‑modified CD4 T‑cell therapy designed to confer resistance to HIV infection and restore HIV‑specific immune function. AGT103‑T is an investigational product; it has not been approved by the U.S. Food and Drug Administration (FDA), and its safety and efficacy have not yet been established. Under the 21st Century Cures Act, companies developing investigational drugs must make public their policy for evaluating and responding to requests for expanded access (also called compassionate use). Expanded access is a potential pathway that enables patients with a serious or life-threatening disease or condition to gain access to an investigational product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available (1).
AGT strongly believes that participation in our clinical trials is the most effective and appropriate way for patients to access AGT103-T. Controlled clinical trials allow us to collect the data necessary to determine whether the therapy is safe and effective, and are designed, conducted, and monitored to ensure that potential therapies are properly evaluated before they are made generally available.
Currently, AGT does not offer AGT103-T outside of clinical trials. However, we have developed the following policy to describe how we will evaluate any future requests for expanded access and to comply with the transparency requirements of the 21st Century Cures Act.
Criteria for Consideration
Requests for expanded access to AGT103‑T will be considered only when all of the following conditions are met. These criteria are based on FDA guidance (1).
Serious or life‑threatening condition. The patient must have a serious or immediately life‑threatening disease (e.g., HIV‑AIDS) for which AGT103‑T may provide benefit.
No satisfactory alternative therapies. There must be no comparable or satisfactory alternative therapy available to diagnose, monitor or treat the disease.
Ineligibility for clinical trials. The patient is unable to participate in an AGT103-T clinical trial, for example due to exclusion criteria or because they do not live within 30 miles of an active clinical site.
Favorable risk‑benefit assessment. Sufficient clinical and non‑clinical data must exist to suggest that the potential benefit to the patient justifies the potential risks. This includes establishing a dosing regimen and preliminary evidence of effectiveness.
No interference with clinical development. Providing AGT103‑T on an expanded‑access basis must not compromise ongoing or planned clinical trials or the overall development program.
Adequate manufacturing capacity and supply. AGT must have sufficient manufacturing capacity and product supply to provide the investigational therapy without affecting clinical trials or other commitments.
Appropriate regulatory and institutional oversight. The treating physician must obtain approval from the relevant Institutional Review Board (IRB), and a regulatory mechanism (e.g., an FDA-approved protocol) must be in place to allow for expanded access.
Submitting a Request
At this time, AGT does not have an expanded access program and does not provide AGT103‑T outside of clinical trials. If, in the future, AGT determines that it can responsibly provide access to AGT103-T through an expanded access program, requests must come only from a licensed physician responsible for treating the patient. The physician should submit the following information to the contact listed below:
- Patient diagnosis and relevant medical history.
- Explanation of why the patient is not eligible for an ongoing AGT103‑T clinical trial.
- Description of prior therapies tried and current treatment options.
- Proposed dosing regimen and monitoring plan.
- Evidence of IRB approval (if available).
AGT will acknowledge receipt of any future expanded‑access request within five business days, consistent with industry practice. Each request will be reviewed by AGT’s medical, clinical, regulatory and senior leadership teams on a case‑by‑case basis, taking into account the criteria listed above.
Contact for Expanded Access Inquiries (physician use only):
Email: wdpalin@americangene.com
Phone: 414 688 6858
Decision and Oversight Drew Palin MD, President AGT
AGT will evaluate each request individually and will make decisions based on scientific evidence, patient safety, product availability and overall program integrity. Approval is not guaranteed; a denial may reflect a need to ensure patient safety or preserve the integrity of ongoing trials, rather than the importance of the request. AGT may consult external experts, patient advocacy groups, and bioethicists to inform its decisions, and it reserves the right to revise this policy at any time.
Investigational status: AGT103‑T is an investigational therapy and has not been proven safe or effective by the FDA. The use of investigational products may or may not provide benefit and can result in unexpected, serious side effects.
Clinical trial participation is the preferred and primary way to access AGT103-T. Such trials help determine whether the therapy works and gather necessary safety data.
Regulatory requirements: Expanded‑access use of AGT103‑T must comply with FDA regulations, including submission of an Investigational New Drug (IND) application supplement, IRB oversight, and reporting requirements. Physicians and patients should consult the FDA guidance on expanded access for more.
Policy Updates
AGT will review this expanded access policy regularly and will update it as new data become available or as regulatory guidance evolves. The current version of this policy will be posted on the AGT website and on the Reagan‑Udall Foundation’s Expanded Access Navigator. Last Updated: October 30, 2025
(1): https://www.fda.gov/news-events/public-health-focus/expanded-access
