Expanded Access Policy

About Expanded Access to Therapies in Development

Sangamo is committed to translating groundbreaking science into tomorrow’s genomic cures.

Developing these innovative therapies – from the laboratory to the marketplace – is a thorough process with multiple steps designed to ensure the therapies are safe and effective. It can take years to complete.

We feel an urgent responsibility to advance new therapies as rapidly and efficiently as possible because we know there are patients who need them now – patients who otherwise lack suitable therapies to treat their condition.

Some patients with serious or immediately life-threatening diseases who could potentially be aided by our investigational therapies may not be eligible for or able to participate in our clinical studies. For them, Sangamo is working expeditiously to conduct necessary clinical studies to generate sufficient safety and efficacy data to determine if an early/expanded access program is safe and appropriate. We are committed to having our work make its earliest possible contribution to patient health and well-being.

Typically, investigational therapies need to reach Phase 3 clinical trials before there is sufficient safety and efficacy data to determine if an expanded access program is appropriate. None of the programs for which Sangamo controls the clinical trials has reached Phase 3 yet. When that time comes, we will announce any early/expanded access program involving our therapies well in advance of launch.

Any future expanded access program Sangamo establishes will be consistent with our commitment to patient safety and the safeguards that are in place to protect public health. In addition, these important principles will apply to any Sangamo program:

• Our program will be consistent with Regulatory Agency guidelines and approval processes;
• Each access request must come from a qualified, treating physician and will be considered on a case-by-case basis using a fair and equitable process;
• The disease condition of the patient involved must be serious or life-threatening, with no other satisfactory treatment options available (including approved products) or open/pending clinical trials available;
• The risk-benefit of receiving the therapy must be assessed and confirmed by the requesting physician and will be further assessed by Sangamo;
• Competent healthcare facilities must be available that are appropriate to administer therapy, manage potential side effects, and monitor the patient with appropriate follow-up;
• Informed consent, regulatory and institutional approvals must be secured in order to act on any request lawfully and responsibly;
• The early access program will not impede the initiation, ongoing progress, or completion of clinical trials or regulatory filings associated with the therapy;
• Adequate supply of the product/therapy exists, manufacturing capability is maintained for ongoing programs, and it is logistically feasible to make the therapy available outside of a clinical trial setting, and
• Our programs will support use of Sangamo therapies for the anticipated disease condition[s] and will not be available to treat other diseases.

For questions, please contact patientadvocacy@sangamo.com.