About Expanded Access to Therapies in Development

At Sangamo, we are committed to developing innovative genomic therapies to transform the lives of patients with serious genetic diseases.

Developing these innovative therapies – from the laboratory to the marketplace – can take years to complete.  We understand that 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, or that they may otherwise lack suitable therapies to treat their condition. We feel an urgent responsibility to advance these new therapies as rapidly and efficiently as possible.

To determine if expanded access is appropriate, we will evaluate if there is sufficient scientific evidence of efficacy and safety for our programs. This usually happens in Phase 3 clinical trials. We have not yet reached this stage of development for any of our programs; accordingly, current access to our investigational therapies is available through participation in one of our clinical trials.

Sangamo is working expeditiously to conduct necessary clinical studies to generate sufficient safety and efficacy data which could justify an early/expanded access program. We are committed to having our work make its earliest possible contribution to patient health and well-being.

Recently, right-to-try laws were enacted to permit terminally ill patients to try experimental therapies that have completed Phase 1 testing but have not been approved by the FDA. At Sangamo, we are committed to the safety of patients and the safeguards that are in place to protect public health. In the future, as data is generated which may support requests for access to investigational medicines, these requests will be evaluated through our expanded access review process which includes independent review by the FDA to assure full consideration of safety data of which the FDA may be uniquely aware.

We will announce any early/expanded access program involving our therapies well in advance of launch. But we already recognize some importance principles – in addition to sufficient evidence of safety and efficacy – that 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 health care 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 indicated disease condition[s] and will not be available to treat other diseases.