Cellectis S.A.

At Cellectis S.A. (“Cellectis”), we are committed to providing novel treatment options to patients living with serious diseases. To do this, Cellectis conducts clinical trials to assess the safety and efficacy of investigational medicines, which may allow us to obtain the necessary regulatory approvals and provide patients with broader access to these medicines. 

Cellectis maintains that participating in clinical trials is the best way for patients to access medicines prior to approval. In some circumstances when a clinical trial is not possible, patients with serious diseases or conditions may seek special access to investigational medicines. This access to investigational medicines is often referred to as expanded access or compassionate use. In the United States, Cellectis is engaging in development of therapies for hematological malignancies. Because of the need to focus development efforts on clinical trials that could support regulatory approval, Cellectis generally will not provide access to its investigational medicines via expanded access or compassionate use. However, Cellectis is willing to evaluate requests for such access from qualified physicians, including consideration of the following: 

- Whether the disease or condition is serious or life-threatening; 

- The requesting physician has the appropriate experience of the administration of cell and gene therapy product and management of patients and the potential associated side effects associated with such products.; 

- The location/site where the treatment would be administered has the necessary equipment and systems available to manage and control the shipment, receipt, storage and administration of a cell-based product; 

- The efficacy and safety data available at the time are sufficient to determine whether the potential clinical benefit to patient would likely outweigh the possible risks, including the outcome of the disease; 

- The patient's physician has determined that treating the patient with the investigational product is in the patient's best interests; 

- The investigational medicinal product will be administered in accordance with applicable laws and regulatory requirements of the country where the patient is treated, including importation requirements; 

- The patient is not eligible or able to participate in a clinical trial or similar sponsored access program; 

- No appropriate therapeutic alternative is available; 

- Providing access to the Cellectis investigational therapy must not interfere with the completion of a clinical trial supportive of a Health Authority approval or compromise the potential development of the therapy; 

- An appropriate dose and/or treatment regimen for the investigational therapy has been determined for the disease for which treatment is sought; 

- There is manufacturing capacity and Cellectis has adequate supply of the investigational therapy; 

- Additional criteria regarding the therapy, its development program, the patient, or other circumstances may impact eligibility, and; 

- It is understood that pre-approval access will be submitted to health authorities for authorization and that any associated data collection will be compliant with regulations and data privacy requirements. 

All questions and requests regarding Cellectis’s expanded access or compassionate use programs should be submitted to Cellectis by email. In the case of requests by treating physicians, the email should contain information sufficient for Cellectis to evaluate the request consistent with this policy, including the following: 

- Date of request; 

- Requesting physician's name, contact information, address (including country), and professional designation (i.e., MD) or qualifications; 

- Name of the requested investigational medicinal product along with physician's intended treatment plan, including therapeutic indication and expected duration of treatment; and 

- Medical rationale for request, including an explanation for why alternative therapy cannot be used, why the patient does not qualify for a clinical trial, and why use of the investigational drug is in the patient's best interests. 

The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals, if applicable, and to comply with all other safety, monitoring, and patient consent requirements defined by Cellectis. Requests for expanded access or compassionate use may only be made by licensed physicians. Cellectis cannot guarantee that the investigational medicine will be available for a particular patient under an expanded access or compassionate use program. If granted, access will be for a designated period of time and then a request must be submitted and reviewed to continue the program, as relevant.