Cell therapy SC291 gets FDA fast track designation for SLE
New status covers treatment's use for relapsed or refractory disease
The U.S. Food and Drug Administration (FDA) has granted fast track designation to experimental cell therapy SC291 as a potential treatment for hard-to-manage systemic lupus erythematosus (SLE).
Fast track status is given to therapies that have the potential to improve care for serious diseases, with the goal of speeding the review and development of possibly important new treatments. The FDA’s designation specifically covers SC291’s use in SLE that is relapsed or refractory, meaning the disease has either worsened after initially responding to treatment, or has not responded to available treatments.
The designation “highlights the need for new treatment options for patients with relapsed/refractory SLE,” Dhaval Patel, MD, PhD, chief scientific officer of developer Sana Biotechnology, said in a company press release.
SLE, the most common and serious form of lupus, is an autoimmune disease driven by antibodies that erroneously target the body’s own healthy tissues, triggering an inflammatory attack. Antibodies are immune proteins made by B-cells, a specialized type of immune cell.
SC291 is a CAR T-cell therapy that’s designed to deplete B-cells. T-cells are a different type of immune cell able to eliminate other cells that have become infected with a virus or turned cancerous. In CAR T-cell therapies, T-cells are equipped with a lab-made protein called chimeric antigen receptor, or CAR, that directs them to go after a specific molecular target. In SC291, T-cells are equipped with a CAR that targets CD19, a protein that’s found on the surface of B-cells.
Potential ‘universal’ therapy
Most CAR T-cell therapies are autologous, meaning that a patient’s own T-cells are collected, taken to a lab to be equipped with the CAR, and then infused back into the patient’s body. SC291 is allogeneic, meaning it uses T-cells collected from healthy donors.
One of the risks with this strategy is that the immune cells harvested from donors may be seen as a potential threat by the patient’s immune system. If that’s the case, the donor cells may be attacked and destroyed, which in turn might compromise treatment efficacy. To overcome this limitation, cells in SC291 are specifically designed with a hypoimmune platform that enables them to evade the patient’s immune system.
“As a [hypoimmune]-modified allogeneic CAR T therapy with a scaled manufacturing process that produces hundreds of patient doses per manufacturing run, SC291 has the potential to serve as a universal off-the-shelf therapy that can address this large unmet need,” Patel said.
Sana is sponsoring a Phase 1 clinical trial called GLEAM (NCT06294236) that’s testing SC291 in adults with severe SLE, including lupus nephritis — a complication of lupus marked by kidney damage and inflammation — and extrarenal SLE, or SLE affecting parts of the body besides the kidneys. GLEAM is enrolling patients with SLE and other severe autoimmune disorders, including antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis.
The study’s main goal is to evaluate treatment’s safety. Recruitment is ongoing at five sites in the U.S. Initial data from the trial are expected in 2025, according to Sana. “We look forward to sharing initial data from the ongoing GLEAM trial,” Patel said.
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