Phase 1 trial to open into SC291, CAR T-cell therapy for AAV

Treatment aims to deplete B-cells that give rise to self-reactive antibodies

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration has approved Sana Biotechnology’s investigational new drug (IND) application requesting a Phase 1 clinical trial of its investigational cell therapy SC291 in people with ANCA-associated vasculitis (AAV) and other autoimmune diseases.

“Our goal is to develop SC291 for patients with multiple B-cell mediated autoimmune diseases, and the clearance of this IND is an important milestone,” Doug Williams, PhD, president of research and development at Sana, said in a company press release.

While further details of the planned trial were not announced, Williams added: “We intend to begin treating patients in the near-term and expect to disclose initial safety and efficacy data across multiple diseases in 2024.”

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AAV cell therapy derived from T-cells of healthy donors

AAV is caused by self-reactive antibodies that stick to blood cells, triggering inflammation in small blood vessels. Antibodies, proteins made by a type of immune cell called B-cells, normally are important for fighting off infections. But in autoimmune diseases like AAV, they attack the body’s own cells to cause a variety of disease symptoms.

SC291 is a CAR T-cell therapy that’s designed to deplete B-cells, which is expected to ease the severity of AAV and other autoimmune diseases driven by B-cells and self-reactive antibodies.

CAR T-cell therapies involve collecting immune T-cells and modifying them in the lab to have a protein receptor, called a CAR (chimeric antigen receptor), that directs the cells to attack a specific target. The modified cells are then infused into patients.

In the case of SC291, the T-cells are equipped with a CAR targeting CD19, a protein that’s found on the surface of B-cells. The cells used also are allogenic, meaning they are collected from healthy donors.

Ordinarily, when cells from another person are put into the body, they are recognized as foreign and destroyed by the immune system. To avoid this, T-cells in SC291 are engineered with Sana’s proprietary hypoimmune platform, reported to “hide” the allogenic CAR T-cells from the immune system and allow for a transplant without immunosuppression.

“SC291 is an allogeneic CAR T cell therapy with a scaled manufacturing process that produces hundreds of patient doses per manufacturing run,” Williams said, adding that using allogenic cells “simplifies the treatment paradigm for doctors and patients alike by eliminating apheresis and individualized patient-by-patient manufacturing of the drug product.”

Apheresis is a blood filtering process used to collect T-cells from donors for CAR T-cell therapies. In the case of allogeneic therapies, the cells are collected from healthy donors and expanded in the lab, while those using T-cells collected from patients require apheresis and manufacturing for each patient.

Notably, SC291 is the first healthy donor-derived allogeneic CAR T-cell therapy to move into clinical trials, the company noted in the release.

The upcoming Phase 1 trial will test SC291 in people with AAV and those with certain forms of lupus, another autoimmune disease driven by B-cells.