Inducing competition for MSCs
Cynata preps to take first iPS cell-derived MSCs into the clinic.
The cells, already in Phase I testing in the U.K. and Australia, would become the first iPS cell-derived therapeutic in the clinic stateside, and offer an alternative to donor-dependent MSCs produced by conventional methods.
On July 5, Cynata announced it received confirmation from FDA that its Cymerus technology for creating MSCs from iPS cells met the necessary CMC expectations, indicating its lead CYP-001 product would likely meet standards for human testing in the U.S. According to CEO Ross MacDonald, the company plans to submit an IND on CYP-001 for graft-versus-host disease (GvHD) in 2018 pending discussions with its partner Fujifilm Holdings Corp.
Cynata said it also received the go-ahead from FDA to apply for a regenerative medicine advanced therapy (RMAT) designation for the product after results from the overseas trial are reported. The RMAT designation, defined by the 21st Century Cures Act, is similar to breakthrough designation for drugs.
MSCs don’t express HLA proteins, which means a single donor can provide cells to many patients, and makes MSC therapy a viable strategy to treat large disease indications.
While conventional methods expand MSCs isolated from healthy donors, Cynata’s platform promises a steady supply of cells from a single donor