Vyriad opens a Phase 1 trial of an in vivo CAR-T at Mayo Clinic

Vyriad, of Rochester, Minnesota, said on 14 September 2026 that the FDA had accepted its investigational new drug application for VV169, and that a Phase 1 trial is open for enrollment at Mayo Clinic in Rochester.

VV169 is an in vivo CAR-T therapy targeting BCMA. Conventional CAR-T requires collecting a patient’s T cells, engineering them outside the body and growing them for weeks before infusion. VV169 is given intravenously and uses a lentiviral vector with a blinded and retargeted VSV-G pseudotype to deliver the anti-BCMA payload to T cells inside the body, which then express the receptor themselves.

The dose-escalation study plans to enroll up to 40 patients with relapsed or refractory multiple myeloma, with Yi Lin as principal investigator. Primary objectives are overall safety, dose-limiting toxicities and a recommended Phase 2 dose; secondary objectives include preliminary efficacy and how far the CAR T cells expand and persist. The company gives the registration as NCT07802717.

Manufacturing time and cost are the constraints that in vivo approaches are meant to remove: a therapy assembled inside the patient needs no cell collection, no production slot and no wait.