Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Orca-Q, Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy for the treatment of high-risk hematologic malignancies.

"The FDA’s RMAT designation for Orca-Q recognises the significant unmet need for patients with serious hematologic malignancies and validates the promising clinical findings from our ongoing phase 1 study,” said Nate Fernhoff, Ph.D., Co-Founder and Chief Executive Officer, Orca Bio. “As this is our second cell therapy candidate to receive RMAT status, we have experienced firsthand how this program can support accelerated development and enhanced communication with the FDA. With our newly expanded phase 1 study continuing to enroll patients and additional data expected later this year, we remain focused on advancing our high-precision approach to deliver an important new treatment to patients with blood cancer.”

The RMAT application included data from the ongoing phase 1 clinical trial (NCT03802695) evaluating Orca-Q for the treatment of hematologic malignancies across six treatment cohorts, including in patients with haploidentical donors, and in patients receiving non-myeloablative or reduced intensity conditioning. Clinical findings demonstrated durability and encouraging outcomes across key metrics, including Overall Survival, acute and chronic Graft Versus Host Disease (GVHD) and non-relapse mortality. Enrollment is ongoing, with new data expected to be presented at upcoming scientific congresses.

RMAT designation is a specialised FDA program created to accelerate the development and review of promising new therapies, including cell therapies, intended to treat serious or life-threatening conditions. To qualify, a therapy must demonstrate preliminary clinical evidence suggesting the potential to address unmet medical needs. This designation provides important opportunities during the drug development process, including increased FDA guidance and eligibility for priority and rolling reviews, as well as accelerated approval pathways. By streamlining these regulatory milestones, the program aims to bring transformative innovations to patients more quickly.