All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional. If you are a patient or carer, please visit Know AML.
The AML Hub website uses a third-party service provided by Google that dynamically translates web content. Translations are machine generated, so may not be an exact or complete translation, and the AML Hub cannot guarantee the accuracy of translated content. The AML Hub and its employees will not be liable for any direct, indirect, or consequential damages (even if foreseeable) resulting from use of the Google Translate feature. For further support with Google Translate, visit Google Translate Help.
The U.S. Food and Drug Administration (FDA) has granted both orphan drug designation and regenerative medicine advanced therapy (RMAT) to ORCA-T, an investigational engineered donor graft therapy.1 The RMAT designation allows for expediated development of new therapies and, as a cellular therapy, ORCA-T will receive early guidance from the FDA regarding manufacturing and clinical development. Orphan drug designation is granted to therapies that treat diseases affecting fewer than 200,000 people in the U.S.
ORCA-T is a T-cell depleted graft that is then reinfused with both purified regulatory T cells and conventional T cells. ORCA-T aims to allow for reconstitution of the immune system without triggering graft-versus-host disease.
The safety and efficacy of ORCA-T are currently being evaluated in a non-randomized, single-arm phase Ib trial (NCT04013685) in patients receiving myeloablative allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia, acute lymphoid leukemia, mixed phenotype leukemia, or high-risk myelodysplastic syndromes.
More data on ORCA-T are expected to be announced at this year’s 62nd American Society of Hematology (ASH) annual meeting.
Outcomes of two or more HLA loci mismatched unrelated donor allo-HCT: An Acute Leukemia Working Party study
The optimal allogeneic hematopoietic cell transplantation (allo-HCT) results depend on the availability of a human leukocyte antigen...
The effect of CD34+ cell dose on responses to haploidentical allo-HSCT: Results from a retrospective analysis
The graft cell dose following allogenic hematopoietic stem cell transplantation (allo-HSCT) has been suggested to influence patient outcome....
Subscribe to get the best content related to AML delivered to your inbox