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.
The gvhd 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 gvhd Hub cannot guarantee the accuracy of translated content. The gvhd 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 GvHD Hub is an independent medical education platform, sponsored by Medac and supported through grants from Sanofi and Therakos. The funders are allowed no direct influence on our content. The levels of sponsorship listed are reflective of the amount of funding given. View funders.
Now you can support HCPs in making informed decisions for their patients
Your contribution helps us continuously deliver expertly curated content to HCPs worldwide. You will also have the opportunity to make a content suggestion for consideration and receive updates on the impact contributions are making to our content.
Find out more
Create an account and access these new features:
Bookmark content to read later
Select your specific areas of interest
View gvhd content recommended for you
FDA grants the Investigational New Drug designation for PBCAR20A - a CAR T-cell therapy
On 16 September 2019, the US Food and Drug Administration (FDA) accepted the Investigational New Drug (IND) application for the off-the-shelf chimeric antigen receptor (CAR) T-cell therapy, PBCAR20A.
PBCAR20A is a CD20-targeting genome-edited allogeneic CAR T-cell therapy candidate that can now be investigated for the treatment of non-Hodgkin lymphoma (NHL), chronic lymphocytic leukemia (CLL), and small lymphocytic lymphoma (SLL). The phase 1/2a clinical trial is planned to start by the end of the year and aims to recruit patients with CLL, SLL, and NHL, including mantle cell lymphoma (MCL), for which the company has received Orphan Drug Designation. The initial report of this nonrandomized, open-label study is expected in 2020.
The approval is based on the preclinical data showing PBCAR20A efficacy in vivo against CD20+ tumor cells. The allogeneic CAR T-cells are created by inserting the CAR gene at the T-cell receptor (TCR) locus of T cells derived from healthy donors. The proprietary manufacturing platform allows fast manufacture of a consistent product, maintaining a high proportion of naïve and central memory CAR T-cells. The technology aims to prevent graft-versus-host disease.
This is the second off-the-shelf CAR T-cell IND product; PBCAR0191, an anti-CD19 CAR T-cell therapy, is currently being investigated in patients with relapsed/refractory (R/R) NHL and R/R B-cell precursor acute lymphoblastic leukemia (B-ALL).
References