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 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 AML Hub is an independent medical education platform, sponsored by Daiichi Sankyo, Johnson & Johnson, and Syndax, and has been supported through an educational grant from the Hippocrate Conference Institute, an association of the Servier Group. 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 moreCreate an account and access these new features:
Bookmark content to read later
Select your specific areas of interest
View aml content recommended for you
The U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to PRGN-3006 for the treatment of patients with relapsed or refractory (RR) acute myeloid leukemia (AML).1,2 PRGN-3006 is an investigational therapy which uses a proprietary UltraCAR-T™ therapeutic platform to reduce the time to manufacture chimeric antigen receptor (CAR) T cells, enabling the administration of CAR T cells one day after non-viral gene transfer. PRGN-3006 UltraCAR-T is a multigenic CAR T-cell product, co-expressing a CD33 CAR and membrane-bound interleukin-15 (mbIL15), together with a ‘kill switch’. CD33 CAR precisely targets malignant cells expressing CD33, while expression of mbIL15 allows the elimination of ex vivo expansion of T cells prior to cell administration. The kill switch provides a mechanism for selective depletion of PRGN-3006 post-administration in order to improve therapeutic control.
There is an ongoing trial of PRGN-3006 (NCT03927261), in patients with RR AML and high-risk myelodysplastic syndromes, which has completed enrolment of the first cohort and initial data is expected later this year. Preclinical data on PRGN-3006 were presented at the 61st American Society for Hematology Annual Meeting & Exposition, in Orlando, US.3
Orphan drug status is granted to medicines aimed at treating, detecting or preventing rare diseases or disorders affecting < 200,000 people in the US. This status grants certain incentives such as a seven-year exclusivity marketing period, applications for funding for phase I and II clinical trials, reduced market application filing fees and rapid regulatory processing later on.
References
Please indicate your level of agreement with the following statements:
The content was clear and easy to understand
The content addressed the learning objectives
The content was relevant to my practice
I will change my clinical practice as a result of this content