Clinical-Grade Therapeutic Progenitors Generated from Universal Donor Tankyrase/PARP-Inhibited Pluripotent Stem Cell Banks

Case ID:
C16548

Unmet Need / Invention Novelty: Regenerative cell therapies with conventional, lineage-primed human induced pluripotent stem cells (hiPSC) are limited by variable differentiation efficiency and poor in vivo functionality. There is an unmet need to establish novel methods for efficiently generating functional and transplantable therapeutic progenitors. 


Technical Details: Researchers at Johns Hopkins have developed a novel method for deriving hiPSC from any human somatic cell. The method, which expands upon previously reported chemical tankyrase/PARP inhibition, chemically reverts conventional, lineage-primed hiPSC lines into a preimplantation clinical-grade naïve epiblast-like pluripotent state with improved differentiation capacities. The resulting patient-specific hiPSC lines are stable, possess transcriptional, epigenetic, and biochemical features of human naïve epiblast, and are marked by high epigenetic plasticity, improved genomic integrity and superior multi-lineage functionality relative to conventional hiPSC. The researchers also developed a method for establishing defined, clinical-grade universal donor banks of multi-lineage progenitor cells from these novel naïve hiPSC lines for use with drug-mediated allogeneic tissue transplantation tolerance strategies. 


Value Proposition: 

  • The methods are current Good Manufacturing Practice (cGMP)-compliant  
  • The novel class of naïve hiPSC overcome the limited differentiation efficiency and poor functionality of conventional patient-specific hiPSC 
  • Universal hiPSC banks offer off-the-shelf regenerative cellular therapies 

Looking for Partners to: Develop & commercialize the technology as novel methods for generating clinical-grade donor-specific and universal donor therapeutic progenitors 


Stage of Development: Pre-Clinical

Data Availability: in vitro & in vivo


Inventors: Elias Zambidis & Ludovic Zimmerlin


Publication(s)/ Associated technology: JHU C16054 


Patent Information:
Title App Type Country Serial No. Patent No. File Date Issued Date Expire Date Patent Status
CLINICAL-GRADE THERAPEUTIC PROGENITORS GENERATED FROM TANKYRASE/PARP-INHIBITED PLURIPOTENT STEM CELL BANKS PCT: Patent Cooperation Treaty United States 18/785,343   7/26/2024     Pending
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For Information, Contact:
Vera Sampels
vsampel2@jhu.edu
410-614-0300
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