A high quality, accessible cell therapy for Parkinson’s Disease produced in a scalable bioreactor system for 3D cell expansion and differentiation

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Translational Candidate Human pluripotent stem cell expanded and differentiated dopaminergic precursor cells to treat Parkinson’s Disease at high quantity and high quality Area of Impact This candidate uses a 3D…

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Development of Autologous Cell Replacement Therapy for Parkinson’s Disease: Path to Personalized Treatment

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Translational Candidate autologous iPSC-derived dopaminergic progenitor cells Area of Impact Parkinson's Disease Mechanism of Action Autologous iPSC-derived dopaminergic progenitor cells represent a promising strategy to replace the nigrostriatal cells which…

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Development of a Gene Therapy for the Treatment of Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting

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Translational Candidate MZ-1866 is a recombinant AAV9 based gene therapy containing the transgene encoding Transcription Factor 4 (TCF4) Area of Impact Pitt Hopkins Syndrome is a rare genetic neurological disease…

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CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia

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Translational Candidate Autologous human CD34+ HSPC of patients with Friedreich’s ataxia, modified ex vivo using CRISPR/Cas9 to remove the GAA expansion mutation in frataxin Area of Impact Friedreich’s ataxia (FRDA)…

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Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia

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Translational Candidate Human induced pluripotent stem cell-derived glial enriched progenitors Area of Impact Vascular dementia and white matter stroke, addressing a current bottleneck of poor scale up for existing cell…

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Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia

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Translational Candidate Human induced pluripotent stem cell-derived glial enriched progenitors Area of Impact Vascular dementia and white matter stroke, addressing a current bottleneck of poor scale up for existing cell…

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Human Neural Stem Cells (hNSCs) for neuroprotection in perinatal hypoxic-ischemic brain injury (HII)-Pre-IND-enabling Studies

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Translational Candidate An established stable human neural stem cell line unmanipulated genetically & propagated under defined conditions Area of Impact Perinatal asphyxia (also called hypoxic-ischemic injury), a major untreatable cause…

Continue ReadingHuman Neural Stem Cells (hNSCs) for neuroprotection in perinatal hypoxic-ischemic brain injury (HII)-Pre-IND-enabling Studies

Development of a human stem cell-derived inhibitory neuron therapeutic for the treatment of chronic focal epilepsy

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Translational Candidate A cellular therapeutic comprised of inhibitory nerve cells produced from human stem cells Area of Impact Drug-resistant chronic temporal lobe epilepsy Mechanism of Action The product candidate is…

Continue ReadingDevelopment of a human stem cell-derived inhibitory neuron therapeutic for the treatment of chronic focal epilepsy