CRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s disease

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Research Objective Discovery of a novel therapeutic candidate for Parkinson’s disease which modifies gene expression using human stem cell-derived neurons to halt the neurodegenerative disease process. Impact Stopping the neurodegenerative…

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Autologous cell therapy for Parkinson’s disease using iPSC-derived DA neurons

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Research Objective Autologous human dopaminergic neurons derived from patient-specific induced pluripotent stem cells Impact Parkinson's disease Major Proposed Activities Characterize differentiation from all 10 patient cell linesCharacterize functionality of patient…

Continue ReadingAutologous cell therapy for Parkinson’s disease using iPSC-derived DA neurons

A new phenotypic screening platform that identifies biologically-relevant targets and lead compounds for the treatment of Parkinson’s disease

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Research Objective Demonstrate that our HitFinder™ library can be screened for phenotypic changes in A53T-IPSC-derived dopaminergic neurons and use a secondary handle to identify the targets responsible. Impact This technology…

Continue ReadingA new phenotypic screening platform that identifies biologically-relevant targets and lead compounds for the treatment of Parkinson’s disease

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…

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

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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A Phase 1b Safety Study for MRI guided delivery of AAV2-GDNF for the treatment of Parkinson’s disease

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Therapeutic Candidate or Device AAV2-GDNF is a gene therapy product encoding Glial cell line-Derived Neurotrophic Factor (GDNF) Indication Parkinson's disease Therapeutic Mechanism AAV2-GDNF will be delivered into the putamen. GDNF…

Continue ReadingA Phase 1b Safety Study for MRI guided delivery of AAV2-GDNF for the treatment of Parkinson’s disease

MRI Guided Delivery of Neural Progenitor Cells Secreting GDNF for the Treatment of Parkinson’s disease

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Therapeutic Candidate or Device CNS10-NPC-GDNF is a neural progenitor cell line transfected with glial cell line derived neurotrophic factor (GDNF) Indication Mid-stage Parkinson's disease (UPDRS stage III or lower) Therapeutic…

Continue ReadingMRI Guided Delivery of Neural Progenitor Cells Secreting GDNF for the Treatment of Parkinson’s disease