Disease Focus: Neurological Disorders
Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.
Translational Candidate Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy. Area of Impact Non-destructive treatment option for drug-resistant focal epilepsy patients with reduced immunosuppression regimen and universal recipient eligibility Mechanism of Action The proposed therapeutic candidate comprises inhibitory interneurons that would be delivered in a […]
Development of AS-241, an UNC13A Targeting Antisense Oligonucleotide (ASO) Treatment for ALS, for IND-enabling Studies
Translational Candidate AS-241, an antisense oligonucleotide Area of Impact Amyotrophic Lateral Sclerosis Mechanism of Action AS-241 targets the cryptic exon (CE) of UNC13A and suppresses CE inclusion during RNA splicing, inhibits nonsense-mediated decay, and increases full length mRNA and protein levels Unmet Medical Need To date, therapeutic options for ALS have been limited, and disease-modifying […]
A high quality, accessible cell therapy for Parkinson’s Disease produced in a scalable bioreactor system for 3D cell expansion and differentiation
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 cell culture technology that addresses the manufacturing bottleneck and creates a high-quality scalable cell therapy. Mechanism of Action Dopamine producing cells are implanted into the […]
Development of Autologous Cell Replacement Therapy for Parkinson’s Disease: Path to Personalized Treatment
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 are lost in Parkinson's Disease (PD). While approaches using fetal tissue / allogeneic stem cells show great promise, they are not sufficiently personalized to provide […]
Development of a Gene Therapy for the Treatment of Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting
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 which causes profound disability and severe health impact Mechanism of Action Patients with Pitt Hopkins Syndrome have heterozygous mutations in the Transcription Factor 4 (TCF4) […]
CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia
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) for which there is no effective treatment available Mechanism of Action The proposed therapy intervention is intended to impact the target indication of Friedreich's ataxia […]
A human neural stem cell therapeutic candidate for the treatment of chronic cervical spinal cord injury
Translational Candidate The therapeutic candidate is a central nervous system tissue-derived GMP line developed under DISC2-10753 with an established GMP qualified seed bank. Area of Impact The target is chronic cervical spinal cord injury, which represents approximately 59% of clinical spinal cord injury cases. Mechanism of Action Integration of transplanted human neural stem cells is […]
Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia
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 differentiation protocols. Mechanism of Action Preliminary in vivo efficacy studies indicate that the MOA is in the promotion of new connections in the brain after […]
Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia
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 differentiation protocols. Mechanism of Action Preliminary in vivo efficacy studies indicate that the MOA is in the promotion of new connections in the brain after […]
Human Neural Stem Cells (hNSCs) for neuroprotection in perinatal hypoxic-ischemic brain injury (HII)-Pre-IND-enabling Studies
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 of cerebral palsy & cognitive disability Mechanism of Action hNSCs rescue the penumbra, the part of the brain lesion following perinatal asphyxia that still has […]