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,…
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…
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…
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)…
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…
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…
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…
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…
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…
Translational Candidate H9 (WA09) embryonic stem cell-derived neural stem cells with a spinal cord identity (H9-NSCsc) Area of Impact Severe spinal cord injury Mechanism of Action Our candidate therapy for…
Get the latest CIRM news, information on upcoming meetings, and funding opportunities delivered to your inbox.
