Therapeutic Candidate or Device Modified adult donor bone marrow-derived mesenchymal stem cells (Modified MSC) Indication Chronic motor deficit secondary to ischemic stroke Therapeutic Mechanism Local intracerebral delivery of Modified MSC…
Therapeutic Candidate or Device A cell therapy that delivers high levels of neurotrophic factors to the CNS Indication Amyotrophic lateral sclerosis (ALS) or Lou Gehrig Disease Therapeutic Mechanism The Cell…
Therapeutic Candidate or Device CNS10-NPC-GDNF - a neural progenitor cell secreting GDNF Indication ALS Therapeutic Mechanism This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As…
Therapeutic Candidate or Device The therapeutic candidate to be studied under this proposal is a gene therapy product for the treatment of DOK7 Congenital Myasthenic Syndrome Indication The target indication…
Therapeutic Candidate or Device Human embryonic stem cell-derived neural stem cells. Indication Spinal cord injury Therapeutic Mechanism The proposed neural stem cell therapy will be transplanted into a spinal cord…
Therapeutic Candidate or Device AAV9 Gene Therapy For An Ultra-Rare Disease Called CMT4J Indication CMT4J or the FIG4 Gene Therapeutic Mechanism Gene Therapy through an Intrathecal One-Time Injection Unmet Medical…
Therapeutic Candidate or Device AS-202, an antisense oligonucleotide Indication Amyotrophic Lateral Sclerosis Therapeutic Mechanism AS-202 targets PIKFYVE, a lipid kinase. By inhibiting PIKFYVE activity, it induces exosomal secretion, which robustly…
Therapeutic Candidate or Device MZ-9138 is an AAV9 gene therapy Indication WWOX-related epileptic encephalopathy Therapeutic Mechanism MZ-9138 delivered to the brain may transduce neurons to create functioning WWOX protein and…
