The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele

  • Post author:
  • Post category:

Therapeutic Candidate or Device Allogeneic Placenta-derived Mesenchymal Stem Cells Seeded on Cook Biodesign® Dural Graft Extracellular Matrix (PMSC-ECM) Indication Myelomeningocele (MMC) -or Spina Bifida -diagnosed prenatally Therapeutic Mechanism Placenta-derived mesenchymal…

Continue ReadingThe CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele

Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A

  • Post author:
  • Post category:

Therapeutic Candidate or Device Investigational personalized antisense oligonucleotide drug (nL-SCN2A-002) Indication SCN2a-associated genetic disorder Therapeutic Mechanism The study participant has a pathogenic de novo p.R853Q gain-of-function amino acid substitution mutation…

Continue ReadingPersonalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A

A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

  • Post author:
  • Post category:

Therapeutic Candidate or Device B cells will be isolated from patients suffering MPSI. These will be transformed with a normal copy of the gene and re-introduced into the patient Indication…

Continue ReadingA Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

Phase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

  • Post author:
  • Post category:

Therapeutic Candidate or Device NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells. Indication Focal epilepsy; drug-resistant mesial temporal lobe epilepsy. Therapeutic Mechanism NRTX-1001 is an…

Continue ReadingPhase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

Safety and Tolerability Study of Neural Stem Cells (NR1) in Subjects with Chronic Ischemic Subcortical Stroke

  • Post author:
  • Post category:

Therapeutic Candidate or Device A human embryonic derived, non-genetically modified neural stem cell (NR1), originally derived from the Wi-Cell H-9 line Indication Patients with chronic motor deficits, from 6 to…

Continue ReadingSafety and Tolerability Study of Neural Stem Cells (NR1) in Subjects with Chronic Ischemic Subcortical Stroke

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

  • Post author:
  • Post category:

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

A Double-Blind, Controlled Ph 2b Study of the Safety and Efficacy of Modified Stem Cells in Patients with Chronic Motor Deficit from Ischemic Stroke

  • Post author:
  • Post category:

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…

Continue ReadingA Double-Blind, Controlled Ph 2b Study of the Safety and Efficacy of Modified Stem Cells in Patients with Chronic Motor Deficit from Ischemic Stroke

A Phase 3, Randomized, Placebo-controlled Multicenter Study to Evaluate Efficacy & Safety of Repeated Administrations of NurOwn® in Patients with ALS

  • Post author:
  • Post category:

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…

Continue ReadingA Phase 3, Randomized, Placebo-controlled Multicenter Study to Evaluate Efficacy & Safety of Repeated Administrations of NurOwn® in Patients with ALS