A phase 1/2 study to evaluate a bispecific CD19/CD20-directed CAR T cell, in refractory lupus nephritis and systemic lupus erythematosus
Therapeutic Candidate or Device IMPT-514 is an autologous T-cell product genetically modified to express a chimeric antigen receptor targeting CD19 and CD20 Indication Active, refractory lupus nephritis (LN) and systemic…
RPESC-RPE-4W Therapy for dry Age-related Macular Degeneration
Therapeutic Candidate or Device Retina pigment epithelial stem cell (RPESC)-derived RPE progeny at 4 weeks of differentiation (RPESC-RPE-4W) Indication Dry age-related macular degeneration (dry AMD). Therapeutic Mechanism Dry AMD involves…
The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele
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…
Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
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…
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