A phase 1/2 study to evaluate a bispecific CD19/CD20-directed CAR T cell, in refractory lupus nephritis and systemic lupus erythematosus

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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…

Continue ReadingA phase 1/2 study to evaluate a bispecific CD19/CD20-directed CAR T cell, in refractory lupus nephritis and systemic lupus erythematosus

Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A

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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

Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients

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Therapeutic Candidate or Device αβdepleted-HSC grafts and a reduced-intensity preparative conditioning regimen containing JSP191 Indication Treatment of patients with all genetic forms of Fanconi Anemia (FA) with evidence of cytopenias…

Continue ReadingReduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients

Sequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant

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Therapeutic Candidate or Device Mobilized peripheral blood stem cells from allogeneic donors depleted of TCRαβ+ T cells/CD19+ B cells Indication Renal failure due to one of four genetic and/or immunological…

Continue ReadingSequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant

A Double-Blind Randomized Placebo-Controlled Investigation of Autologous Muscle Derived Progenitor Cells for the Treatment of Dysphagia

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Therapeutic Candidate or Device Autologous Muscle Derived Progenitor Cells (AMDC) isolated from skeletal muscle biopsy Indication Subjects with dysphagia (swallowing difficulties) that develops following treatment for head and neck cancer…

Continue ReadingA Double-Blind Randomized Placebo-Controlled Investigation of Autologous Muscle Derived Progenitor Cells for the Treatment of Dysphagia