Autologous BCMA CAR-T Cells for the Treatment of Relapsed Refractory Light Chain Amyloidosis

  • Post author:
  • Post category:

Therapeutic Candidate or Device NXC-201 Indication AL Amyloidosis Therapeutic Mechanism Genetically modified T-cell targeting B-cell maturation antigen (BCMA) Unmet Medical Need Amyloid light-chain (AL) amyloidosis is a devastating, rare plasma…

Continue ReadingAutologous BCMA CAR-T Cells for the Treatment of Relapsed Refractory Light Chain Amyloidosis

Phase 1 Study of Autologous E-SYNC T Cells in Adult Participants with EGFRvIII+ Glioblastoma

  • Post author:
  • Post category:

Therapeutic Candidate or Device Human T cells transduced with a lentiviral vector encoding anti-EGFRvIII synNotch-primed anti-EphA2/IL-13Rα2 chimeric antigen receptor. Indication Patients with MGMT unmethylated newly diagnosed GBM (Cohort 1), and…

Continue ReadingPhase 1 Study of Autologous E-SYNC T Cells in Adult Participants with EGFRvIII+ Glioblastoma

A Phase 2b Study of the Efficacy of a Novel Pro-Neurogenesis/Pro-Plasticity Drug for Bipolar Depression Using a Precision Psychiatry Approach

  • Post author:
  • Post category:

Therapeutic Candidate or Device ALTO-100 Indication Bipolar depression (I or II) Therapeutic Mechanism ALTO-100 increased hippocampal neurogenesis and neuroplasticity. Its effects are mediated by signaling through the brain derived neurotrophic…

Continue ReadingA Phase 2b Study of the Efficacy of a Novel Pro-Neurogenesis/Pro-Plasticity Drug for Bipolar Depression Using a Precision Psychiatry Approach

A PHASE 1B STUDY EVALUATING THE SAFETY AND EFFICACY OF AN ALLOGENEIC CELL THERAPY IN SUBJECTS WITH CLEAR CELL RENAL CELL CARCINOMA (ccRCC)

  • Post author:
  • Post category:

Therapeutic Candidate or Device An allogeneic anti-CD70 CAR-T cell product will be evaluated for the treatment of advanced or metastatic clear cell renal cell carcinoma (ccRCC). Indication Advanced or metastatic…

Continue ReadingA PHASE 1B STUDY EVALUATING THE SAFETY AND EFFICACY OF AN ALLOGENEIC CELL THERAPY IN SUBJECTS WITH CLEAR CELL RENAL CELL CARCINOMA (ccRCC)

A Phase I/IIa Study to Evaluate the Efficacy of Toca 511/Toca FC with Standard of Care Therapy in Newly Diagnosed High Grade Glioma

  • Post author:
  • Post category:

Therapeutic Candidate or Device DB107 (Toca 511), a retroviral replicating vector expressing yeast cytosine deaminase, which converts an antifungal prodrug to an anticancer drug Indication Newly diagnosed high-grade glioma Therapeutic…

Continue ReadingA Phase I/IIa Study to Evaluate the Efficacy of Toca 511/Toca FC with Standard of Care Therapy in Newly Diagnosed High Grade Glioma

A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease

  • Post author:
  • Post category:

Therapeutic Candidate or Device RP-A501, a recombinant Adeno-Associated Virus Serotype 9 containing the LAMP2B transgene Indication Danon Disease Therapeutic Mechanism The proposed product, RP-A501, is an in vivo gene therapy…

Continue ReadingA Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease

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

Phase I Trial of Locoregionally Delivered Autologous B7-H3 CAR T Cells (B7-H3CART) in Adults with Recurrent Glioblastoma Multiforme

  • Post author:
  • Post category:

Therapeutic Candidate or Device Autologous T cells genetically engineered to express a Chimeric Antigen Receptor targeting B7-H3 (B7-H3CART) Indication Brain tumors in adults: Glioblastoma Multiforme (GBM) Therapeutic Mechanism Progenitor B7-H3CART…

Continue ReadingPhase I Trial of Locoregionally Delivered Autologous B7-H3 CAR T Cells (B7-H3CART) in Adults with Recurrent Glioblastoma Multiforme