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

Phase I Study of Chimeric Antigen Receptor Engineered T Cells targeting CD33 for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia

  • Post author:
  • Post category:

Therapeutic Candidate or Device Immune T cells from a patient’s transplant donor engineered to express chimeric antigen receptors for targeted leukemia killing Indication Relapsed or refractory acute myeloid leukemia Therapeutic…

Continue ReadingPhase I Study of Chimeric Antigen Receptor Engineered T Cells targeting CD33 for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia

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

Stem-Derived IL13Ra2 Chimeric Antigen Receptor T cells for Patients with Melanoma and Advanced Solid Tumors

  • Post author:
  • Post category:

Therapeutic Candidate or Device Adult Stem-Like T cells engineered with chimeric antigen receptor (CAR) to target cancers expressing IL13Ra2, including melanoma. Indication Advanced cancers that express IL13Ra2, including melanoma. Therapeutic…

Continue ReadingStem-Derived IL13Ra2 Chimeric Antigen Receptor T cells for Patients with Melanoma and Advanced Solid Tumors

A Phase 2b, Randomized, Assessor-Masked Clinical Trial to Assess the Safety and Efficacy of the CPCB-RPE1 Implant in Subjects with Geographic Atrophy

  • Post author:
  • Post category:

Therapeutic Candidate or Device A patch comprised of a layer of stem cell-derived retinal pigmented epithelial (RPE) cells on a supporting matrix that is implanted under the retina Indication Geographic…

Continue ReadingA Phase 2b, Randomized, Assessor-Masked Clinical Trial to Assess the Safety and Efficacy of the CPCB-RPE1 Implant in Subjects with Geographic Atrophy

Evaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIV

  • Post author:
  • Post category:

Therapeutic Candidate or Device Cytomegalovirus (CMV)-specific T cells that express a chimeric antigen receptor (CAR) which targets and eliminates HIV-infected cells Indication Management of human immunodeficiency virus (HIV)/acquired immunodeficiency syndrome…

Continue ReadingEvaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIV

Phase 2b Clinical Study of KPI-012 Topical Ophthalmic Human Mesenchymal Stem Cell Secretome for the Treatment of Persistent Corneal Epithelial Defect

  • Post author:
  • Post category:

Therapeutic Candidate or Device KPI-012 is a human bone marrow mesenchymal stem cell secretome formulated as a topical ophthalmic therapeutic Indication Treatment of persistent corneal epithelial defect, a cornea defect…

Continue ReadingPhase 2b Clinical Study of KPI-012 Topical Ophthalmic Human Mesenchymal Stem Cell Secretome for the Treatment of Persistent Corneal Epithelial Defect