Development of Lentiviral Vectors for Gene Therapy in Skeletal Dysplasia

Time: 11:00 am
day: Post-Conference Discussion Day - Track B


  • InnoSkel focuses on gene therapy for inherited skeletal dysplasia
  • Type II collagenopathies (COL2A1 deficiency), such as Spondyloepiphyseal Dysplasia congenita (SEDc) and related, are rare pediatric disease indications with unmet medical needs
  • Systemic delivery of lentiviral vectors (LVV) can efficiently transduce cartilage cells in growth plates of juvenile SEDc disease mouse model and rescue the severe phenotype.
  • Discussing considerations on safety risks and mitigation strategies for the systemic delivery of integrative 3rd generation LVV