Gene therapy

Gene therapy is emerging as a new modality that aims to address the root cause of a disease with inherited defects or a disorder that can be altered through genetic modification. The approach involves different mechanisms to modify the genetic composition of cells, including replacing or inactivating a disease-causing gene with a functional copy of the gene or through a gene-silencing approach, or introducing a new or modified gene into the cells that help treat a disease.

From ideation to clinical

Skyline Therapeutics’ deep expertise in the development of innovative adeno-associated virus (AAV) gene therapy is translated into our proprietary AAV platforms that enable the construction, characterization, and validation of vectors from ideation through to clinical development.

In AAV-based gene therapy development, the virus is used as gene transfer vector to deliver therapeutic transgenes. The discovery of novel AAV capsids is essential to enhancing the specificity, safety, and efficacy profile of next-generation AAV gene therapy.

Systematic capsid evolution

Skyline’s proprietary capDRIVE™ is an AAV capsid engineering and screening platform based on capsid encoding nucleic acid recovery technology. Performed through multi-round biopanning, the platform drives the efficient identification of novel AAV capsids with desired tissue tropism and transduction efficiency.

Diverse AAV libraries

Enabled by capDRIVE™, Skyline’s approach to novel AAV capsid discovery leverages both rational design based on our deep know-how of AAV tropism and structure, as well as experimental screenings carried out in multiple animal species to accelerate the process of identifying and characterizing capsids of interest.

Cutting-edge vector genome design

Skyline’s AAV vector engineering platform focuses on the creative design of therapeutic genes, as well as the regulatory elements. Our proprietary transgene genome engineering and optimization capabilities result in the enhanced quality and novelty of the transgene. The approach also involves de-targeting the non-target tissues, if necessary, to further elevate the therapeutic safety of the vector.