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.

Our pipeline

Skyline’s cutting-edge vector engineering and industry-leading capsid discovery capability, combined with deep understanding of disease biology, powers us to rapidly advance the development of novel therapies that target diseases across multiple therapeutic areas.

Discovery
Preclinical
Clinical
Open
Ocular
Wet Age-related Macular Degeneration
In-house
Age-related Macular Degeneration (AMD)
Age-related macular degeneration (AMD) is a progressive disease that leads to severe impairment of central vision. It is a leading cause of irreversible blindness in the elderly population. AMD can be categorized into two main groups: dry or non-neovascular AMD, and wet or neovascular AMD. wAMD is primarily driven by vascular endothelial growth factor (VEGF). wAMD presents with the growth of abnormal vessels, most often from the choroidal vessels, or from the retinal vessels, causing acute vision loss through leakage, edema and bleeding, and if chronic through fibrosis and atrophy.
Undisclosed
In-house
Undisclosed
In-house
Undisclosed
In-house
Neurological
Spinal Muscular Atrophy
In-house
Spinal muscular atrophy (SMA)
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder caused by a lack of a functional survival motor neuron 1 (SMN1) gene, which results in rapid and irreversible loss of motor neurons in the spinal cord. The patients with SMA suffer severely from muscle dysfunctions, including difficulties in breathing, swallowing and basic movement, which can lead to pre-mature death when left untreated in the most severe forms of the disease.
Undisclosed
In-house
Cardiovascular
Dilated Cardiomyopathy (Target 1)
In-house
Dilated cardiomyopathy
Dilated cardiomyopathy is a serious cardiac disorder in which structural or functional abnormalities of the heart muscle can lead to complications such as arrhythmia and heart failure, resulting in substantial morbidity and mortality. More than 50 mutated genes associated with DCM have been identified, accounting for 40-50% of familial DCM cases. Many of these genes encode proteins with important known functions in cardiomyocytes related to cytoskeletal, sarcomere and nuclear envelope biology.
Dilated Cardiomyopathy (Target 2)
In-house
Dilated cardiomyopathy
Dilated cardiomyopathy is a serious cardiac disorder in which structural or functional abnormalities of the heart muscle can lead to complications such as arrhythmia and heart failure, resulting in substantial morbidity and mortality. More than 50 mutated genes associated with DCM have been identified, accounting for 40-50% of familial DCM cases. Many of these genes encode proteins with important known functions in cardiomyocytes related to cytoskeletal, sarcomere and nuclear envelope biology.
Metabolic & Blood
Fabry
In-house
Fabry
Fabry disease is a rare inherited X-linked lysosomal storage disorder. It is caused by mutations in the alpha-galactosidase A (GLA) gene, which encodes the alpha-galactosidase A (α-Gal A) enzyme that breaks down complex glycolipids in the lysosomes. Loss of functional α-Gal A leads to a continuous build-up of glycolipids in the body, resulting in the cellular abnormalities and organ dysfunction that particularly affect small blood vessels, the heart, and kidneys.
Pompe
In-house
Pompe
Pompe disease is a rare autosomal recessive lysosomal storage disorder. It is caused by mutations in the acid-a-glucosidase (GAA) gene, which encodes an enzyme that breaks down glycogen to glucose. Deficiency in the GAA enzyme results in the aberrant accumulation of glycogen in various organs including heart, muscles, and CNS. This leads to a variety of symptoms including heart dysfunction, muscle weakness, and eventually respiratory failure.
Undisclosed
In-house
Undisclosed
In-house