At Skylark Bio, we are developing gene therapies designed to deliver a functional copy of the gene needed to restore normal cellular function to the appropriate cell populations. Our platform combines engineered AAV vectors, cell-specific promoters, and targeted local administration to maximize precision and specificity.
By restoring expression and function of a missing or altered gene, we aim to address the underlying cause of disease rather than simply managing its symptoms.
Engineering Precision at Every Step
Targeted, Micro‑dose Gene Therapies
Our differentiated AAV capsids are designed for precise local delivery of micro-dose gene therapies, minimizing systemic exposure, and immunogenicity while supporting lower doses and more efficient manufacturing.
Cell-specific Expression Control
Our promoter-based gene regulatory elements activate expression only in the appropriate cell populations, enabling broad reach with precise control of protein production.
Designed for Durability
In the monogenic diseases we are pursuing, the target cells are long-lived and largely non-dividing. Because these cells are not routinely replaced, therapeutic genetic material is not progressively diluted through normal cell turnover, supporting long-term durability after a single administration.
Focused Programs. Scalable Platform.
We are developing locally delivered gene therapies for monogenic diseases in which loss of protein function drives disease pathology. By precisely targeting the affected cells, our therapies aim to restore protein production in the cells where it has been lost. Our hearing loss programs serve as the first application of a broader platform with potential across multiple genetically defined disorders, particularly in the CNS.
Publications
Translating Discovery into Impact.
Skylark Bio's scientific foundation is built on pioneering research spanning gene delivery, inner-ear biology, and genetic hearing loss.
