Science

TfR1 CapX (CNS-Targeted Capsid)

Shuttling therapies to the CNS

We have engineered a central nervous system (CNS)-targeting AAV capsid that binds to the human Transferrin Receptor 1 (TfR1).

Apertura’s TfR1 CapX can cross the blood-brain barrier (BBB) and deliver therapeutic cargo throughout the central nervous system (CNS) via systemic administration.

Why TfR1 CapX?

hTfR1 is a well-characterized receptor with more than 40 years of research, an approved therapy, and several clinical programs leveraging its biology. Apertura is the first to successfully target hTfR1 with an AAV capsid, enabling a known and reliable BBB-shuttling mechanism across patient populations.
Our capsid was engineered for human receptors from the outset. This increases the likelihood of clinical translation compared to approaches that rely on non-human primate (NHP) discovery followed by back-mapping to human biology.

All mammalian brains are heavily vascularized, with no cell in the brain being more than approximately 20 microns from a blood vessel (Ref). For a drug to have broad distribution across the CNS, IV is the route of choice. Since all mammalian brains are similarly vascularized, IV administration facilitates interspecies scaling, avoiding the limitations of local administration.

TfR1 CapX is expected to enter the clinic in several programs over the next 6-12 months. This positions TfR1 CapX as the leading IV-administered, BBB-crossing AAV capsid. Clinical readiness is supported by preclinical development, regulatory engagement, and established CDMO manufacturing.

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