Cirrus Therapeutics announced the completion of an $11 million seed financing round to advance its novel adeno-associated virus (AAV) gene therapy targeting dry age-related macular degeneration (AMD), a leading cause of vision loss affecting over 200 million people worldwide. The Cambridge-based ocular immunology biotech secured funding from ClavystBio, Polaris Partners, and SEEDS to support IND-enabling studies for its lead program.
The company's innovative approach focuses on restoring IRAK-M protein, a central regulator of the eye's immune homeostasis that naturally decreases with age and falls more sharply in people with dry AMD. This protein loss leaves the retina vulnerable to chronic inflammation, mitochondrial dysfunction, and oxidative stress that drive AMD onset and progression.
Novel Therapeutic Target Addresses Aging Mechanism
"Current approved treatments for dry AMD, and much of the therapeutic pipeline, target a single disease-implicated pathway -- primarily the complement cascade. To date, however, existing approaches have yet to demonstrate functional benefits," said Professor Andrew Dick, Co-founder and Chief Scientific Advisor of Cirrus. "Replenishing IRAK-M expression offers an exciting opportunity to target an underlying driver of retinal degeneration – aging itself – thwarting the multi-pathway activity that leads to AMD and preventing or reversing vision loss."
The therapeutic target emerged from breakthrough research published in Science Translational Medicine in June 2024 by Cirrus Therapeutics, the University of Bristol, and the UCL Institute of Ophthalmology. The study identified IRAK-M as a key regulator of ocular health expressed predominantly in retinal pigment epithelial (RPE) cells. In preclinical models, restoring IRAK-M to normal levels significantly protected against retinal degeneration.
Gene Therapy Paradigm Shift for Common Disease
AMD affects people aged 50 and over, with projections indicating the global burden will surge to 288 million by 2040 as populations age. The progressive disease destroys the macula, the central part of the retina responsible for sharp, detailed vision, typically beginning with poor low-light vision and slight blurriness before expanding into a central blind spot that impairs reading, driving, and face recognition.
"By pairing the disease-modifying IRAK-M target with a modality that would enable a one-time treatment, we aim to protect and preserve vision," said Ying Kai Chan, PhD, Chief Executive Officer and Co-founder of Cirrus Therapeutics. "This not only represents a major medical advance to address a highly prevalent blinding disease, but also heralds a paradigm shift for the application of gene therapy, for which approved products have been reserved for rare, monogenic diseases thus far."
Advantages of Ocular Gene Therapy Platform
The ocular application offers compelling advantages for gene therapy deployment. A durable, sustained effect from a one-time treatment would contrast sharply with the continual monthly or bi-monthly intraocular injections required by existing approved therapies. The eye's small size requires only minimal therapy doses, providing manufacturing and cost advantages that make large population deployment practical.
Additionally, the eye functions as a contained and immune-privileged tissue compartment, reducing off-target effects and immunogenicity challenges often associated with systemic gene therapy applications.
"Cirrus' bold approach focuses on pioneering novel yet well-validated biology from human genetics and aging data to deliver transformational impact in dry AMD, a disease of high unmet need," said Khoo Shih, PhD, Chief Executive Officer of ClavystBio. "At ClavystBio, we are committed to backing Cirrus and its talented founders Kai and Andrew to advance this important therapy toward the clinic, and build its earlier stage pipeline."
The seed funding will advance Cirrus's lead program into IND-enabling studies while supporting development of a broader pipeline of next-generation ocular medicines targeting chronic blinding diseases.
