Cascade Biotechnology creates gene‑therapy platforms that deliver a stabilized, catalytically active complement component 3 variant (sC3) to specific tissues using lentiviral vectors, conjugate‑guided delivery, or localized administration. By engineering permanent, tissue‑specific expression of sC3, the company provides sustained, localized complement inhibition after a single dose, aiming to treat complement‑driven diseases such as Alzheimer’s, macular degeneration, renal inflammation, and autoimmune disorders.
Funding
Funding not disclosed
Founders
Product
Problem
Over‑activation of the complement system contributes to a wide range of diseases—including renal inflammatory disorders, neurodegenerative and psychiatric conditions, autoimmune neuromuscular diseases, and age‑related macular degeneration—but existing therapies lack precise tissue targeting and often require chronic dosing, leading to limited efficacy and systemic side effects.
Solution
Cascade Biotechnology develops gene‑therapy‑based complement depletion platforms that deliver a stabilized, catalytically active complement component 3 variant (sC3) to specific cell types using lentiviral vectors, conjugate‑guided delivery, or localized administration. By engineering permanent expression of sC3 in target tissues (e.g., renal cells, retinal pigment epithelium, neuronal cells), the approach provides sustained, localized complement inhibition after a single dose, reducing off‑target exposure. The platform integrates structural protein engineering to enhance sC3 stability and potency, and leverages tissue‑specific promoters or conjugate ligands to achieve precise cellular targeting. This enables long‑lasting therapeutic modulation of complement‑driven pathology across multiple disease indications.
Target Audience
Primary customers are pharmaceutical and biotech companies developing treatments for complement‑mediated diseases, as well as academic and clinical research groups seeking a durable, tissue‑specific complement inhibition platform.
Features
- Lentiviral vector system engineered for permanent integration and continuous secretion of sC3 in target cells
- Protein engineering of sC3 to increase catalytic efficiency, serum stability, and resistance to degradation
- Tissue‑specific targeting via cell‑type promoters, viral tropism, or conjugate‑guided delivery for retina, kidney, and CNS applications
- Single‑dose administration strategy designed to provide lifelong complement depletion in the treated organ
- Modular platform adaptable to multiple disease programs (e.g., diabetic nephropathy, lupus nephritis, AMD, Alzheimer’s, schizophrenia, NMO, MG)
- Preclinical validation pipelines including in‑vivo expression, complement activity assays, and disease‑model efficacy studies