Unmet Need
Antiphospholipid
syndrome (APS) is a rare disorder of the immune system that causes an increased
risk of blood clots. Long-term anticoagulation with a vitamin K antagonist
remains the standard of care for thrombotic APS, yet in severe cases termed
catastrophic APS (CAPS), best available treatments still reach >40%
mortality. Current methods lack an effective assay to identify patients with
complement-mediated diseases who could benefit from treatment with complement
inhibitors, as existing technologies do not adequately assess complement
activity and pathway-specific inhibition. There is a need for therapies
beyond anti-coagulation and also a diagnostic tool to differentiate severe CAPS
cases from treatable APS.
Technology
Overview
Complement
inhibition has emerged as an attractive therapeutic strategy based on evidence
of complement activity in patients with APS. The JHU inventors have developed a
diagnostic assay that uses flow cytometry and modified Ham assays to identify
patients with complement-mediated diseases. The assay measures C5b-9
deposition, enabling targeted complement inhibitor treatment based on specific
pathway involvement, thus improving treatment efficacy and reducing adverse
effects.
This approach allows for precise identification of patients likely to benefit from complement inhibitors, enabling targeted treatment and potentially reducing adverse effects by assessing complement activation and pathway involvement.
Stage
of Development
The
inventors have conducted a study with APS patients to collect data on assay
efficacy.
Data Availability
Data available upon request.
Publication
Unmet Need
Antiphospholipid
syndrome (APS) is a rare disorder of the immune system that causes an increased
risk of blood clots. Long-term anticoagulation with a vitamin K antagonist
remains the standard of care for thrombotic APS, yet in severe cases termed
catastrophic APS (CAPS), best available treatments still reach >40%
mortality. Current methods lack an effective assay to identify patients with
complement-mediated diseases who could benefit from treatment with complement
inhibitors, as existing technologies do not adequately assess complement
activity and pathway-specific inhibition.
There is a need for therapies beyond anti-coagulation and also a
diagnostic tool to differentiate severe CAPS cases from treatable APS.
Technology Overview
Complement
inhibition has emerged as an attractive therapeutic strategy based on evidence
of complement activity in patients with APS. The JHU inventors have developed a
diagnostic assay that uses flow cytometry and modified Ham assays to identify
patients with complement-mediated diseases.
The assay measures C5b-9 deposition, enabling targeted complement
inhibitor treatment based on specific pathway involvement, thus improving
treatment efficacy and reducing adverse effects.
This approach allows for precise identification of patients likely to benefit from complement inhibitors, enabling targeted treatment and potentially reducing adverse effects by assessing complement activation and pathway involvement.
Stage of Development
The
inventors have conducted a study with APS patients to collect data on assay
efficacy.
Data Availability
Data available upon request.
Publication
Ranjan N, Cole MA, Gerber GF, Crowther MA, Braunstein EM, Flores-Guerrero D, Haddaway K, Reed A, Streiff MB, McCrae KR, Petri M, Chaturvedi S, Brodsky RA. Genetic and epigenetic dysregulation of CR1 is associated with catastrophic antiphospholipid syndrome. Ann Rheum Dis. 2025 Dec;84(12):2034-2048. doi: 10.1016/j.ard.2025.07.016. Epub 2025 Aug 21. PMID: 40841298.
Pending patents
