Transforming lives of patients with rare disease

Redefining science for treating rare diseases

Our science matters for rare diseases

Reaching out to unknowns via innovation

Pioneering treatments for unmet needs

Dedicated to providing quality of life

What we do

We have developed anti-complement antibodies as a treatment for complement-mediated disorders. These humanized monoclonal antibodies selectively block the complement alternative pathway (CAP) and do not block the complement classical pathway (CCP) which is required to control infections. Our two lead candidate humanized monoclonal antibodies (Anti-C3b and Anti-Bb) are unique in their mechanism of action and uniquely block the CAP but not the CCP.

Both antibodies inhibit C3b and C5b-9 formation in serum from PNH patients suggesting that our two lead candidates are suitable for treating PNH disease with superiority over Soliris. Anti-Bb and Anti-C3b antibodies bind their target proteins with picomolar affinity.

We have generated significant in vitro and ex vivo data on Anti-C3b and Anti-Bb humanized monoclonal antibodies demonstrating that these antibodies block the CAP by blocking the formation of C3b and C5b-9. Unlike Soliris, our antibodies do not block the CCP. We have created an exciting pipeline of therapeutic candidates especially for orphan diseases such as Paroxysmal Nocturnal Hemoglobinuria (PNH), C3-Glomerulopathy (C3G), and Neuromyelitis Optica (NMO). We believe that our portfolio of therapeutic antibodies has the potential for a better safety and an upgraded efficacy profile than currently marketed drugs including those that are under development.

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Research & development

Our technology for selectively targeting the alternative pathway is supported by numerous issued patents and those in the process of being issued. Our know-hows and trade secrets protect the selection, identification and development of therapeutic candidates that are being developed for rare (orphan and ultra orphan) diseases. We have streamlined our drug development path to enable the development of highly innovative therapeutic clinical antibodies.  Our portfolio of therapeutic antibodies have undergone a rigorous re-iterative selection process to identify the best-fit candidates to be used as a drug.

We have achieved remarkable success in advancing our drug candidates that have a unique mechanism of action compared to other known research and commercial programs. Our current success in pre-clinical, pharmacological, and toxicological studies is a testament to the quality of our lead selected candidates. These data together provides compelling evidence that our selected antibodies would have an excellent safety and efficacy profile in patients.  Our success in creating high concentration formulations along with longer half-life of our antibodies warrants the development of subcutaneously administered drug candidates providing easier dosing schedule for chronic indications such as hemolytic disorders. 

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Product Pipeline

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