We are leveraging the duality of our therapeutic
and diagnostic approaches to seek to become the leader in precision treatment of neurodegenerative diseases. The goal of precision
medicine is to deliver optimally targeted and timed interventions tailored to an individual's molecular drivers of disease. The
biggest limitation in neurodegenerative disease management is the lack of appropriate biomarkers and reliable diagnostics for early
disease detection and the absence of approved disease-modifying therapies. We believe that the future treatment paradigm for neurodegenerative
diseases will likely involve early disease diagnosis and combination therapy, leveraging both symptomatic and disease-modifying
treatments, with different disease-modifying treatments used at various points in the progression of the disease. We believe that
our multi-pronged approach to neurodegenerative disease diagnosis and treatment may result in the generation of individualized
treatment options for patients and improve clinical outcomes.
Strategically collaborating or selectively
partnering for the development and commercialization of product candidates
Historically, we have relied on collaboration
agreements with leading pharmaceutical companies to leverage their scientific, development, manufacturing and commercialization
expertise and other resources in order to accelerate the development of our product candidates. To date, we have entered into collaboration
agreements with leading global pharmaceutical companies, including two collaborations with Genentech, one with Lilly and one with
Janssen, a Johnson & Johnson company. We believe that these partnerships validate our core strategy of discovering safe
and efficacious therapies using our proprietary platforms and advancing them through the various stages of regulatory approval.
In the future, for any approved products targeting large markets, we may selectively partner with leading companies that we believe
can contribute manufacturing and marketing expertise, geographic reach and other resources and know-how that can enhance the value
of these approved products. In this respect, we established a strategic partnership with WuXi Biologics for their expertise in
manufacturing biologicals as well as the application of AC Immune’s vaccine portfolio in China and potential collaborations
with AC Immune’s SupraAntigen platform.
Our Approach to Treating Diseases Related
to Protein Misfolding
Protein folding and unfolding are important
ways of regulating the protein’s biological activity and cellular location. Misfolding of proteins occurs due to a breakdown
of cellular quality control systems, and is a common feature of many neurodegenerative diseases. Research has shown that misfolded
proteins are not only unable to carry out their normal functions, but also aggregate to form deposits in the brain that eventually
lead to neuronal damage and cell death. The progression of neurodegenerative diseases, such as AD and PD, is linked to the misfolded
conformations of proteins, such as Abeta, Tau, alpha-synuclein and TDP-43.
The diagram above shows how, in today’s
understanding, misfolded proteins play a key role in the pathology of neurodegenerative diseases. Typically, protein misfolding
occurs during cellular stress, which can be triggered by many different causes, including oxidation and a lack of growth factors.
A cascade of molecular events begins with the misfolding of single proteins within a cell that then continue to aggregate to ultimately
form plaques and tangles. These misfolded proteins are then exported and spread to healthy cells nearby, causing normal proteins
to misfold in a process known as seeding. This process eventually leads to cell death in various areas of the brain and is linked
to a decline in cognitive function.