Proteins that misfold and aggregate to form amyloid are associated with more than 25 human diseases that can gravely damage organs. Amyloid can affect any organ in the body, including the brain and peripheral organs.

We optimally target misfolded proteins to achieve clinical benefit across a number of indications, focusing on proteins like amyloid beta (Aβ), tau, alpha-synuclein, light chain and transthyretin.

Scientifically, we focus on genetically-associated and biologically-validated targets implicated in disease. For example:

  • The misfolding and aggregation of the Aβ peptide leads to a build-up of amyloid protein in the brain, which most scientists believe is the primary cause of Alzheimer’s disease.
  • A rare, progressive, and fatal disease called AL amyloidosis where clonal plasma cells overproduce light chain proteins that misfold, aggregate, and deposit as amyloid in vital organs such as the heart.

Decades of our own scientific investigation augmented by the work of others have elucidated that antibody therapeutics can result in meaningful clinical benefit for patients with the right combination of targeting the appropriate epitope, optimal binding strength, and the right clinical trial design with the appropriate endpoints in the right patient population.

Increasingly, these elements are aligning, and because of advances in our knowledge that informs the basic, clinical and regulatory sciences, we find ourselves with an expanding pipeline of investigational therapies.

While we are modality agnostic, we have deep expertise in antibody targeting and leverage insights and understanding around neurological dysfunction and the biology of misfolded proteins to develop a diverse pipeline that also includes small molecule and vaccine approaches. Advances in clinical trial design have revealed a path forward that makes developing new therapies possible, targeting the pathogenic proteins that underlie progressive and fatal neurodegenerative and peripheral amyloid diseases to address areas of high unmet need for patients.

Learn more about Prothena’s pipeline