BSIM's mission is to deliver quality drug candidates with high capital efficiency against transthyretin-mediated amyloidosis, including clinical manifestations and related pathologies that still represent unmet medical needs.
Our first products are directed against a rare and fatal pathology called Hereditary ATTR amyloidosis with Polyneuropathy (hATTR-PN), also known as Familial Amyloid Polyneuropathy (TTR-FAP). We are developing drug candidates that fit into different target-product profiles and thus enable the treatment of a broader range of hATTR-PN manifestations, including oculopathy (e.g. vitreous opacities, glaucoma) and central nervous system symptoms (e.g. seizures, stroke-like events, progressive dementia).
We are also working on development candidates for the treatment of TTR-related cardiomyopathies such as hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM), also known as Familial Amyloid Cardiomyopathy (TTR-FAC), and wild-type ATTR amyloidosis (wtATTR), also known as Senile Systemic Amyloidosis (SSA).