At BSIM Therapeutics we are developing the next generation of therapies tailored to treat transthyretin-mediated amyloidoses and their associated co-morbidities.
ATTR - PNS/CNS
Our most potent transthyretin stabilizers display optimal balance between potency, selectivity for TTR and safety in long-term treatments. They were designed in ways that enable them to reach the blood plasma, thus stalling the progression of PNS symptoms in hATTR-PN, and also to cross of the blood brain barrier (BBB) and the blood-cerebrospinal fluid barrier (BCSFB) to reach the cerebrospinal fluid – thus preventing ATTR CNS manifestations.
ATTR - HEART
Because some of our most potent transthyretin stabilizers are also resilient to the effect of mutations in TTR, they may be used in the treatment of cardiomyopathies mediated by different TTR forms, such as TTR V122I and wild-type TTR, that deposit as amyloid fibrils in the heart.
ATTR - EYE
Endowed with specific molecular properties, some of our potent transthyretin stabilizers have been crafted to reach the inner compartments of the eye and thus prevent vitreous opacities, conjunctival microaneurysms, dry eye and glaucoma.
Phase I/ II
D - Discovery
O - Optimization
PC - Preclinical
C - Clinical (Phase I/II)