ALN-APP

Alnylam showcased multiple potential blockbuster therapies in ATTR amyloidosis, cardiovascular disease, and neuroscience, including the TRITON Phase 3 program for nucresiran, which offers greater than 95% TTR knockdown with twice-annual dosing.

Alnylam anticipates FDA approval for vutrisiran to treat ATTR amyloidosis with cardiomyopathy by March 23, 2025, and aims for EU and Japan approvals later in the year.

Mivelsiran, an siRNA targeting APP, demonstrates durable reduction of APP metabolites in CSF and is being evaluated for cerebral amyloid angiopathy (CAA).

The FDA has placed a clinical hold on Rapport Therapeutics' RAP-219 IND application for diabetic peripheral neuropathic pain (DPNP) due to protocol design concerns.

Early-stage data on mivelsiran, an RNA therapeutic, shows encouraging results for treating patients with early Alzheimer's disease.