Patisiran

Researchers developed liposomal lipid nanoparticles (LNPs) with high bilayer lipid content that exhibit enhanced mRNA encapsulation and transfection potency compared to conventional Onpattro-like formulations.

• Acuitas Therapeutics, Children's Hospital of Philadelphia, and University of Pennsylvania successfully delivered the world's first personalized LNP-delivered CRISPR gene-editing therapy to an infant with urea cycle disorder, with no adverse events reported. • The groundbreaking therapy was developed, manufactured, and delivered in just six months, establishing a new model for rapid development of personalized gene therapies through cross-functional partnerships. • Acuitas also presented advances in targeted LNP delivery, including DARPin-conjugated formulations achieving up to 98% binding and 90% expression in human CD8+ T cells, expanding therapeutic applications beyond the liver.

siRNA therapeutics are experiencing rapid growth with over 150 industry-sponsored clinical trials conducted between 2020-2024, demonstrating a 79.5% CAGR and significant pharmaceutical investment.

• An FDA advisory committee voted 9-3 in favor of approving Alnylam's Onpattro (patisiran) for transthyretin amyloidosis cardiomyopathy (ATTR-CM), with a final decision expected by October 8th. • Despite FDA reviewers questioning the clinical significance of Onpattro's modest efficacy in the APOLLO-B trial, the panel was swayed by the drug's established safety profile and potential to address the underlying disease mechanism. • If approved, Onpattro would compete with Pfizer's Vyndamax/Vyndaqel (tafamidis), which generated $2.5 billion in sales last year, marking another milestone for RNA interference technology following its historic 2018 approval for ATTR polyneuropathy.

The FDA has approved Oxlumo (lumasiran), the first-ever treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare genetic disorder that causes kidney damage through excessive oxalate production.