Alnylam Pharmaceuticals, Inc.

Novartis Ag

Mark Keating

Mark K Schlegel

James D McIninch

Adam Castoreno

Charalambos Kaittanis

Elane Fishilevich

Kristina Yucius

Sarah Solomon

Martin A Maier

Stuart Milstein

Kevin Fitzgerald

Vasant R Jadhav

Jayaprakash K Nair

Scott Glaser

Igor Splawski

Robert W Dunn

Kirk Brown

Mangala Meenakshi Soundarapandian

John Dominy

Carla Klattenhoff

Patrick Haslett

The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the Coagulation Factor V (F5) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an F5 gene and to methods of treating or preventing an F5-associated disease, e.g., a disorder associated with thrombosis, in a subject.

Coagulation factor V (F5) iRNA compositions and methods of use thereof

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the complement component C3 gene (C3). The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., cold agglutinin disease (CAD), warm autoimmune hemolytic anemia, and paroxysmal nocturnal hemoglobinuria (PNH), lupis nephritis (LN), bullous pemphigoid, Pemphigus, e.g., Pemphigus vulgaris (PV) and Pemphigus foliaceus (PF), and C3 glomerulopathy.

Complement component C3 iRNA compositions and methods of use thereof

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the complement component C3 gene (C3). The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., cold agglutinin disease (CAD), warm autoimmune hemolytic anemia, and paroxysmal nocturnal hemoglobinuria (PNH), lupis nephritis (LN), bullous pemphigoid,, e.g.,(PV) and(PF), and C3 glomerulopathy.

The present disclosure is directed to methods of immunization and methods for generating antibodies using compositions comprising cationic lipids and polynucleotide molecules, such as polyribonucleotide molecules, e.g., mRNA, which code for immunogens (e.g., a target protein or a fragment thereof).

mRNA-mediated immunization methods

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as cerebral amyloid angiopathy (CAA) and early onset familial Alzheimer disease (EOFAD or eFAD), using such dsRNAi agents and compositions.

Amyloid precursor protein (APP) RNAi agent compositions and methods of use thereof

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Weston, MA, United States of America

Mark Keating