Sirna Therapeutics, Inc.

Inozyme Pharma, Inc.

Other

Alexion Pharmaceuticals, Inc.

Tayeba Khan

Vasant R Jadhav

Duncan F Brown

Walter R Strapps

Laura Sepp-Lorenzino

Kim Lynette Askew

Marija Tadin-strapps

Anthony Quinn

Gregory A Grabowski

Nelson Hsia

Zhiliang Cheng

W Charles O'Neill

Andre Marozsan

The present invention provides methods for improving cardiovascular function in a human patient (e.g., reducing hypertension), as well as methods of treating a cardiovascular disease, by administering a recombinant human soluble ectonucleotide pyrophosphatase phosphodiesterase (hsNPP1), active fragment or fusion protein thereof.

Methods of improving cardiovascular function and treating cardiovascular disease using a recombinant ectonucleotide pyrophosphatase phosphodiesterase (NPP1)

The present invention provides a method of treating NPP1 deficiency or NPP1-associated disease such as idiopathic infantile arterial calcification (IIAC), pseudoxanthoma elasticum, vascular calcification in chronic kidney disease (VCCKD), insulin resistance, hypophosphatemic rickets, myocardial ischemia, joint calcification, angioid streaks, and ossification of the posterior longitudinal ligament of the spine. The present invention provides a method for treating tissue calcification by administering soluble NPP1 to produce a transient increase in serum pyrophosphate levels.

Methods of treating tissue calcification

The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration. Specifically, the invention relates to methods comprising the in vivo delivery of short interfering nucleic acid (siNA) molecules that target a myostatin gene expressed by a subject, wherein the siNA molecule is conjugated to a lipophilic moiety, such as cholesterol. The myostatin siNA conjugates that are delivered as per the methods disclosed are useful to modulate the in vivo expression of myostatin, increase muscle mass and/or enhance muscle performance. Use of the disclosed methods is further indicated for treating musculoskeletal diseases or disorders and/or diseases or disorders that result in conditions in which muscle is adversely affected.

Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety

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Lexington, MA, United States of America

Tayeba Khan