Dicerna Pharmaceuticals, Inc.

Shire Human Genetic Therapies, Inc.

Althea Technologies, Inc.

Sujit Basu

Bob Dale Brown

David Aaron Schwartz

Zahra Shahrokh

Pericles Calias

Jing Pan

Teresa Leah Wright

Lawrence Charnas

Wen-Li Chung

Paul Campolieto

Nazila Salamat-Miller

Katherine Taylor

Allister Fraser

Keethkumar Jain

Lawrence Bush

Sergey Pechenov

The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g. Metachromatic Leukodystrophy Disease), In some embodiments, the present invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.

Stable formulations for CNS delivery of arylsulfatase A

The present invention is based, at least in part, upon discovery of a process for identifying phase changing peptides. Such phase changing peptides are capable of enhancing in vitro and in vivo delivery of oligonucleotides (e.g., dsRNAs) in lipidic, vesicular, micellar and/or naked oligonucleotide formulations.

Phase changing formulations of nucleic acid payloads

The present invention relates to lipid compounds and uses thereof. In particular, the compounds include a class of cationic lipids having an amine moiety, such as an amino-amine or an amino-amide moiety. The lipid compounds are useful for in vivo or in vitro delivery of one or more agents (e.g., a polyanionic payload or an antisense payload, such as an RNAi agent).

Amine cationic lipids and uses thereof

Formulations containing complexed human growth hormone crystals are described. Also described are needleless injection systems for crystalline proteins.

Crystallized recombinant human growth factor formulations

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Newton, MA, United States of America

Sujit Basu