Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer). The synthetic processes described herein may be advantageous to scaling up oligomer synthesis while maintaining overall yield and purity of a synthesized oligomer.

Provided herein are lipid compounds that can be used in combination with other lipid components, such as neutral lipids, cholesterol and polymer conjugated lipids, to form lipid nanoparticles for delivery of therapeutic agents (e.g., nucleic acid molecules) for therapeutic or prophylactic purposes, including vaccination. Also provided herein are lipid nanoparticle compositions comprising said lipids.

N.sup.2-phosphinyl amidine compounds, N.sup.2-phosphinyl amidinates, N.sup.2-phosphinyl amidine metal salt complexes, N.sup.2-phosphinyl amidinate metal salt complexes are described. Methods for making N.sup.2-phosphinyl amidine compounds, N.sup.2-phosphinyl amidinates, N.sup.2-phosphinyl amidine metal salt complexes, and N.sup.2-phosphinyl amidinate metal salt complexes are also disclosed. Catalyst systems utilizing the N.sup.2-phosphinyl amidine metal salt complexes and N.sup.2-phosphinyl amidinate metal salt complexes are also disclosed along with the use of the N.sup.2-phosphinyl amidine compounds, N.sup.2-phosphinyl amidinates, N.sup.2-phosphinyl amidine metal salt complexes, and N.sup.2-phosphinyl amidinate metal salt complexes for the oligomerization and/or polymerization of olefins.

N.sup.2-phosphinyl amidine compounds, N.sup.2-phosphinyl amidinates, N.sup.2-phosphinyl amidine metal salt complexes, N.sup.2-phosphinyl amidinate metal salt complexes are described. Methods for making N.sup.2-phosphinyl amidine compounds, N.sup.2-phosphinyl amidinates, N.sup.2-phosphinyl amidine metal salt complexes, and N.sup.2-phosphinyl amidinate metal salt complexes are also disclosed. Catalyst systems utilizing the N.sup.2-phosphinyl amidine metal salt complexes and N.sup.2-phosphinyl amidinate metal salt complexes are also disclosed along with the use of the N.sup.2-phosphinyl amidine compounds, N.sup.2-phosphinyl amidinates, N.sup.2-phosphinyl amidine metal salt complexes, and N.sup.2-phosphinyl amidinate metal salt complexes for the oligomerization and/or polymerization of olefins.

The invention features compounds of the general formula (I) in which the variable groups are as defined herein, and to their preparation and use.

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The invention features compounds of the general formula (I) in which the variable groups are as defined herein, and to their preparation and use.

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The present disclosure relates to antisense oligomers and related compositions and methods for inducing exon inclusion as a treatment for glycogen storage disease type II (GSD-II) (also known as Pompe disease, glycogenosis II, acid maltase deficiency (AMD), acid alpha-glucosidase deficiency, and lysosomal alpha-glucosidase deficiency), and more specifically relates to inducing inclusion of exon 2 and thereby restoring levels of enzymatically active acid alpha-glucosidase (GAA) protein encoded by the GAA gene.

The present disclosure relates to antisense oligomers and related compositions and methods for inducing exon inclusion as a treatment for glycogen storage disease type II (GSD-II) (also known as Pompe disease, glycogenosis II, acid maltase deficiency (AMD), acid alpha-glucosidase deficiency, and lysosomal alpha-glucosidase deficiency), and more specifically relates to inducing inclusion of exon 2 and thereby restoring levels of enzymatically active acid alpha-glucosidase (GAA) protein encoded by the GAA gene.

Morpholino oligonucleotides can be produced efficiently in a high yield by a liquid-phase synthesis method, which includes subjecting a reaction mixture of a condensation reaction to an extraction operation and separating the morpholino oligonucleotide as a resultant product to the organic layer side.

The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.