Provided are methods or processes for producing ferulic acid from a plant material, for example, a rice bran or its derivatives. Provided are methods comprising an ion swapping and solvent extraction process followed by a chromatographic separation operations that are coupled into a process which functions to recover a fraction rich in gamma-oryzanol, thus enabling the subsequent production of a high purity ferulic acid. Provided are methods comprising an ion swapping and solvent extraction process followed by a process which functions to recover a fraction rich in gamma-oryzanol, or a mixture of ferulic acid esters of phytosterols and triterpenoids, optionally comprising cycloartenyl ferulate, 24-methylenecycloartanyl ferulate, and/or campesteryl ferulate, to enable the production of a high purity ferulic acid. Provided are methods comprising a saponification and solvent extraction process followed by recovering a fraction rich in gamma-oryzanol to enable the production of a high purity ferulic acid.

The present invention provides compositions of recombinant human lysosomal acid lipase having particular glycosylation patterns for internalization into target cells, a vector containing the nucleic acid encoding human lysosomal acid lipase, a host cell transformed with the vector, pharmaceutical compositions comprising the recombinant human lysosomal acid lipase and method of treating conditions associated with lysosomal acid lipase deficiency.

The present invention provides compositions comprising one or more active pharmaceutical ingredients, wherein the compositions are in the form of high solids concentration pastes capable of being injected in relatively low volumes into an animal using standard commercially available syringes. The invention also provides methods of making such compositions, particularly those compositions comprising high molecular weight active ingredients (e.g., antibodies, enzymes and other proteins and peptides) at relatively high therapeutic concentrations in the high solids concentration pastes. The invention further provides methods of using such formulations in treating, preventing and/or ameliorating certain diseases and physical disorders in animals, including humans, in need thereof. The invention also provides kits comprising the formulations of the invention and a suitable syringe, which in some aspects may be pre-loaded or pre-filled with a composition of the invention.

The present invention provides methods of treating LAL deficiency comprising administering to a mammal a therapeutically effective amount of lysosomal acid lipase with an effective dosage frequency. Methods of improving growth and liver function, increasing LAL tissue concentration, and increasing LAL activity in a human patient suffering from LAL deficiency are also provided.

The present invention provides compositions of recombinant human lysosomal acid lipase having particular glycosylation patterns for internalization into target cells, a vector containing the nucleic acid encoding human lysosomal acid lipase, a host cell transformed with the vector, pharmaceutical compositions comprising the recombinant human lysosomal acid lipase and method of treating conditions associated with lysosomal acid lipase deficiency.

Compositions and methods relating to potentially pathogenic mutations in the nucleotide sequence of a human LIPA gene. Some LIPA gene products have been discovered to be associated with reduced lysosomal acid lipase (LAL) activity.

Recombinant human lysosomal acid lipase (rhLAL) containing an N-terminal truncation, a composition of truncated recombinant human LAL (TLAL), an isolated mixture comprising TLAL and at least one other form of rhLAL are disclosed. A method of purifying TLAL from a mixture of LAL proteins, pharmaceutical compositions comprising TLAL and methods of producing TLAL are further disclosed.

The present disclosure provides antisense oligonucleotides, compositions, and methods that target a LIPA intron flanking exon 8, thereby modulating splicing of LIPA pre-mRNA to increase the level of LIPA mRNA molecules having exon 8, e.g., to provide a therapy for Wolman Disease or Cholesteryl Ester Storage Disease. The present disclosure provides an antisense oligonucleotide including a nucleobase sequence at least 70% complementary to a LIPA pre-mRNA target sequence in a 5′-flanking intron, a 3′-flanking intron, or a combination of exon 8 and the 5′-flanking or 3′-flanking intron.

Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient LAL protein expression or a subject having Cholesteryl Ester Storage Disease.

Detergent compositions that include a lipase variant of a parent lipase which has lipase activity and includes one or more substitutions corresponding to G23S, D27N, A40, F51I,L, E56R, D57N, V60E,K, K98I, N101D, R118, G163S, T231R, N233R, Y220F, T244E, and P256T using SEQ ID NO: 2 for numbering. Water-soluble unit dose articles including water-soluble film and a detergent composition including lipase variants. Methods of cleaning and/or treatment of surfaces using such compositions.