Patent classifications
A61K47/549
COMPOSITIONS AND METHODS RELATED TO TRANSFERRIN RECEPTOR-BINDING APTAMERS
Described herein are aptamers that bind to the transferrin receptor (e.g., CD71) and can be used, in part, for depleting transferrin receptor-expressing cells from a population of therapeutic cells. These aptamer compositions can be used in methods for isolating and/or enriching cells expressing CD71 or depleting cell populations of cells expressing CD71, including for example, tumor cells. Further provided are methods of using the aptamers or cell populations generated using them in the methods disclosed herein for therapies and/or drug delivery.
MODIFIED DOUBLE STRANDED OLIGONUCLEOTIDE
The disclosure is directed to dual variable domain immunoglobulin double-stranded RNA conjugates that are advantageous for inhibition of target gene expression, as well as compositions suitable for therapeutic use. The dual variable domain immunoglobulin comprises a first variable domain that binds to a binding target, and a second variable domain that comprises a reactive residue, where the linker is covalently conjugated to the reactive residue. The dsRNA is linked to the linker and is capable of inhibiting the expression of the target gene by RNA interference. The disclosure also provides pharmaceutical compositions comprising these conjugate and methods of inhibiting the expression of a target gene by administering these conjugates, e.g., for the treatment of various disease conditions.
Method of treating cancer by using siRNA nanocomplexes
The present invention is related to a method for treating cancer by using siRNA nanocomplex consisting of a nucleic acid molecule, a monocationic drug and a biocompatible polymer surfactant. The present nanocomplex can deliver an active ingredient (for example, a nucleic acid molecule and monocationic drug) into a cell/tissue of interest in a stable manner, and may be effectively applied for treating or detecting diverse disorders (practically, cancers).
COMPOSITIONS AND METHODS RELATED TO MOLECULAR CONJUGATION
The invention relates to activated Michael acceptor (AMA) compounds that can undergo conjugation with biomolecules containing Michael donor moieties, thereby providing plasma-stable antibody-drug conjugates (ADCs). Pharmaceutical compositions of the ADCs are disclosed as well. Also provided herein are a number of applications (e.g., therapeutic applications) in which the compositions are useful.
ALKYLATED NUCLEOSIDES, AND COMPOSITIONS AND METHODS THEREOF FOR NUCLEIC ACID DELIVERY
The invention provides novel compounds, compositions and formulations of liposomes, microbubbles and/or nanodroplets, and emulsions thereof, that are useful in delivery of various nucleic acids and genes (e.g., single stranded RNA, DNA, si-RNA and CRISPR constructs), as well as methods of preparation and use thereof including methods of imaging and gene delivery using ultrasound activation.
PROCESS FOR BUILDING NANOPARTICLE-BASED DRUG CARRIERS VIA PROTEIN CORONA MODULATION
The invention relates to the method for building nanoparticle-based drug carriers and the nanoparticle based drug delivery system able to manipulate the corresponding protein corona for specific and potent drug delivery to cancer cells.
Mitochondria-Targeted Polypeptide, Preparation Method thereof, and Use thereof
Disclosed are a type of mitochondria-targeted polypeptides, the preparation method and the uses thereof. The polypeptide is abbreviated as MTP. The synthesis method of the present disclosure is simple, and the mitochondria-targeted polypeptide prepared by the method can specifically target the mitochondria of cells and are basically non-toxic to cells. In addition, these synthesized polypeptides demonstrate good cell-membrane-penetrating properties, and can conveniently undergo further multi-functional derivation and modification, thereby providing a potential delivery tool for the preparation of a mitochondria-targeted medicament.
Oligonucleotide conjugation process
The present invention relates to the field of oligonucleotide conjugates and to methods of synthesis thereof. In the present method a low-water content solvent environment allows a more efficient conjugation, reducing the amount of conjugate moiety needed and increasing the conjugation reaction speed.
NEW COMPOSITION
There is provided a composition comprising a plurality of particles of a weight-, number-, or volume-, based mean diameter that is between amount 10 nm and about 700 .Math.m, which particles are made up of: (a) a solid core, which solid core preferably comprises a biologically active agent; (b) one or more discrete layers surrounding said core, said one or more layer s each comprising at least one separate coating material; and (c) a final overcoating layer of a coating material, which overcoating layer surrounds, encloses and/or encapsulates said core and said previously-applied layers of coating material, and which final layer is of a thickness that is less than said previously-applied layers. Said layers (b) and (c) are preferably applied by way of a gas phase coating technique, such as atomic layer deposition. When the cores comprise biologically active agent, the compositions may provide for the delayed or sustained release of said active agent without a burst effect.
Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof
The present disclosure relates to a compound including a nucleic acid sequence conjugated to an anti-microRNA or a microRNA-mimic or a compound including a modified anti-microRNA sequence, compositions of such a compound, and method of treatment of a disease, and method of suppressing microRNA activity by the disclosed compound or composition.