A61K47/554

POLYPEPTIDE, AND PREPARATION METHOD THEREFOR AND USE THEREOF
20220409694 · 2022-12-29 ·

Provided is a polypeptide containing the amino acid sequence as shown in SEQ ID NO. 1, and the present invention belongs to the field of biomedicine. The polypeptide can inhibit the infection with novel coronavirus 2019-nCoV (SARS-CoV-2) and SARS-like viruses, and can thus provide good candidate drugs for the prevention and treatment of 2019-nCoV and SARS-like viruses which may break out in the future. Further provided is a polypeptide derivative having palmic acid or cholesterol modifications.

RNAi therapy for hepatitis B virus infection

Described are compositions and methods for inhibition of Hepatitis B virus gene expression. RNA interference (RNAi) triggers and RNAi trigger conjugates for inhibiting the expression of Hepatitis B virus gene are described. Pharmaceutical compositions comprising one or more HBV RNAi triggers optionally with one or more additional therapeutics are also described. Delivery of the described HBV RNAi triggers to infected liver in vivo provides for inhibition of HBV gene expression and treatment.

Therapeutic compositions

This application relates to therapeutic siRNA agents and methods of making and using the agents.

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

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.

LIPID COMPOSITIONS COMPRISING PEPTIDE-LIPID CONJUGATES

A lipid composition containing a nucleic acid, wherein the lipid composition comprises a peptide-lipid conjugate, is provided. The peptide of the peptide-lipid conjugates can be from 4 to 52 amino acids in length. Methods of using the lipid composition in the in vivo delivery of nucleic acids are further provided.

SYNERGISTIC IMMUNOSTIMULATION THROUGH THE DUAL ACTIVATION OF TLR3/9 WITH SPHERICAL NUCLEIC ACIDS

The present disclosure is generally directed to spherical nucleic acids (SNAs) comprising multiple TLR agonists that enable the simultaneous activation of multiple TLR pathways for maximally synergistic immune activation. In some aspects, the present disclosure provides a spherical nucleic acid (SNA) comprising: (a) a nanoparticle core; (b) one or more toll-like receptor 3 (TLR3) agonists encapsulated in the nanoparticle core; and (c) a shell of oligonucleotides attached to the external surface of the nanoparticle core, the shell of oligonucleotides comprising one or more toll-like receptor 9 (TLR9) agonist oligonucleotides. Methods of using the SNAs are also provided herein.

Oligomers and oligomer conjugates

The present invention relates to an oligomer conjugate for use in the treatment of a viral disorder. The oligomer conjugate comprises: a) an oligomer capable of modulating a target sequence in HBx and/or HBsAg of Hepatitis B Virus (HBV) to treat said viral disorder; and b) a carrier component capable of delivering the oligomer to the liver which is linked, preferably conjugated, to the oligomer.

Amphiphilic oligodeoxynucleotide conjugates as adjuvant enhancers
11504425 · 2022-11-22 · ·

Amphiphilic oligonucleotide conjugates that enhance adjuvant function are disclosed. The conjugates typically include: a lipophilic component, and conjugated thereto (directly or indirectly) an immunomodulating oligonucleotide that, if it were not conjugated to the lipophilic component, would suppress TLR7 and/or TLR8 stimulation. In the presence of albumin, these conjugates significantly enhance adjuvant function, in particular the function of TLR7/8-mediated adjuvants such as an imidazoquinolinamine. The conjugates can be administered, along with an adjuvant compound, to a subject in order to cause and/or enhance an immune response (for instance, to an infectious agent or a cancer antigen) in the subject.

RNA interference in ocular indications

The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.

Methods to induce targeted protein degradation through bifunctional molecules

The present application provides bifunctional compounds which act as protein degradation inducing moieties. The present application also relates to methods for the targeted degradation of endogenous proteins through the use of the bifunctional compounds that link a cereblon-binding moiety to a ligand that is capable of binding to the targeted protein which can be utilized in the treatment of proliferative disorders. The present application also provides methods for making compounds of the application and intermediates thereof.