Patent classifications
A61K47/54
BIFUNCTIONAL BRIDGING COMPOSITIONS FOR VIRAL TRANSDUCTION
This disclosure provides compositions and methods for delivering a viral composition to cells, e.g., for cell surface receptor-mediated uptake, and enhanced viral transduction. Viral transduction can be achieved via a bifunctional bridging composition that includes a moiety that binds to a cell surface receptor ligand and a linked bridging moiety that binds to a viral composition. Also provided are modified viral compositions comprising a bridging composition specifically bound via its bridging moiety to the viral composition. Modified viral compositions and methods for reducing levels or titers of neutralizing antibodies in a subject in need of viral therapy, e.g., gene therapy, are provided. In some embodiments, the modified viral composition includes empty viral particles that bind and internalize neutralizing autoantibodies. Modified viral compositions including empty viral particles can be administered prior to viral therapy. Also provided are pharmaceutical compositions and kits including a bifunctional bridging composition and/or modified viral compositions.
NOVEL BIS (HYDROXY BENZYLIDENE) CYCLIC KETONE BASED TETRA-AZA CORAND
A tetra-aza corand compound of formula (Ia) and compound of formula (Ib) and salts thereof. The tetra-aza corand of formula (Ia) and (Ib) of the present invention relates to novel corand entity having a substantially enclosed volume and a framework structure, the compounds are designed as therapeutic carriers for molecule therapeutics delivery and pharmaceutical compositions thereof.
IONIZABLE LIPIDS AND COMPOSITIONS FOR NUCLEIC ACID DELIVERY
The present disclosure relates to a compound of Formula (I)
##STR00001##
or a pharmaceutically acceptable salt thereof, which can be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid.
Formulated and/or Co-Formulated Liposome Compositions Containing Immunogenic Cell Death (ICD) Inducing Prodrugs Useful In The Treatment of Cancer and Methods Thereof
Formulated and/or co-formulated nanocarriers (e.g., LNPs and/or SLNPs) comprising ICD Prodrugs and methods of making the nanocarriers are disclosed herein. The ICD prodrug compositions comprise a drug moiety, a lipid moiety, and linkage unit that induce immunogenic cell death (ICD). The ICD Prodrugs can be formulated and/or co-formulated into a nanocarrier to provide a method of treating cancer, immunological disorders, and other disease by utilizing a targeted drug delivery vehicle.
Short chain ceramide-based lipids and uses thereof
Provided herein, in some aspects, are delivery vehicles comprising a glycosphingolipid and an agent to be delivered attached to the glycosphingolipid. In some embodiments, the glycosphingolipid comprises an oligosaccharide and a short chain (e.g., C0-C3) ceramide. In some embodiments, the agent to be delivered is a therapeutic agent. The glycosphingolipid is able to deliver the agent to a cell or to a cellular compartment, as well as across the musical barrier. In some embodiments, agents delivered using the glycosphingolipid described herein exhibit longer half-life, compared to agents delivered alone. Methods of delivering a therapeutic agent to a subject for treating a disease using the glycosphingolipid delivery vehicle are also provided.
Short chain ceramide-based lipids and uses thereof
Provided herein, in some aspects, are delivery vehicles comprising a glycosphingolipid and an agent to be delivered attached to the glycosphingolipid. In some embodiments, the glycosphingolipid comprises an oligosaccharide and a short chain (e.g., C0-C3) ceramide. In some embodiments, the agent to be delivered is a therapeutic agent. The glycosphingolipid is able to deliver the agent to a cell or to a cellular compartment, as well as across the musical barrier. In some embodiments, agents delivered using the glycosphingolipid described herein exhibit longer half-life, compared to agents delivered alone. Methods of delivering a therapeutic agent to a subject for treating a disease using the glycosphingolipid delivery vehicle are also provided.
Targeting mitochondrial fission through mDIVI-1 derivatives
Derivatives of mDIVI-1 may be used to target and eliminate cancer stem cells. Disruption in the mitochondrial dynamics balance plays a role in cancer. Proteins involved in regulating mitochondrial dynamics represent potential targets for cancer treatment. Mitochondrial fission protein DRP1 is such a target. Derivatives of mDIVI-1 inhibit DRP1, and have demonstrated inhibition of tumorsphere forming capacity, migration and stemness-related signaling in breast cancer cells. These properties result from induction of mitochondrial oxidative stress and reduction of mitochondrial metabolism in the target cancer cells. The potency of an mDIVI-1 derivative may be dramatically increased through addition of at least one membrane-targeting signal and/or a mitochondria-targeting signal.
Targeting mitochondrial fission through mDIVI-1 derivatives
Derivatives of mDIVI-1 may be used to target and eliminate cancer stem cells. Disruption in the mitochondrial dynamics balance plays a role in cancer. Proteins involved in regulating mitochondrial dynamics represent potential targets for cancer treatment. Mitochondrial fission protein DRP1 is such a target. Derivatives of mDIVI-1 inhibit DRP1, and have demonstrated inhibition of tumorsphere forming capacity, migration and stemness-related signaling in breast cancer cells. These properties result from induction of mitochondrial oxidative stress and reduction of mitochondrial metabolism in the target cancer cells. The potency of an mDIVI-1 derivative may be dramatically increased through addition of at least one membrane-targeting signal and/or a mitochondria-targeting signal.
Hydrogel and method for preparing the same
The present application provides a method for preparing a hydrogel and the obtained hydrogel. The method including: forming a first part by mixing a first single-stranded nucleotide with a first liposome, and forming a second part by mixing a second single-stranded nucleotide with a second liposome, wherein the first single-stranded nucleotide and the second single-stranded nucleotide have complementary sticky ends; forming a hydrogel by mixing the first part and the second part.
Benzonorbornadiene derivatives and reactions thereof
A bioorthogonal molecule can include a molecule having a structure according the above wherein R.sup.1-R.sup.8 are independently selected from H, a substituted or unsubstituted C.sub.1-C.sub.4 alkyl or alkylene group, COOH, COOR.sup.9, COR.sup.9, CONR.sup.9R.sup.10, CN, CF.sub.3, and SO.sub.2R.sup.9, and where R.sup.9 and R.sup.10 are independently selected from H and a substituted or unsubstituted C.sub.1-C.sub.4 alkyl or alkylene group, with the proviso that one of R.sup.3-R.sup.8 comprises a leaving group, and wherein X is O, S, N, SO, SO.sub.2, SR.sup.+, Se, PO.sub.2.sup.−, or NRR′.sup.+, and where R and R′ are independently selected from H or a substituted or unsubstituted C.sub.1-C.sub.4 alkyl or alkylene group. ##STR00001##