This disclosure demonstrated that cyanobacteria bioengineered with cyanobacterial lipid-biosynthetic-promoting genes could produce large quantities of SDA, as well as rarely observed ETA. Importantly, the biosynthesized omega-3 fatty acids, such as SDA and ETA, are found conjugated to more bioavailable glycolipids, including MGDG and DGDG. Novel compositions include MGDG, DGDG, SQDG, and PG molecular species that contain the following n-3 PUFAs and n-3 LC-PUFAs at both sn-1 and sn-2 positions: 18:3/18:4, 18:3/20:4, 18:3/20:5, 18:4/18:4, 18:4/20:4, 18:4/20:5. These compositions are not found in nature and result from the engineering of cyanobacteria; and can serve as highly bioavailable, cost-effective anti-inflammatory compounds. These compositions, therefore, have strong anti-inflammatory properties with the likely capacity to block activities of cyclooxygenases, lipoxygenases, and cytochrome P450s that metabolize PUFAs and LC-PUFAs to pro-inflammatory mediators. These compounds also inhibit the uptake of pro-inflammatory PUFAs and LC-PUFAs into cells and especially inflammatory cells.

Aspects of the disclosure relate to complexes and other aspects relate to formulations (e.g., aqueous, lyophilized forms) comprising such complexes comprising an oligonucleotide (e.g., useful for targeting DMPK) covalently linked to an antibody (e.g., anti-TfR1 antibody).

Provided herein are conjugated oligonucleotides that are characterized by efficient and specific tissue distribution.

Formulated and/or co-formulated nanocarriers (e.g., LNPs and/or SLNPs) comprising PD-1 Prodrugs and methods of making the nanocarriers are disclosed herein. The PD-1 Prodrug compositions comprise a drug moiety, a lipid moiety, and linkage unit that inhibit PD-1-L1/L2. The PD-1 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.

The present disclosure relates to a pharmaceutical composition for treating, preventing and/or ameliorating spinal muscular atrophy (SMA) and a method of administration thereof. A pharmaceutical composition for treating, preventing and/or ameliorating SMA according to an embodiment of the present disclosure may comprise a SMN protein, which is the cause of the onset of SMA. The pharmaceutical composition may be administered directly to a subject suffering from SMA via intracerebroventricular, intrathecal or intra-cisterna magna administration, thereby treating or preventing SMA or ameliorating symptoms of SMA. The pharmaceutical composition for treating, preventing or ameliorating SMA according to one embodiment of the present disclosure is safe, cost-effective and/or accessible compared to other previously approved drugs and can achieve substantially equivalent therapeutic effects.

Provided herein are, inter alia, nucleic acid conjugates including a non-cell penetrating ribonucleic acid compound attached at its 3 end to a phosphorothioate polymer. Attachment of the phosphorothioate polymer to the non-cell penetrating ribonucleic acid conveys stability to and allows for efficient intracellular delivery of the non-cell penetrating ribonucleic acid. The nucleic acid conjugates provided herein including embodiments thereof are useful, inter alia, for the treatment of cancer, inflammatory disease, and pain.

Biodegradable Periodic Mesoporous Organosilica (BPMO) nanomaterials and methods of making BPMOs loaded with Neutron Capture Agents are disclosed herein. Consequently, the BPMOs loaded with Neutron Capture Agents provide a method of treating cancer, immunological disorders and other disease by utilizing a Neutron Capture Therapy modality.

The present invention relates to EP4 agonist-bisphosphonate conjugates or related compounds and uses thereof. Said conjugates or related compounds may be used to provide delivery of an EP4 agonist or related compound to a desired site of action, such as a bone. Bisphosphonate moieties, linked to the EP4 agonists via amide linkers, have been implicated in the inhibition of bone resorption and bone targeting.

The present invention aims to provide a vaccine pharmaceutical composition universally usable for induction of humoral immunity against various antigens and exerting a high antibody production inducing effect. The present invention relates to a vaccine pharmaceutical composition for inducing humoral immunity, including: an antigen; and an immunity induction promoter that is a bisphosphonate.

The described invention provides a compound comprising an organometallic complex comprising a core of ruthenium and sphingomyelin, with the option of added zinc, that, in a therapeutic amount, through further connections with contributing organic ligands, are effective as chemotherapeutic agents that have an anti-tumor or anti-cancer effect on a tumor comprising tumor cells including a cytotoxic effect on of the tumor cells, inhibition of the growth of the tumor comprising the tumor cells, inhibition of migration of the tumor cells, or any combination of these effects.