Abstract: This invention relates to methods and compositions useful for treatment of subjects with dry age-related macular degeneration or geographic atrophy secondary to dry age-related macular degeneration. The methods involve administration of a pharmaceutical composition comprising an anti-C5 agent ARC1905, which comprises a C5-specific aptamer conjugated to a polyethylene glycol moiety via a linker, in an amount effective for slowing or inhibiting loss of low luminance visual acuity in the subject. The aptamer consists of the sequence fCmGfCfCGfCmGmGfUfCfUfCmAmGmGfCGfCfUmGmAmGfUfCfUmGmAmGfUfUfUAfCf CfUmGfCmG-3T, wherein fC and fU=2? fluoro nucleotides, mG and mA=2?-OMe nucleotides, all other nucleotides are 2?-OH, and 3T indicates an inverted deoxythymidine. Dosages and administration schedules are disclosed.

Abstract: Disclosed herein are non-naturally occurring vesicle comprising a chimeric vesicle localization moiety comprising a surface-and-transmembrane domain of a first vesicle localization moiety and a cytosolic domain of a second vesicle localization moiety, the method of making said vesicle and uses thereof.

Abstract: This disclosure is drawn to substituted pyridine compounds and compositions, and associated methods, useful for inhibition of SARM1 activity and/or for treating or preventing a neurological diseases.

Abstract: Provided herein are systems and methods for screening desirable biological variants using a high-throughput integrated system. The integrated system may be configured to input a plurality of parameters from functional studies of biological variants under applied conditions, in conjunction with integrated libraries of biological variants, and filter the inputs to produce desirable biological variants based on an input performance requirement. The system may output optimized strains, molecules, or novel molecules expected to have a desirable functional characteristic. Accordingly, the methods and systems disclosed herein enable multi-parametric studies of biological diversity and conditional diversity in systems biology.

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a suspension of preformed lipid nanoparticles and mRNA.

Abstract: The present invention describes a second-generation tetanus toxoid vaccine and a process for the preparation thereof, comprising the steps of: inducing an E. Coli culture OD 600=0.5 by adding 0.2 mM IPTG; growing the culture at 14-16° C. for 14 to 20 hours; suspending the culture in 25 mM phosphate buffer containing 200 mM sodium chloride; adding 1% of triton-X-100 to the phosphate buffer, and adding the buffer to the culture; sonicating the culture for a period of 3 minutes (at 5 sec on/off pulse) at 4° C. on cold beads; centrifuging the culture for 60 to 90 minutes; collecting and purifying a supernatant using Ni-NTA affinity column with an eluant; and combining the supernatant into a pool with contaminated bands and concentrating using Centriprep-30 centrifuge filters (30 kDa pores).

Abstract: Inhibitors of CDK9 that are pyrazolo[1,5-a]pyrimidine derivatives and salts thereof, corresponding to formula (I):

Abstract: The present disclosure provides non-intergeneric remodeled microbes that are able to fix atmospheric nitrogen and deliver such to plants in a targeted, efficient, and environmentally sustainable manner. The utilization of the taught microbial products will enable farmers to realize more productive and predictable crop yields without the nutrient degradation, leaching, or toxic runoff associated with traditional synthetically derived nitrogen fertilizer. The remodeled microbes taught herein are able to be combined with leading agricultural chemistry and elite germplasm. Furthermore, the disclosure provides seed treatments comprising remodeled microbes.

Abstract: Disclosed are cationic lipids which are compounds of Formula (I?). Cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

Abstract: Compositions comprising the L complex or the M complex of the botulinum neurotoxin E with its associated neurotoxin binding protein(s) (NBP), methods of making, and methods of use to treat pain and wrinkles by injecting locally. Methods of making and isolating the L and M complex comprise a multi-step process of: providing a bacterial cultured media comprising the complex of botulinum neurotoxin E and neurotoxin binding proteins; centrifuging and pelletizing the cultured media; stirring the pelletized culture at 0 to 10° C. over a period of 4 to 24 hours; centrifuging the stirred solution to obtain a supernatant; precipitating the supernatant and filtering to obtain the precipitate; and dissolving the precipitate, centrifuging and filtering to obtain a solution comprising M complex and/or L complex of botulinum neurotoxin E and its NBP; and passing the solution through a functionalized sephadex ion exchange column to isolate the complex.

Abstract: Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

Abstract: The present invention relates to methods for producing oxygenated terpenoids. Polynucleotides, derivative enzymes, and host cells for use in such methods are also provided.

Abstract: Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

Abstract: Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

Abstract: The present disclosure provides improved compositions for adoptive T cell therapies for treating, preventing, or ameliorating at least one symptom of a cancer, infectious disease, autoimmune disease, inflammatory disease, and immunodeficiency, or condition associated therewith.

Abstract: The disclosure relates to anti-cancer compounds derived from a payload which binds to poly(ADP-ribose) polymerase (PARP) and a nuclear steroid-targeting epitope (B of Formula I), which components are covalently linked via a linker, to products containing the same, as well as to methods of their use and preparation.

Abstract: This disclosure concerns an engineered polynucleotide that interacts with a pre-mRNA and a spliceosome to regulate gene expression. The engineered polynucleotide may have stem-loop structure that recruits the spliceosome and targeting sequences that are complementary to a target sequence at an exon-intron splice junction and may include nucleotides with 2? modifications and phorphorothioate linkages.

Abstract: This disclosure is drawn to substituted pyridine compounds and compositions, and associated methods, useful for inhibition of SARM1 activity and/or for treating or preventing neurological diseases.

Abstract: This disclosure features chemical entities (e.g., a compound or a pharmaceutically acceptable salt and/or hydrate and/or prodrug of the compound) that modulate (e.g., agonize) the apelin receptor (also referred to herein as the APJ receptor; gene symbol APLNR). This disclosure also features compositions containing the same as well as other methods of using and making the same. The chemical entities are useful, e.g., for treating a subject (e.g., a human) having a disease, disorder, or condition in which a decrease in APJ receptor activity (e.g., repressed or impaired APJ receptor signaling; e.g., repressed or impaired apelin-APJ receptor signaling) or downregulation of endogenous apelin contributes to the pathology and/or symptoms and/or progression of the disease, disorder, or condition.

Abstract: Provided are high-filtration reusable face masks with replaceable filters. Reusable face masks can comprise a single piece mask body configured to cover a nose and a mouth of a human face and comprising at least one filter attachment port, wherein the at least one filter attachment port comprises a filter support structure, defines an opening in the mask body, and extends from the exterior surface of the mask body; wherein the at least one filter attachment port is configured to connect to a replaceable filter and support the replaceable filter by separating portions of filter media of the replaceable filter when the replaceable filter is attached to the at least one filter attachment port.