AAV9 delivers an SRSF1 antagonist to neuronal cells, aiming to limit neurotoxicity while protecting against degeneration in sporadic ALS.
Bifunctional PROTACs recruit SMARCA2 to VHL for ubiquitination and degradation, addressing hard-to-bind cancer targets.
Selective benzyltryptamines target 5-HT2A while avoiding 5-HT2B activation, preserving CNS efficacy with lower cardiac risk.
Selective activation of TRPA1- or CGRP-linked vagal and brainstem neurons shifts cytokines to control immune responses in disease.
Targets PI3K-AKT-mTOR somatic mutations with mTOR inhibitors to reduce seizures and abnormal neuron generation in intractable epilepsy.
Specific excipient combinations lower viscosity in concentrated nucleic acid formulations, easing injection and processing while preserving stability.
Pressing cannabis biomass with edible oil enables solvent-free extraction, then carbon black purification removes contaminants while preserving cannabinoids and terpenes.
Novel N-desmethyl ruboxistaurin L-lactate polymorphs improve solubility and stability while reducing CYP3A4 metabolism and QT risk.
Direct ERK targeting with azaindole derivatives helps suppress tumor growth while avoiding the ERK reactivation that drives RAF and MEK inhibitor resistance.
Machine learning adapts opioid dose reduction to withdrawal, anxiety, and side effects, improving tapering support with personalized glide paths.
Ionizable stealth LNPs extend circulation while targeting T, B, and NK cells for safer, more efficient nucleic acid delivery.
Compounds recruit ubiquitination and proteasomal degradation to remove resistant EGFR mutants such as T790M, L858R, and C797S.
Adding hydroxycitric acid during T cell culture improves persistence and antitumor activity while reducing apoptosis for cancer therapy.
Vision sensors and AI quantify outdoor ad exposure, viewing duration, and attention in real time to improve campaign measurement and targeting.
Combining Seralutinib with Sotatercept targets multiple PAH pathways to better reduce pulmonary pressure and right ventricular hypertrophy.
Deuterated gamma-carbolines combine 5-HT2A antagonism and mu-opioid partial agonism to extend OCD relief with fewer side effects.
A topical Udonitrectag gel or emulgel improves microcirculation and wound healing in diabetic ulcers where conventional care often fails.
Iodide shifts the iodine equilibrium during gamma sterilization, keeping dilute povidone-iodine sterile, effective, and ready to use.
Heterobifunctional CyTaCs bridge CCR2-expressing cells and exogenous antibodies to boost ADCC while improving selectivity, stability, and manufacturability.
Fine gepotidacin granules disperse in water within 3 minutes to improve swallowability, palatability, and dose uniformity.
Periodic INX-315 dosing and amorphous spray-dried dispersion lower Cmax/Cmin spikes while sustaining anti-cancer exposure and limiting side effects.
A 10-amino camptothecin scaffold improves solubility and stability while preserving cytotoxicity for low-immunogenic immunoconjugates.
Novel glutamatergic compounds aim to deliver antidepressant effects within hours while reducing the side effects seen with ketamine.
High-aspect ratio paclitaxel crystals anchored in a porous balloon substrate improve coating robustness and make local vascular drug release more consistent.
ITIH1 suppresses HCC by competitively binding ITGB1/ITGA5 and inhibiting integrin-FAK signaling, supporting new drug development.
A tetrahydrocurcumin and silymarin blend improves liver metabolism and function while reducing enzyme markers, inflammation, and apoptosis.
Topical copper agents boost lysyl oxidase to strengthen corneal collagen and stabilize keratoconus without invasive CXL.
Blocking Galectin-9 signaling with antibodies boosts T cell and cytokine responses, helping treat cancers resistant to PD-1 or CTLA-4 therapy.
A lecithin and guar gum oral powder lets ibuprofen be taken dry or with a little water, improving taste, mouthfeel, and adherence.
Controlled rapamycin dosing activates DARIC signaling while limiting CAR T cytokine toxicity, neurotoxicity, and T cell exhaustion.
A microneedle delivers drug and hydrogel push formulations into the suprachoroidal space to localize ocular therapy and limit off-target spread.
Spray-dried amorphous dispersions and solubilized formulations raise Compound (I) bioavailability while preserving stability for oral dosing.
Pyridazine and phthalazine derivatives inhibit NLRP3 while improving selectivity, supporting treatment of inflammatory and degenerative diseases.
HPBCD extracts membrane cholesterol to improve sickle cell red blood cell deformability while reducing hemolysis risk.
Defined 50-800 mg daily dosing keeps the active metabolite above therapeutic plasma levels to improve inflammatory bowel disease treatment.
Azabicycle derivatives target autotaxin binding regions to deliver potent inhibition for fibrotic, inflammatory, autoimmune, and cancer-related diseases.
Isolating and purifying CBD-C1 solves cannabis extract variability while preserving anticonvulsant activity for consistent epilepsy dosing.
S1P1 modulators address lupus treatment limits by reducing inflammation, improving kidney function, and lowering side-effect burden.
Oral fluorescent challenge molecules enable rapid, non-invasive gut barrier assessment without phlebotomy or specialized lab equipment.
Small-molecule sulfonamide derivatives block IgE-FcεRI binding to inhibit mast cell degranulation and histamine release in allergic disease.
A self-microemulsifying oral formulation uses surfactants and co-solvents to solubilize a hydrophobic ALK inhibitor while preserving stability.
A membrane-localized insulin receptor fragment activates signaling without repeated insulin dosing, helping avoid hypoglycemia in neurological treatment.
RISC-cleaving dsRNA lowers Serpinc1 so hemophilia bleeds can be managed with reduced factor or bypassing-agent doses.
Direct NLRP3 inhibition with 5-FUMCL reduces IL-1β, IL-6, and IL-18 while limiting off-target effects in inflammatory disease models.
A novel GalNAc-linked nucleic acid conjugate improves liver-cell uptake, in vivo stability, and gene silencing with low toxicity.
Low-molecular-weight cyclic phosphatidic acid derivatives offer a safe, cost-effective approach to slow dementia progression and ease symptoms.
A defined crystal form improves thermal stability, compressibility, and static control for pharmaceutical preparation and long-term storage.
Amorphous ramelteon dispersed in a sublingual film bypasses first-pass loss, dissolves within 1 minute, and improves bioavailability.
Non-covalent purine-pyrimidine dimer cations reduce oxidative stress and promote cell proliferation to improve repair and wound healing.
IN10018 reduces tumor fibrosis so monoclonal antibodies and antibody conjugates can penetrate stroma and reach higher tumor concentrations.