Direct CSF infusion helps engineered T cells bypass CNS access limits, improving tumor targeting, persistence, and toxicity control.
Convection-enhanced striatal rAAV infusion expands vector spread into the cortex, improving gene expression for neurologic disorders.
AAV9-delivered phosphomimetic Hsp22 improves learning, memory, and synaptic plasticity in tauopathy without changing tau levels.
PKR-associated compounds disrupt the PKR/PI4K2A network and lysosomal function to overcome therapy resistance in cancer cells.
A multimodal diazepam nasal spray offsets food effects to improve dosing accuracy, rapid absorption, and seizure rescue efficacy.
Specific salivary miRNAs enable earlier, more sensitive mild TBI assessment than clinical scores or imaging alone.
Structural changes to HNK derivatives extend efficacy beyond one week while sharply lowering addiction risk for depression and pain treatment.
Targeting TDRP with antibodies helps separate autoimmune treatment benefits from anxiety-related harm while enabling mental disorder monitoring.
Selective CTPS1 inhibitor scaffolds curb T- and B-cell proliferation while sparing CTPS2-linked activity to reduce toxicity in proliferative disease treatment.
FAAH- and esterase-activated thyromimetics boost brain exposure for remyelination while limiting cardiotoxicity and bone demineralization.
Truncated Ac-TMP-2 hookworm proteins retain anti-inflammatory activity, offering broader inflammation treatment with simpler targeted protein therapy.
Controlled-release MAOI dosing over 6-16 hours lowers peak concentrations, reducing cardiovascular side effects and tyramine interactions.
Small-molecule TRPC5 modulators reduce proteinuria and kidney disease risk while avoiding the high relapse and side effects of existing treatments.
Ultra-low THC with melatonin and curcumin targets amyloid, tau, and mitochondrial dysfunction while reducing CNS treatment side effects.
Codon-optimized UBE3A expression cassettes use viral delivery to restore therapeutic protein levels and rescue Angelman Syndrome phenotypes.
Crystalline pyrazolopyrimidine carboxamide polymorphs offer an oral treatment route for Gaucher and Parkinson's disease with lower cost and fewer side effects.
Small molecules modulate ATXN3 pre-mRNA splicing to lower full-length ataxin-3 and potentially slow Spinocerebellar Ataxia 3 progression.
Controlled acetonitrile/water crystallization forms APC hydrochloride hemihydrate with lower 2-chloropropane contamination and better stability.
Leptin receptor agonists such as metreleptin improve depressive symptoms without relying on inconsistent baseline leptin measurements.
Chemically modified RNAi duplexes selectively lower tau RNA and protein while limiting off-target toxicity in tauopathy treatment.
PP2A-modulating 3-diarylmethylenes drive FOXO nuclear translocation, suppress oncogenic kinases, and help restore chemotherapy sensitivity.
A VEGF-mimicking peptide binds Aβ oligomers, blocks toxic VEGF interaction, and restores synaptic function in Alzheimer's models.
Combining h2E2 antibody with a dopamine 1 receptor antagonist reduces cocaine brain effects, drug-seeking behavior, and relapse risk.
pH-tuned C1s-binding molecules improve FcRn interaction and target neutralization, lowering dose needs for complement-mediated disorders.
Sequential antibody dosing followed by daratumumab directly targets amyloidogenic proteins in Stage IV AL amyloidosis to improve survival and cardiac outcomes.
Protein and genetic biomarker detection in blood or other samples enables earlier, less invasive neurodegenerative disease diagnosis and monitoring.
Radiolabeled brain-penetrant HDAC6 compounds improve isoform selectivity, reduce side effects, and enable PET imaging in the brain.
Sub-anesthetic ketamine dosing targets NMDA receptors to rapidly relieve PTSD symptoms with minimal side effects.
Selective IP6K1 inhibitors raise AKT phosphorylation to improve insulin sensitivity while reducing renal toxicity and supporting mitochondrial function.
Specific lithium-magnesium-potassium ratios curb inflammation by restoring ion homeostasis and reducing lithium toxicity.
Combining nicotinic acid with TTA supports mitochondrial function, reduces inflammation, and strengthens liver-brain energy flux.
Direct apoE4 binding by tramiprosate disrupts the Arg61-Glu255 salt bridge to induce an apoE3-like conformation with fewer off-target effects.
Agonistic anti-VISTA antibodies suppress T cell proliferation and proinflammatory cytokines for autoimmune, inflammatory, and transplant-related conditions.
Phosphosulindac targets central sensitization by reducing CNS neuronal signaling, helping relieve neuropathic pain where NSAIDs fall short.
Vitamin E TPGS masks bitterness in oral atomoxetine and other bitter drugs, improving compliance with formulations stable for at least 45 days.
PTB-targeted CRISPR/Cas delivery in glial cells enables in vivo neuron conversion in retina and striatum to help restore visual and motor function.
Intravenous albumin addresses persistent cognitive impairment in cirrhosis by reducing inflammation and endothelial dysfunction.
Novel TRPM3 antagonist derivatives improve potency while reducing side-effects for treating pain, epilepsy, and inflammatory hypersensitivity.
Cis- and trans-resveratrol oppositely modulate EndoG to control paternal mitochondrial elimination and treat related disease states.
Small molecules directly modulate clock proteins to shift circadian phase and period, improving treatment consistency beyond light or melatonin.
Novel KOR ligands selectively modulate dynorphin-kappa signaling to reduce dysphoria, stress-induced relapse, and neuroplasticity disruption.
Novel boric acid proteasome inhibitors use targeted substituent changes to preserve inhibition while improving oral bioavailability.
Targeted hinge-region cysteine changes in recombinant IgG2 eliminate disulfide heterogeneity, yielding stable antibodies with consistent properties.
TIM receptor-modulating compounds in lipid particles reduce therapeutic immunogenicity and promote self-tolerance for autoimmune treatment.
Engineered neural stem cells deliver VEGF to the SVZ to restore cell migration, reduce inflammation, and improve function in neurodegeneration.
Tolcapone is repurposed to cross the blood-brain barrier, inhibit AKR1B1, reduce oxidative stress, and promote neuronal maturation in Rett syndrome.
Sigma-1 receptor targeting with pridopidine and analogs helps reduce motor impairment and slow functional decline in Huntington's disease.
By targeting the GRK2 RGS domain instead of kinase activity, these compounds restore receptor signaling and improve responsiveness.
Defined decanoic and octanoic acid ratios support mitochondrial energy and seizure reduction without strict ketogenic diet management.
A pH-tuned AXN-001 injectable composition enables rapid plasma exposure for acute migraine while improving solubility, tolerability, and recurrence control.