Cone-specific promoters and WPRE target depolarizing optogenetic proteins to human cones, restoring signals to ganglion cells.
This case uses cyclic peptide derivatives and pharmaceutical carriers to reduce retinal thickness, neovascularization, and vision loss.
QX008N targets TSLP directly to neutralize inflammatory signaling across multiple diseases.
A recombinant complement regulator combines selected CCP domains to improve serum stability, dimerization, and therapeutic duration.
A staged floating-culture protocol uses BMP and Wnt pathway control to improve retinal progenitor production and reduce contamination risk.
This case addresses limited LPAR1 treatments through triazole compounds engineered for selectivity, low toxicity, and metabolic stability.
This case uses ligand-specific gold clusters to inhibit Aβ aggregation, reduce RGC apoptosis, and protect optic function in glaucoma.
This case uses heteroaryl butanoic acid derivatives to inhibit LTA4H, elevate lipoxin A4, and address chronic inflammation.
CR2-FH fusion vectors combine retinal targeting with Factor H activity to inhibit alternative complement activation in eye disease.
A PLL-PEG copolymer anchors to biological surfaces, improves wettability, and limits protein and cellular adhesion.
This case uses oil, aqueous dispersion, and polysorbate 80 to deliver agents to the dermis while limiting systemic effects.
This case uses suspension embryoid bodies and staged WNT/SMAD inhibition to produce pigmented RPE cells at scalable volume.
This case combines NSAIDs with solid and liquid lipids to sustain relief at inflamed tissues while limiting systemic side effects.
A reservoir chamber stores vitreous humor for repeated marker analysis while porous diffusion supports ocular therapy.
Site-directed PH20 modifications at position 324 improve resistance to heat and preservatives while retaining hyaluronidase activity.
Structural changes preserve CYP activity while improving metabolic stability and bioavailability for inflammatory and neovascular disorders.
Anionic or cationic micelles use protecting agents to stabilize sparingly soluble ingredients for sterile eye and nasal drops.
This case uses high-affinity TL1A antibodies to selectively inhibit an inflammatory pathway implicated in IBD and limit surgery needs.
AAV-delivered RPF2 uses small-molecule-responsive destabilization domains to limit inflammation while preserving retinal function.
After three monthly doses, disease-activity assessments guide 8- or 12-week VEGF antagonist intervals for macular degeneration.
This case combines codon-optimized CYP4V2 with CAG, WPRE, and SV40 polyA elements to improve rAAV expression for BCD.
This case uses ADPR as a cytoprotective component alongside antivirals to preserve activity while mitigating drug-related cell toxicity.
This case uses humanized and chimeric CB1 antibodies to preserve receptor modulation while limiting brain penetration and psychiatric risks.
This case uses local nasal receptor activation to boost tears and improve ocular surface health without crossing the blood-brain barrier.
A detachable guide positions the expandable reservoir in the lacrimal sac while hydrogel porosity regulates extended drug delivery.
This case addresses costly, complex genome editing with programmable guide RNAs and optimized Cas orthologs for multi-site targeting.
This case uses hexylene glycol in roflumilast formulations to maintain particle size and reliable bioavailability during storage.
CRISPR targets mutant and wild-type alleles, then adds a codon-modified gene to restore function without renewed targeting.
A single lacrimal gland AAV2/9 injection sustains NGF in tears to support corneal reinnervation after abrasion.
This case uses NCS1 agonists to stabilize NCS1 and increase mitochondrial respiratory chain activity in Wolfram Syndrome.
Transferrin receptor targeting helps BDNF cross the blood-brain barrier.
This case uses codon-optimized GJB2 and regulatory elements in rAAV vectors to express protein and support cochlear homeostasis.
IL-4 receptor targeting delivers Bcl-2 activity to inhibit apoptosis.
This case uses SEQ ID aptamers to bind GPCR autoantibodies, inhibit receptor interactions, and support therapy or diagnosis.
Compact IL-17A-binding fusion proteins use albumin binding to extend half-life while supporting soluble, non-Fc therapeutic formats.
This case shows how a SPINK2 mutant peptide targets active MMP-9, avoiding non-selective inhibition and enabling detection.
This case explores phenoxy carboxylic acid compounds that regulate glucose and lipids while reducing liver fat and weight gain.
This case replaces injection-dependent antibody therapy with tyrosine hydroxylase inhibitors delivered through oral, nasal, or other routes.
This case uses TGFβ-binding antibodies to reduce bone resorption and promote bone deposition in osteogenesis imperfecta.
A porous release mechanism and expandable reservoir sustain ocular drug levels while reducing bolus peaks and repeated injections.
Teprotumumab-based IGF-IR inhibition addresses prior-treatment failure, reducing proptosis and clinical activity in thyroid eye disease.
Computationally reconstructed AAV capsids aim to reduce human serum neutralization while preserving gene transfer capability.
This case shows how C-17 oleanolic acid modifications combine antioxidant activity with iNOS and COX-2 modulation.
This case uses nuclease-stable, self-delivering oligonucleotides to correct mutations without exogenous proteins or nucleic acids.
This case uses modular antibody variants and optimized variable regions to reduce cell-surface Sortilin and raise Progranulin levels.
This case shows how RARγ-selective targeting inhibits collagen atrophy and preserves photoreceptor function in retinal tissue.
A charged polymer core and biodegradable shell extend ocular drug release to six months, reducing frequent injections and side effects.
This case uses selective RIPK1 inhibitor compounds to curb inflammatory signaling while limiting off-target effects.
This case uses overlapping OTOF fragments, promoters, and splice sites to support recombination and hearing rescue in deficient mice.
This case uses topical isoxazoline formulations to target Demodex mites on the eye while limiting systemic side effects.