Orphan and Rare Diseases Summaries
Oiknine, Liam ; Tang, Abby ; Urano, Fumihiko
T-021494
— Published Date: 8/5/2025 Value Proposition: Disease severity scoring system that can predict the progression of Wolfram syndrome. Technology Description Researchers at Washington University in St. Louis have developed a genotype-based severity scoring system to predict the onset of cardinal sym…
Novel Composition Designed to Treat Nematode InfectionsBanas, Victoria ; Janetka, James ; Mahoney, Matthew ; Mitreva, Makedonka
T-019949
— Novel Composition Designed to Treat Nematode Infections Value proposition: Small molecule treatments designed to target the adult worm to treat nematode infections in animals. Technology Description Researchers at Washington University in St. Louis have developed novel small molecule treatment…
Novel Antiviral Treatment for Yellow Fever VirusChong, Zhenlu ; Diamond, Michael ; Fremont, Daved ; Hui, Sean
T-021452
— Published date: 1/14/2026 Value Proposition: First antiviral treatment that uses soluble receptors to treat yellow fever. Technology Description Researchers at Washington University in St. Louis have developed new soluble receptor decoy Fc-fusion protein inhibitors for the treatment of yellow f…
Antibodies that can Identify C. Acnes InfectionsAleem, Alexander ; Ilagan, Maxenia ; McCoy IV, William
T-020481
— Value Proposition: This invention uses antibodies to detect Cutibacterium acnes (C. acnes) infections. Technology Description Researchers at Washington University in St. Louis have developed antibodies that can identify Cutibacterium acnes (C. acnes), which opportunistically causes infection rang…
Neurofibromin/dopamine signaling as a biomarker for cognitive and behavioral problems in children with neurofibromatosis type 1 (NF1)Anastasaki, Corina ; Gutmann, David
T-015422
— Background: Neurofibromatosis type 1 (NF1) is a common neurological condition affecting roughly 1 in every 2,500 individuals. NF1 is associated with a host of clinical presentations, including malignant tumors and cognitive defects ranging from attention deficits, mental retardation, and autism. The…
Soluble Decoy Receptors for Treating Tick-Borne Encephalitis VirusDiamond, Michael ; Fremont, Daved ; Hui, Sean ; Li, Pengfei
T-021652
— Published date: 1/14/2026 Value Proposition: Entry receptors that bind directly to TBEV, promoting virus attachment to treat TBEV and other viruses. Technology Description Researchers at Washington University in St. Louis have developed LRP8 soluble decoy receptors (minimal domains-Fc fusion …
Pan-Receptor Decoy Protein to Treat Encephalitic AlphavirusesDiamond, Michael ; Palakurty, Sathvik ; Raju, Saravanan ; Sariol, Alan
T-021655
— Published date: 1/14/2026 Value Proposition: Soluble engineered decoy receptor protein that on its own can be used to treat multiple alphavirus types. Technology Description Researchers at Washington University in St. Louis have developed a composition of matter that consists of a mutagenized…
Gene-Specific Therapy Treatment for Predicting Limb-Girdle Muscular DystrophyHaller, Gabriel ; Li, Chengcheng ; Weihl, Conrad
T-020804
— Published Date: 5/14/2025 Value Proposition: Method that uses gene-therapy to detect limb-girdle muscular dystrophy (LGMD) type 4R/2E. Technology Description Researchers at Washington University in St. Louis have developed a method of predicting limb-girdle muscular dystrophy (LGMD) type 4R/2…
New Strategy for Treatment of Neurodegenerative Diseases with AxonopathyDiAntonio, Aaron ; Geisler, Stefanie ; Mao, Xianrong ; Milbrandt, Jeffrey ; Summers, Daniel
T-017543
— Value Proposition: This invention uses a novel gene therapy that blocks the axon degeneration typical of neurodegenerative diseases. Technology Description Researchers at Washington University in St. Louis have developed a new strategy for treatment of neurodegenerative diseases with axonopathy…
Treatments for Wolfram syndromeClark, Amy ; Mahadevan, Jana ; Urano, Fumihiko
T-015862
— Technology Description Dr. Fumihiko Urano at Washington University in St. Louis (WUSTL) and colleagues have developed therapeutic strategies to treat Wolfram syndrome. Wolfram syndrome is a rare, life-threating, genetic disorder characterized by insulin-dependent diabetes mellitus, diabetes insipi…
