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Compositions and Methods for Treatment of Eye Diseases
Unmet NeedOxidative stress contributes to the progression of multiple eye disorders, including retinitis pigmentosa, age-related macular degeneration, and diabetic retinopathy. Ocular oxidative stress is caused by an imbalance of antioxidants and pro-oxidants in the eye, leading to elevated levels of free radicals. Free radicals contribute to the damage...
Published: 3/13/2026   |   Inventor(s): Peter Campochiaro, Andrea Cook
Keywords(s): Disease Indication, Eye Disorders, Non-novel, Predicted Novelty, Retinitis pigmentosa, Single, Therapeutic Matter, Therapeutics, Therapy Type
Category(s): Clinical and Disease Specializations > Neurology > Neurodegeneration, Clinical and Disease Specializations > Ophthalmology > Retinitis Pigmentosa, Clinical and Disease Specializations > Rare Diseases
Combinations of ASOs for the Modulation of SMN Expression in SMA Patients
Unmet NeedSpinal muscular atrophy (SMA) is a devastating neuromuscular disease that predominantly affects children and is the most common cause of hereditary infant mortality. The disease is caused by low levels of survival motor neuron (SMN) protein, due to recessive mutations in the SMN1 gene. Importantly, the copy number variation of SMN2, a homologous...
Published: 3/13/2026   |   Inventor(s): Charlotte Sumner, Constantin d'Ydewalle, Frank Rigo, C. Bennett
Keywords(s): CNS and Neurological Disorders, Combination, Disease Indication, Drug Delivery Vehicle, Muscle Atrophy, Muscular Disorders, Neurodegenerative Diseases, Peptides/Prodrugs, Single, Targeted Therapy, Therapeutic Matter, Therapeutics, Therapy Type
Category(s): Clinical and Disease Specializations > Neurology > Motor Neuron Diseases, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Therapeutic Modalities > Proteins
PSMA-targeted Nanoparticles for Therapy of Prostate Cancer
PSMA-targeting nanoparticle for prostate cancer therapyJHU Ref #: C10180Value Proposition:·        Engineered nanoparticle system with potent urea-based PSMA inhibitor·        Drug loaded nanoparticle·        Specific and selective tumor cytotoxicity Unmet NeedProstate cancer affects millions of men globally and is a leading cause of cancer death in...
Published: 3/12/2026   |   Inventor(s): Martin Pomper, Ronnie Mease, Sachin Chandran, Sangeeta Ray, Samuel Denmeade
Keywords(s): Cancers, Disease Indication, Drug Delivery Vehicle, Nanoparticles, Prostate Cancer, Single, Targeted Therapy, Therapeutic Matter, Therapeutics, Therapy Type
Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Men's Health, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Therapeutic Modalities > Targets, Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Oncology > Prostate Cancer
Mimetic peptides derived from collagen type IV for the treatment of angiogenesis-dependent diseases
C11348: Novel Anti-Angiogenic Collagen Derived Mimetic PeptidesNovelty:modified peptides from collagen IV derived parent peptide Pentastatin-1Value Proposition:The invention describes the synthesis of novel anti-angiogenic by substituting particular amino acids of the peptide Pentastatin-1 derived from collagen IV. Other advantages include:• Ability...
Published: 3/12/2026   |   Inventor(s): Niranjan Pandey, Elena Rosca, Corban Rivera, Jacob Koskimaki, Amir Tamiz, Aleksander Popel
Keywords(s): Biologics, Cancers, Disease Indication, Peptide, Protein, Single, Targeted Therapy, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type
Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Proteins, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Therapeutic Modalities
SYNGAP antisense oligonucleotide as therapy for human cognitive disorders
Unmet Need: Recent human genetic studies have suggested that mutations in the SYNGAP1 gene are linked to intellectual disability (ID), autism spectrum disorders (ASD), neurodevelopmental disorders, high rates of epilepsy, and schizophrenia. SYNGAP1 is a gene that encodes SynGAP, a GTPase-activating protein that is highly enriched in nerve cells in the...
Published: 3/13/2026   |   Inventor(s): Richard Huganir, Ingie Hong, Yoichi Araki
Keywords(s): Biologics, CNS and Neurological Disorders, Cognitive Impairment, Disease Indication, Gene Therapy, Nucleic Acid, Single, Target, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type
Category(s): Clinical and Disease Specializations > Psychiatry > Autism Spectrum Disorder, Clinical and Disease Specializations > Psychiatry > ADHD, Clinical and Disease Specializations > Psychiatry > Mood Disorders, Technology Classifications > Therapeutic Modalities > Gene Therapies
Method for using alternative splicing to control specificity of gene therapy
Unmet NeedViral-based gene therapy holds tremendous promise for the treatment of human diseases. Using genetically engineered carriers called vectors to deliver genes into cells, gene therapy can help replace a mutated gene with a healthy copy or even introduce a new gene into the body to fight a disease. However, an important concern when designing...
Published: 3/13/2026   |   Inventor(s): Jonathan Ling, Seth Blackshaw
Keywords(s): Drug Delivery Mechanism, Gene Therapy, Single, Therapeutic Matter, Therapeutics, Therapy Type
Category(s): Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Research Tools > Vectors & Plasmids
Hexosamine Analogs for Regenerative Medicine and Diseases Associated with the Musculoskeletal System
Unmet Need:Osteoarthritis (OA) is the most common form of arthritis, affecting an estimated 32.5 million adults in the United States, with an increasing incidence as the population ages (see CDC) . OA is associated with considerable morbidity as a consequence of joint damage, resulting in pain, loss of joint range of motion, and disability. The disease...
Published: 3/12/2026   |   Inventor(s): Kevin Yarema, Jennifer Elisseeff, Jeannine Coburn, Udayanath Aich
Keywords(s): Antagonists/Inhibitors, Arthritis, Biologics, Carbohydrates, Disease Indication, Drug Delivery Mechanism, Drug Delivery Vehicle, Joint Disorders, Novel, Osteoarthritis, Predicted Novelty, Single, Small Molecules, Targeted Therapy, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type
Category(s): Clinical and Disease Specializations, Technology Classifications > Therapeutic Modalities > Small Molecules, Technology Classifications > Therapeutic Modalities > Targets, Clinical and Disease Specializations > Rheumatology, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Rheumatology > Arthritis, Clinical and Disease Specializations > Rheumatology > Osteoarthritis
Bioreducible Poly(beta-amino ester)s for siRNA Delivery
C12218: Efficient Delivery Vehicle for Controlled Gene Knockdown Novelty: This technology comprises the use of bioreducible polymer based nanoparticle system that can release siRNA cargo to the cytoplasm of cells to knockdown target gene expression in tissues. Value Proposition: Gene therapy is a developing medical field and there is a need for...
Published: 3/12/2026   |   Inventor(s): Jordan Green, Stephany Tzeng, Kristen Kozielski
Keywords(s): Biologics, Brain Cancer, Cancers, Cell Therapy, Disease Indication, Drug Delivery Vehicle, Glioma, Nucleic Acid, Polymers, Single, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type
Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Neurology, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Cell Therapies, Technology Classifications > Therapeutic Modalities > Dendrimers & Polymers, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Oncology > Brain Cancer, Clinical and Disease Specializations > Oncology > Glioma
N-Hydroxysulfonamide Derivatives as New Physiologically Useful Nitroxyl Donors
Technology OverviewThe invention relates to N-hydroxysulfonamide derivatives that donate nitroxyl (HNO) under physiological conditions and are useful in treating and/or preventing the onset and/or development of diseases or conditions that are responsive to nitroxyl therapy, including heart failure and ischemia/reperfusion injury. Novel N-hydroxysulfonamide...
Published: 3/12/2026   |   Inventor(s): Andrew Cohen, Frederick Brookfield, Vincent Kalish, Stephen Courtney, Lisa Frost, John Toscano
Keywords(s): Cardiovascular Diseases, Congestive Heart Failure, Congestive Heart Failure (CHF), Disease Indication, Single, Small Molecules, Targeted Therapy, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type
Category(s): Clinical and Disease Specializations, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Therapeutic Modalities > Small Molecules, Clinical and Disease Specializations > Cardiovascular, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Cardiovascular > Congestive Heart Failure
N-Substituted Hydroxylamine Derivatives with Carbon-Based Leaving Groups as Physiologically Useful Nitroxyl (HNO) Donors
Technology OverviewThe disclosed subject matter provides certain N-substituted hydroxylamine derivative compounds, pharmaceutical compositions and kits comprising such compounds, and methods of using such compounds or pharmaceutical compositions. In particular, the disclosed subject matter provides methods of using such compounds or pharmaceutical compositions...
Published: 3/12/2026   |   Inventor(s): Daryl Guthrie, John Toscano
Keywords(s): Cardiovascular Diseases, Disease Indication, Single, Small Molecules, Targeted Therapy, Therapeutic Matter, Therapeutic Substance, Therapeutic Substance Synthesis Method, Therapeutics, Therapy Type
Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Cardiovascular, Technology Classifications > Therapeutic Modalities > Small Molecules, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Therapeutic Modalities
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