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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: 5/9/2024 | 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

Albumin-Flt3L Fusion Protein to Enhance Anti-Tumor Immunity and Antigen-specific T-cell Cytotoxicity

Unmet Need: Active immunotherapy, such as anti-PD-1/PD-L1 and anti-CTLA-4 checkpoint blockage, involves the administration of molecules to mobilize host immune cells to recognize and kill tumors. Proper presentation of tumor antigens through antigen presenting cells is a prerequisite to elicit a potent immune response. Current strategies enhance antigen...

Published: 5/9/2024 | Inventor(s): T.c. Wu, Chien-Fu Hung, Brandon Lam

Keywords(s): Biologics, Cancers, Discovery/Research Tools, Disease Indication, In Vitro Research Tool, Research Instrument, Target, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Technology Classifications > Research Tools > Proteins, Ligands & Receptors, Clinical and Disease Specializations > Immunology, Technology Classifications, Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Targets

Targeting PTPN22 in Cancer Therapy

Unmet NeedTumor-infiltrating lymphocytes (TILs) such as B-cells, T-cells, and natural killers are gaining traction in anti-tumor immune responses. TILs involve the use of special immune cells called T-cells, otherwise known as lymphocytes, which protect the body from viral infections, fight cancer, and coordinate the activities of other cells during...

Published: 5/9/2024 | Inventor(s): Elizabeth Jaffee, Won Jin Ho, Soren Charmsaz

Keywords(s): Cancers, Combination, Discovery/Research Tools, Disease Indication, In Vivo Research Tool, Knock Out Mouse Model, Mouse Model, Target, Targeted Therapy/Immunotherapy, Therapeutic Matter, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Therapeutic Modalities > Immunotherapies, Technology Classifications > Research Tools > Animal Models

Preventing Myocardial Loss and Sudden Death in Patients with Arrhythmogenic Cardiomyopathy via Rescuing of AIF/thioredoxin-2 Signaling

Unmet NeedAbout 400,000 to 460,000 people die each year from sudden cardiac death (SCD). Patients with Arrhythmogenic Cardiomyopathy (ACM) are at increased risk for heart failure and SCD triggered by exercise. Common ACM driver mutations occur in about 1 out of every 10,000 people. However, the physiological mechanisms that drive myocardial loss and...

Published: 5/9/2024 | Inventor(s): Stephen Chelko, Nazareno Paolocci, Gizem Keceli, Daniel Judge, Nunzianna Doti, Menotti Ruvo, Alessandra Monti, Nuria Amat, Peter Andersen

Keywords(s): Cardiac Arrest, Cardiovascular Diseases, Disease Indication, Small Molecules, Target, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Clinical and Disease Specializations > Cardiovascular > Cardiac Arrest, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Therapeutic Modalities > Small Molecules

Pharmacological Intervention of the Arachidonic Acid Pathway to Cure Amyotrophic Lateral Sclerosis

Unmet NeedAmyotrophic Lateral Sclerosis (ALS) affects as many as 30,000 people in the United States alone, with 5,000 new cases diagnosed every year. ALS is a fatal neuromuscular disease that is characterized by a progressive degeneration of the motor nerve cells and spinal cord. There are only two drugs approved by FDA for the treatment of ALS, and...

Published: 5/9/2024 | Inventor(s): Gabsang Lee, Hyungjin Eoh, Hojae Lee, Thomas Lloyd, Nicholas Maragakis

Keywords(s): Amyotrophic Lateral Sclerosis, CNS and Neurological Disorders, Disease Indication, Natural compounds, Non-novel, Predicted Novelty, Target, Therapeutic Matter, Therapeutics

Category(s): Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Therapeutic Modalities > Repurposed Drugs

Long Noncoding RNAs as Therapeutic and Diagnostic Markers for Childhood Cancer Medulloblastoma

Unmet NeedMedulloblastoma (MB) is the most common pediatric brain tumor and requires aggressive therapy in high-risk cases. MB is frequently resistant to treatment and is associated with high morbidity and mortality in affected individuals. There are four distinct molecular subgroups of MB (WNT, SHH, Group 3, and Group 4), which are characterized by...

Published: 5/9/2024 | Inventor(s): Ranjan Perera, Keisuke Katsushima

Keywords(s): Biomarker, Cancers, Clinical Diagnostics, CNS and Neurological Disorders, Disease Indication, In Vitro Diagnostics (Old), Novel, Predicted Novelty, Target, Therapeutic Matter, Therapeutics

Category(s): Clinical and Disease Specializations > Oncology > Medulloblastoma, Clinical and Disease Specializations > Rare Diseases, Technology Classifications > Diagnostics > Diagnostic Biomarkers, Technology Classifications > Therapeutic Modalities > Targets

Targeting Interleukin 17 and Senescent Cells for Treatment of the Foreign Body Response

Unmet Need13 million medical devices are implanted annually in the United States. Synthetic biomaterials are the building blocks of these implanted devices. Although biomaterials are selected for durability and minimal host immune response, all implanted biomaterials induce the foreign body response (FBR) to some degree, which creates a capsule of fibrous...

Published: 5/9/2024 | Inventor(s): Jennifer Elisseeff, Drew Pardoll, Franck Housseau, Liam Chung

Keywords(s): Antagonists/Inhibitors, Implants, Small Molecules, Target, Therapeutic Devices, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Clinical and Disease Specializations > Traumatic Injury, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Medical Devices > Prosthetics

Long-Circulating PSMA-Targeted Phototheranostic Agent

Unmet NeedProstate cancer is the leading cancer in the U.S. population and the second leading cause of cancer death in men. A promising precision cancer treatment known as photodynamic therapy (PDT) offers a way to selectively target prostate cancer cells for destruction. Prostate PDT uses a ligand to attach a light-sensitizing agent known as porphyrin...

Published: 5/9/2024 | Inventor(s): Martin Pomper, Ronnie Mease, Ying Chen, Sangeeta Ray, Juan Chen, Marta Overchuk, Gang Zheng, Kara Harmatys

Keywords(s): Cancers, Disease Indication, Imaging and Sensing Systems, In Vivo Medical Imaging, Prostate Cancer, Target, Therapeutic Matter, Therapeutics

Category(s): Clinical and Disease Specializations > Oncology > Prostate Cancer, Clinical and Disease Specializations > Men's Health, Technology Classifications > Medical Devices > Imaging, Technology Classifications > Therapeutic Modalities > Targets

Targeting TSC2 S1366 (mouse, rat) S1365 (human) by gene engineering or phosphorylation to Regulate mTOR Signaling Cascades for Therapeutic Indications

Unmet NeedThe mechanistic target of rapamycin complex-1 (mTORC1) coordinates cellular processes and regulates recycling pathways in order to control cell growth and metabolic homeostasis. Additionally, in immune cells when the T-cell receptor is activated, the mTORC1 signaling pathway is also engaged and thus plays an important role in T-cell activation,...

Published: 5/9/2024 | Inventor(s): David Kass, Kristen Kokkonen, Brittany Dunkerly-Eyring, Mark Ranek

Keywords(s): Disease Indication, Inflammatory Disorders, Target, Therapeutic Matter, Therapeutics

Category(s): Technology Classifications > Therapeutic Modalities > Targets

A Membrane-targeting Construct of NF1 GAP-related Domain (GRD) that Suppresses RAS Activity in NF1-related MPNST, NF1 and other Rasopathies

Unmet NeedNeurofibromatosis type 1 (NF1) is a common genetic disorder that affects approximately 1 in 3,000 individuals worldwide. Patients with NF1 have DNA alterations to the neurofibromin 1 gene which cause RASopathies, a family of diseases caused by altered Ras signaling. RASopathies can include both benign and malignant tumors, including malignant...

Published: 5/9/2024 | Inventor(s): Verena Staedtke, Renyuan Bai

Keywords(s): Adeno-Associated Virus, Biologics, Cancers, Discovery/Research Tools, Disease Indication, Nucleic Acid, Research Reagent, Target, Therapeutic Matter, Therapeutic Substance, Therapeutics, Translational Research Biomarker, Viruses

Category(s): Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Research Tools > Vectors & Plasmids

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