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Antisense Oligonucleotides to Prevent Genetic Mis-Splicing for ALS/FTD Therapeutics
SHORT DESCRIPTION
Antisense oligonucleotides (ASOs) that correct TDP‑43–dependent mis‑splicing of KCNQ2 thereby reducing neuronal hyperexcitability in ALS/FTD and related TDP‑43 proteinopathies.
INVENTORS
Evangelos Kiskinis*
Northwestern University Feinberg School of Medicine, Department of Neurology
Jonathan...
Published: 5/12/2026
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Updated: 5/7/2026
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Inventor(s):
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, FTD - Frontotemporal Dementia, Neurodegenerative disease, Neurologic disease, Neurology, Nucleic Acids, Rare diseases, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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STING Agonist 8803 for Blood-Brain Barrier Opening in Glioblastoma Treatment
SHORT DESCRIPTION
Chemical compound that opens the blood-brain barrier to enhance delivery of therapeutics for treating glioblastoma.
INVENTORS
Amy Heimberger*
Northwestern University Feinberg School of Medicine, Department of Neurological Surgery
* Principal Investigator
NU Tech ID: NU 2025-212
IP...
Published: 5/7/2026
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Updated: 4/27/2026
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Inventor(s):
Keywords(s): AD - Alzheimer’s Disease, Adjunct therapy, ALS - Amyotrophic Lateral Sclerosis, Animal Model, Autoimmune disease, Bladder cancer, Brain cancer, Breast cancer, Cancer Treatment, Cancer/Oncology, Cervical cancer, CNS - Central Nervous System, Colon cancer, Drug delivery, Endometrial cancer, Gastric cancer, GBM - Glioblastoma, Head and neck cancer, Immunology, Immunotherapy, Liver cancer, Lung cancer, MS - Multiple Sclerosis, Neurodegenerative disease, Neurologic disease, Ovarian cancer, Pancreatic cancer, PD - Parkinson's Disease, Prostate cancer, Repurposed Drugs, Research tool, Small molecule, Targeted therapy, TBI - Traumatic brain injury, Therapeutics
Category(s): Life Sciences > Therapeutics, Life Sciences > Biomarkers & Biomedical Research Tools
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Neuronal nitric oxide synthase (nNOS) inhibitors for targeted therapy of melanoma and neurodegenerative disorders
NU 2025-113 (NU 2016-137, NU 2018-176, NU 2019-179, NU 2023-007, NU 2024-250, NU 2025-038)
INVENTORS
Richard Silverman
Weinberg College of Arts and Sciences, Department of Chemistry
SHORT DESCRIPTION
Novel highly selective neuronal nitric oxide synthase (nNOS) inhibitors for targeted therapy of melanoma and other cancers with high expression...
Published: 4/30/2026
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Updated: 4/1/2026
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Inventor(s):
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, AUTM26C1, BIAUTM1, Cancer/Oncology, Cardiology and Cardiovascular disease, Immunodeficiency, Immunology, Immunotherapy, Melanoma, Neurodegenerative disease, Neuroinflammation, nNOS, Small molecule, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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Pyrimidine-2,4,6-Trione Derivatives Preparation and Their Use for the Treatment of Amyotrophic Lateral Sclerosis (ALS) ALS Therapy
NU 2009-072
INVENTORS
Richard Morimoto*
Richard Silverman*
Gavin Guoyao Xia
SHORT DESCRIPTION
This invention discloses novel pyrimidine-2,4,6-trione derivatives (PYT compounds) that prevent abnormal protein aggregation associated with ALS, offering a promising new therapeutic approach for neurodegenerative diseases.
BACKGROUND
Amyotrophic...
Published: 11/18/2025
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Updated: 11/18/2025
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Inventor(s):
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, Neurodegenerative disease, Therapeutics
Category(s): Life Sciences > Therapeutics
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Therapeutic Intervention for ALS and Dementia
NU 2021-058
INVENTORS
Evangelos Kiskinis*
Juan Alberto Ortega Cano Marco Boccitto
SHORT DESCRIPTION
Design of an RNA-based oligonucleotide that protects from dipeptide repeat protein toxicity for the treatment of ALS and FTD.
BACKGROUND
Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are devastating neurodegenerative diseases...
Published: 5/7/2026
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Updated: 1/22/2025
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Inventor(s):
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, CNS - Central Nervous System, DPR - Dipeptide Repeat Proteins, FTD - Frontotemporal Dementia, Neurodegenerative disease, Neurology, RNA - Ribonucleic Acid, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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ASOs targeted towards mis-spliced UNC13A
NU2022-200
INVENTORS
Wanhao Chi
Jonathan Watts
Evangelos Kiskinis*
BACKGROUND
Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative that leads to 1 in 500 deaths in adults. There is no cure to stop or reverse ALS, thus there is a need to better understand its pathobiology and develop effective therapeutics. Northwestern...
Published: 4/13/2026
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Updated: 10/17/2024
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Inventor(s):
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, FTD - Frontotemporal Dementia, Neurodegenerative disease, Neurology, Nucleic Acids, Rare diseases, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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Combination Treatment to Reduce Pathological Proteins in Neurodegenerative and Lysosomal Storage Diseases
NU 2021-206
INVENTORS
Joseph Mazzulli*
Iva Stojkovska
SHORT DESCRIPTION
A small molecule combination treatment to improve lysosomal function in neurodegenerative and lysosomal storage diseases
BACKGROUND
Many neurodegenerative disorders are characterized by a collapse in proteostasis, as shown by the accumulation of insoluble protein aggregates...
Published: 5/6/2026
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Updated: 5/23/2022
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Inventor(s):
Keywords(s): AD - Alzheimer’s Disease, ALS - Amyotrophic Lateral Sclerosis, HD - Huntington's Disease, Neurodegenerative disease, Neurology, PD - Parkinson's Disease, Rare diseases, Small molecule, Therapeutics
Category(s): Life Sciences > Therapeutics
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