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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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Inhibitors of MNK Kinase for Targeted Cancer and CNS Therapeutics
NU 2015-098 (NU 2015-098, NU 2018-036, NU 2025-036)
INVENTORS
Gary Schiltz (Weinberg College of Arts and Sciences, Department of Chemistry)*
SHORT DESCRIPTION
MNK kinase inhibitors that reduce cancer cell viability and proliferation and penetrates the brain for potential treatment of glioblastoma and CNS disorders.
BACKGROUND
Leukemia and brain...
Published: 5/13/2026
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Updated: 4/29/2026
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Inventor(s):
Keywords(s): AD - Alzheimer’s Disease, AML - Acute myeloid leukemia, ASD - Autism Spectrum Disorder, Bladder cancer, Brain cancer, Breast cancer, Cancer/Oncology, CNS - Central Nervous System, Colon cancer, Lung cancer, Neurodegenerative disease, Pancreatic cancer, Prostate cancer, Small molecule, Targeted protein degradation, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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Use of Nucleotide Synthesis Inhibitors for Targeted Therapy in MLL3/4 COMPASS Mutant Cancer
NU 2022-053
INVENTORS
Ali Shilatifard (Feinberg School of Medicine, Department of Biochemistry and Molecular Genetics)*
Zibo Zhao (Feinberg School of Medicine, Department of Biochemistry and Molecular Genetics)*
SHORT DESCRIPTION
A therapeutic platform that targets MLL3/4 COMPASS mutant cancers by using nucleotide synthesis inhibitors to disrupt...
Published: 5/26/2026
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Updated: 4/29/2026
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Inventor(s):
Keywords(s): Bladder cancer, Breast cancer, Cancer/Oncology, Colon cancer, Gastric cancer, Leukemia, Lung cancer, Lymphoma, Prostate cancer, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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Precision Gene Therapy for Diseases Driven by Endothelial Dysfunction
SHORT DESCRIPTION
Dual promoter system constructs for tissue-specific and cell-type-specific gene expression, enabling precision gene therapy targeting endothelial dysfunction in specific organs.
INVENTORS
Youyang Zhao*
Ann & Robert H Lurie Children's Hospital of Chicago
* Principal Investigator
NU...
Published: 5/8/2026
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Updated: 4/27/2026
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Inventor(s):
Keywords(s): Cardiology and Cardiovascular disease, CMD - Cardiometabolic disease, CPD - Cardiopulmonary disease, Gene therapy, Pulmonary disease, Regenerative medicine, Targeted therapy, Therapeutics, Vascular disease
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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Selective PAD2 Inhibitors for Neuroprotection in Traumatic Brain Injury
SHORT DESCRIPTION
Novel PAD2-selective inhibitor platform targeting neuroinflammation to support both acute neuroprotection and long-term functional recovery as a first-in-class treatment of traumatic brain injury.
INVENTORS
Hasan Alam*
Northwestern University Feinberg School of Medicine, Department of Surgery (Trauma...
Published: 5/12/2026
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Updated: 4/27/2026
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Inventor(s):
Keywords(s): CNS - Central Nervous System, CVA - Cerebrovascular Accident, Neurodegenerative disease, Neuroinflammation, Neurologic disease, Neurology, Small molecule, Targeted therapy, TBI - Traumatic brain injury, Therapeutics
Category(s): Life Sciences > Therapeutics
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Biomolecular Condensates as Protein Degradation Tools for Intracellular Targets
SHORT DESCRIPTION
A targeted-protein degradation platform for intracellular targets, addressing resistant oncogenic and neurodegenerative targets.
INVENTORS
Shana Kelley*
Weinberg College of Arts & Sciences, Department of Chemistry
Yi Li
* Principal Investigator
NU Tech ID: NU 2025-050
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, Biologic, Biomolecular condensates, Cancer/Oncology, Drug delivery, Nanoparticle, Neurodegenerative disease, Neurologic disease, Neurology, PD - Parkinson's Disease, Platform technology, Targeted protein degradation, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics, Life Sciences > Biomarkers & Biomedical Research Tools
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Methods to Treat Neurodegenerative Disorders by Reducing Nuclear Aggregates and Modulating Adenosine‑to‑Inosine RNA Editing
SHORT DESCRIPTION
Novel disease‑modifying approach that dissolves toxic nuclear RNA–protein aggregates and restores synaptic function in neurodegenerative diseases by inhibiting adenosine‑to‑inosine (A‑to‑I) RNA editing and modulating ADAR enzymes.
INVENTORS
Joseph Mazzulli*
Northwestern University Feinberg School...
Published: 5/13/2026
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Updated: 4/27/2026
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Inventor(s):
Keywords(s): Lewy bodies, Neurodegenerative disease, Neurology, PD - Parkinson's Disease, RNA - Ribonucleic Acid, Small molecule, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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Mitigating aberrant Cdk5 Activation in SMA – A Novel Therapeutic Strategy Targeting Mitochondrial Function
SHORT DESCRIPTION
A targeted strategy that mitigates aberrant Cdk5 activity to restore mitochondrial function and reduce motor neuron degeneration in SMA models.
INVENTORS
Yongchao Ma*
Northwestern University Feinberg School of Medicine, Department of Pediatrics
* Principal Investigator
NU Tech...
Published: 5/7/2026
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Updated: 4/27/2026
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Inventor(s):
Keywords(s): Adjunct therapy, Neurodegenerative disease, Neurologic disease, Neurology, Rare diseases, SMA - Spinal muscular atrophy, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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Use of Neuronal NOS Inhibitors for Alzheimer's Therapeutic Intervention
PROPOSED TITLE (cut & paste into Alternate Title): Use of Neuronal NOS Inhibitors for Alzheimer's Therapeutic Intervention
NU 2023-007
SHORT DESCRIPTION
A small molecule based approach that mitigates Alzheimer's neurodegeneration by inhibiting neuronal nitric oxide synthase to prevent tau phosphorylation and reduce Aβ oligomer formation.
INVENTORS
Richard...
Published: 4/27/2026
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Updated: 4/27/2026
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Inventor(s):
Keywords(s): AD - Alzheimer’s Disease, Covalent inhibitor, Kinase Inhibitor, Neurodegenerative disease, Neurology, nNOS, Small molecule, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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