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Gene therapy for neurofibromatosis type 1

JHU Ref #: C18504Value Proposition· Targets direct genetic cause of NF1 rather than only symptoms.· Improves therapeutic options for NF1 patients.· AAV vector is one of the safest viral vectors for gene therapy.· Market for NF1 gene therapies is quite open.Unmet NeedNeurofibromatosis type 1 (NF1) affects approximately 1 in 2,500...

Published: 3/13/2026 | Inventor(s): Renyuan Bai, Verena Staedtke

Keywords(s):

Category(s): Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms

Bicistronic Genes and Fusion Proteins for Immune Activation

Value Proposition:· Dual specific targeting for improved full T-cell and amplification.· Interchangeable protein components can be customized for cancer, infectious disease, and autoimmune disorder therapies.· Deliverable by nanoparticles into cells in vivo, in vitro, and ex vivo for immune activation.Unmet Need:Immunotherapy is a rapidly...

Published: 3/13/2026 | Inventor(s): Jordan Green, Joanna Yang, Stephany Tzeng, Jamie Spangler

Keywords(s):

Category(s): Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Immunotherapies, Technology Classifications > Therapeutic Modalities > Proteins, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Research Tools > Vectors & Plasmids, Technology Classifications > Research Tools > Proteins, Ligands & Receptors, Technology Classifications > Research Tools > Nucleic Acids

Methods for High-throughput Identification of Genomic Harbors for Transgene Expression in Cells

Value Proposition:· High-throughput screening of loci for exogenous gene insertion · Quantifies expression level of exogenous genes in cell type of interest Unmet Need· Exogenous or synthetic genes that are inserted into the genome serve various purposes, depending on the context and goals of the genetic modification. This includes...

Published: 3/13/2026 | Inventor(s): Reza Kalhor, Jinwoo Jun, Kathleen Leeper

Keywords(s):

Category(s): Technology Classifications > Research Tools > Assays, Technology Classifications > Research Tools > Nucleic Acids, Technology Classifications > Research Tools > Vectors & Plasmids, Technology Classifications > Research Tools > Bioinformatics, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Therapeutic Modalities > Gene Therapies

A CRISPR-based technology to treat cytomegalovirus (CMV) and other Viral Infections.

Value PropositionNovel Mechanism: Utilizes a Crispr/Cas9 system to target the viral genome to prevent production of the disease-causing protein 1E1 in CMV.Widely Adaptable: Design allows modulation to fit a wide variety of other viral genomes to combat other infectious diseases.Therapeutic Potential: Administration of proposed technology to infected...

Published: 3/13/2026 | Inventor(s): Akira Sawa, Koko Ishizuka, Kazuhiro Ishii, Stephanie Tankou

Keywords(s):

Category(s): Technology Classifications, Technology Classifications > Therapeutic Modalities > Cell Therapies, Clinical and Disease Specializations, Clinical and Disease Specializations > Infectious Diseases > Cytomegalovirus, Technology Classifications > Therapeutic Modalities > Gene Therapies

Proinflammatory Immature Myeloid Cells and Their Use in Treatment of Cancer

Value Proposition:· Gene-editing approach creates myeloid cells polarized into a pro-inflammatory phenotype.· Engineered cells persist even in an immune-suppressive environment. · High potential as anti-cancer therapy, as cells are present in all tissues and can easily infiltrate tumors. · Cells demonstrate robust expression of pre-inflammatory...

Published: 3/13/2026 | Inventor(s): Alan Friedman

Keywords(s):

Category(s): Technology Classifications > Therapeutic Modalities > Cell Therapies, Technology Classifications > Therapeutic Modalities > Gene Therapies, Clinical and Disease Specializations > Oncology, Clinical and Disease Specializations > Immuno-Oncology

Kinetically controlled methods to prepare concentrated shelf-stable nucleic acid transfection vehicles

Value Proposition:· Technology creates particles optimally size for use as vehicles for cell transfection.· The reagents and processes for particle creation are kinetically controlled and reproducible.· Cationic particles created using these methods can carry larger payloads than current vehicles.· Reagents and nucleic acid-loaded...

Published: 3/13/2026 | Inventor(s): Hai-Quan Mao, Yizong Hu, Jinghan Lin

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mRNA Intensifier to treat Haploinsufficiencies

Unmet Need / Invention Novelty: Haploinsufficiency occurs when one gene allele is inactivated and the amount of gene product expressed from the remaining active allele is insufficient for proper gene function. Targeted restoration of mRNA expression and translation may offer a therapeutic window. Technical Details: Johns Hopkins researchers designed...

Published: 3/13/2026 | Inventor(s): Jeffery Coller, Bahareh Torkzaban

Keywords(s):

Category(s): Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Proteins

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

Gene therapy for SYNGAP1 encephalopathy and SYNGAP1-related disorders

Unmet Need: SYNGAP1-related Intellectual Disability (SRID, MRD5) is a severe neurodevelopmental disorder (NDD) characterized by encephalopathy, intellectual disability (ID), autism spectrum disorder (ASD), and epilepsy and accounts for 0.5-1% of all NDDs and ~1% of the ~200 million ID cases worldwide (SYNGAP Research Fund, 2020). Supportive management...

Published: 3/13/2026 | Inventor(s): Richard Huganir, Ingie Hong, Yoichi Araki, Richard Johnson

Keywords(s):

Category(s): Clinical and Disease Specializations > Rare Diseases, Clinical and Disease Specializations > Neurology, Technology Classifications > Therapeutic Modalities > Gene Therapies

Compositions of Kinetic Nanoparticles containing Nucleic Acids, Polycations, and Lipids with Defined Sizes, and Methods of Producing the Same

Value Proposition:· Scalable, efficient, and tunable nanoparticle creation technique that can deliver a variety of payloads· Polymer-lipid nanoparticles are biodegradable and are being tested for immunogenicity· Can be used for in vivo gene therapy delivery as well as in vitro cell transfection and research applications Technology...

Published: 3/13/2026 | Inventor(s): Hai-Quan Mao, Stephany Tzeng, Jordan Green, Yizong Hu, Sashank Reddy, Leonardo Cheng

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