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Improved Gene Therapy to Enhance mRNA Expression

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: 5/13/2024 | 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: 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

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: 5/10/2024 | 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: 5/10/2024 | Inventor(s): Hai-Quan Mao, Stephany Tzeng, Jordan Green, Yizong Hu, Sashank Reddy, Leonardo Cheng

Keywords(s):

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: 5/9/2024 | 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

Compositions and Methods of Increasing RNA Translation

Value Proposition mRNA use for therapeutic purposes has proven useful in treating a wide array of diseases. Major challenges of mRNA therapies include immunogenicity, poor expression, and low stability. This therapy contains modifications improving mRNA stability, efficiency, and immunogenicity. This technology allows for the translation of almost...

Published: 5/10/2024 | Inventor(s): Jeffery Coller, Zachary Mandell

Keywords(s):

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

Polymer-Nucleic Acid Nanoparticles for Gene Editing

Inventors: Jordan J. Green, Stephany Yi Tzeng, Garry R. Cutting, Erin W. Kavanagh Unmet NeedCystic fibrosis (CF) is a progressive, genetic disease that affects the lungs, pancreas, and other organs. There are close to 40,000 children and adults living with cystic fibrosis in the United States (see CFF). In people with CF, mutations in the cystic fibrosis...

Published: 5/10/2024 | Inventor(s): Jordan Green, Stephany Tzeng, Garry Cutting, Erin Kavanagh

Keywords(s):

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

Improving the sensitivity of CRISPR off-target detection through modulation of DNA repair

Value Proposition:· Utilizes components of existing workflow to better detect off-target gene editing· Capable for use in in vivo or in vitro settings· Maximizes impact of CRISPR-Cas9 in therapeutic developmentTechnology Description· Researchers at Johns Hopkins have developed a method to detect off-target editing with high...

Published: 5/10/2024 | Inventor(s): Roger Zou, Yang Liu, Taekjip Ha, Oscar Reyes Gaido

Keywords(s):

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

Identification of DNA Polymerase Theta Inactivation Mechanism

Unmet NeedLoss of DNA repair is an early and frequent event in the growth of tumors, occurring in 40 to 50% of breast, lung and ovarian cancer patients. The inability to repair damaged DNA provides a selective growth advantage to tumor cells as this results in genetic instability and enhanced mutation rates, which can drive tumor growth. These DNA...

Published: 5/9/2024 | Inventor(s): Marc Greenberg, Daniel Laverty

Keywords(s):

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

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: 5/9/2024 | Inventor(s): Kristen Kozielski, Stephany Tzeng, Jordan Green

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

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