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Specialized adeno-associated virus for central nervous system gene therapy
Specialized adeno-associated virus for central nervous system gene therapy
Background
Unlike other cell types in the human body, nerve cells (neurons) have an extremely limited ability to replace themselves through division following damage to the nervous system. Most injuries and diseases of the central nervous system (CNS) involve damage to axons,...
Published: 9/16/2025
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Updated: 6/9/2023
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Inventor(s): Zhigang He, Xue Yao, Zicong Zhang, Benedikt Brommer
Keywords(s): CNS, Gene Therapy, Therapeutic, Trauma
Category(s): Technology Classifications > Disease Area, Technology Classifications > Disease Area > Neurology, Technology Classifications > Invention Type, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Drug Delivery
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Methods and compositions for treating ataxia telangiectasia
Methods and compositions for treating ataxia telangiectasia
Background
Ataxia telangiectasia (A-T) is an incurable autosomal recessive disease that occurs in 1 in 40,000 to 100,000 people worldwide. It is caused by mutations in the Ataxia Telangiectasia Mutated (ATM) gene. A-T is characterized by neurodegeneration in the central nervous system, immune...
Published: 6/26/2025
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Updated: 5/30/2023
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Inventor(s): Timothy Yu, Jinkuk Kim
Keywords(s): CNS, Gene Therapy, Genetic Disorder, Rare Disease
Category(s): Technology Classifications > Invention Type, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > ASOs, Technology Classifications > Disease Area, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Disease Area > Neurology
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Oligonucleotide approach to treat exon skipping diseases
Oligonucleotide approach to treat exon skipping diseases
Background
Almost half of mammalian genomes contain repeat DNA sequences known as transposable elements (TE) or transposons. Many transposons have the ability to mobilize and change location in the genome, yet their role in human health and disease is not well defined. RNA transposons (i.e.,...
Published: 3/20/2026
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Updated: 5/30/2023
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Inventor(s): Timothy Yu, Alice Lee
Keywords(s): CNS, Gene Therapy, Genetic Disorder, Rare Disease
Category(s): Technology Classifications > Invention Type, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > ASOs, Technology Classifications > Disease Area, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Disease Area > Neurology
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Antibody mediated conditioning with immunosuppression to enable allogeneic transplantation
Antibody mediated conditioning with immunosuppression to enable allogeneic transplantation
Background
Hematopoietic (blood-forming) stem cell transplantation (HSCT) is the transplantation of autologous or allogeneic hematopoietic stem cells used to treat patients with cancer and disorders of the blood and immune system. Hematopoietic stem cells are...
Published: 3/2/2026
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Updated: 5/25/2023
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Inventor(s): Derrick Rossi, Agnieszka Czechowicz, Philip Murphy, Zhanzhuo Li
Keywords(s): Allogeneic Transplantation, Autoimmmune, Cancer, CRISPR, Gene Therapy
Category(s): Technology Classifications > Invention Type, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Cell Therapy, Technology Classifications > Disease Area, Technology Classifications > Disease Area > Hematology
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Nanoparticles Targeting Gene Editing Systems to Cancer Cells
Nanoparticles Targeting Gene Editing Systems to Cancer Cells
Targeted gene therapy delivery platform for oncology indications with stable nanoparticles and no cytotoxicity
Background
To date, existing gene editing delivery systems involve viral vectors or cationic materials, which carry a high human safety concern. Researchers at Boston Children’s...
Published: 6/30/2025
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Updated: 5/5/2023
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Inventor(s): Marsha Moses, Peng Guo, Jiang Yang, Debra Auguste
Keywords(s): Cancer, Gene Therapy, Oncology, Viral Vector
Category(s): Technology Classifications > Disease Area, Technology Classifications > Disease Area > Oncology, Technology Classifications > Invention Type, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Drug Delivery
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