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Enhanced Genome Editing Technologies for Long Polynucleotide Integration
Enhanced Genome Editing Technologies for Long Polynucleotide Integration
Background
Prime editing represents a promising frontier in genome editing, primarily enabling precise single-point mutations and small insertions or deletions. However, these methods traditionally support modifications up to about 100 base pairs, leaving a gap in the capability...
Published: 3/24/2026
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Updated: 3/24/2026
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Inventor(s):
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Disease Area > Rare Diseases
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Advancing Treatment of Hemoglobinopathies: Precision Gene-Editing and Base Editing to Restore Fetal Hemoglobin Production
Advancing Treatment of Hemoglobinopathies: Precision Gene-Editing and Base Editing to Restore Fetal Hemoglobin Production
Background
Hemoglobinopathies, such as β-thalassemias and sickle cell anemia, are genetic disorders with significant impact on global health, affecting millions with severe anemia due to abnormal or deficient β-globin....
Published: 3/24/2026
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Updated: 3/24/2026
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Inventor(s):
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Disease Area > Hematology
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A base editing approach for Shwachman-Diamond-Syndrome
A base editing approach for Shwachman-Diamond-Syndrome
Background
Shwachman-Diamond-Syndrome (SDS), is life threatening genetic condition caused in 90% of cases by a mutation in the SBDS-gene, which leads to defective splicing and loss of protein expression. Patients may develop many symptoms such as frequent infections due to low white blood cell...
Published: 3/26/2026
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Updated: 12/8/2025
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Inventor(s): Christian Brendel
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Therapeutics > Gene Therapy
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Advancing Hemoglobinopathy Care: Innovative BCL11A Inhibition for Sustained Fetal Hemoglobin Production
Advancing Hemoglobinopathy Care: Innovative BCL11A Inhibition for Sustained Fetal Hemoglobin Production
Background
Hemoglobinopathies, including sickle cell anemia and β-thalassemias, are genetic disorders caused by mutations that impair the production or structure of of β-globin chains in hemoglobin. These conditions result in patients...
Published: 3/25/2026
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Updated: 12/5/2025
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Inventor(s): Daniel Bauer, Yuxuan Wu, Jing Zeng, Scot Wolfe, Mehmet Bolukbasi, Benjamin Roscoe, Pengpeng Liu, Luk.Kevin
Keywords(s): Sickle Cell Anemia
Category(s): Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Invention Type > Therapeutics > Gene Editing
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Advanced CRISPR/Cas Technology for Targeted Fetal Hemoglobin Reinduction in Hemoglobinopathy Treatment
Advanced CRISPR/Cas Technology for Targeted Fetal Hemoglobin Reinduction in Hemoglobinopathy Treatment
Background
Hemoglobinopathies, such as β-thalassemias and sickle cell anemia, represent a significant unmet medical need affecting millions globally. These genetic disorders are characterized by defective or deficient production of normal β-globin,...
Published: 3/26/2026
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Updated: 10/9/2025
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Inventor(s): Daniel Bauer
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology, Technology Classifications > Invention Type > Therapeutics > Gene Therapy
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TAZ gene replacement therapy
TAFAZZIN gene replacement therapy
Background
Barth Syndrome (BTHS) is an X-linked, potentially lethal genetic disease that affects about 1 in 0.3 to 0.4 million live births. It is characterized by cardiomyopathy, skeletal myopathy, neutropenia, growth delay, poor feeding, and organic aciduria, with cardiac complications and neutropenia being the...
Published: 3/30/2026
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Updated: 3/10/2025
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Inventor(s): William Pu, Suya Wang
Keywords(s):
Category(s): Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Disease Area > Cardiology
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Temp: Repair of ELANE therapeutic gene editing for congenital neutropenia
Therapeutic Gene Editing for Severe Congenital Neutropenia
Background
Severe congenital neutropenia (SCN) is a rare inherited disorder characterized by low absolute neutrophil counts which increases the risk of life-threatening infections. Occurrence of SCN is attributed to changes in various genes, but an estimated 50% of severe congenital...
Published: 3/26/2026
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Updated: 4/11/2024
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Inventor(s): Daniel Bauer, Scot Wolfe, Peter Newburger, Josias Frazão, Shuquan Rao
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Disease Area > Rare Diseases
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In vivo high efficiency somatic homology directed repair
In vivo high-efficiency somatic homology directed repair
Background
Recent advances in genome sequencing techniques and analysis methods have significantly accelerated the ability to catalog and map genetic factors associated with a diverse range of biological functions and diseases, but precise genome targeting technologies are needed to enable...
Published: 3/30/2026
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Updated: 2/23/2024
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Inventor(s): William Pu, Nathan VanDusen, Yanjiang Zheng
Keywords(s):
Category(s): Technology Classifications > Disease Area > Cardiology, Technology Classifications > Invention Type > Therapeutics > Gene Therapy
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Choroid plexus targeted NKCC1 gene therapy for the treatment of hydrocephalus
Choroid Plexus Targeted NKCC1 Gene Therapy for the Treatment of Hydrocephalus
AAV-targeted augmentation of NKCC1 expression to reverse blood-induced ventriculomegaly and increased CSF clearance capacity
Background
A subset of infants and adults with hemorrhagic stroke and intraventricular hemorrhage (IVH) ultimately develop a life-threatening accumulation...
Published: 3/27/2026
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Updated: 4/18/2023
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Inventor(s): Maria Lehtinen, Cameron Sadegh, Huixin Xu, Ryann Fame, Jason Sutin, Benjamin Warf, Pei-Yi (Ivy) Lin
Keywords(s):
Category(s): Technology Classifications > Invention Type, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Disease Area, Technology Classifications > Disease Area > Neurology
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GATA1 Gene Therapy for Diamond-Blackfan Anemia
GATA1 Gene Therapy for Diamond-Blackfan Anemia
A unified lentiviral vector based gene therapy approach, which has potential to treat DBA in all patients
Background
Diamond-Blackfan anemia (DBA) is a rare inherited disorder that that occurs when the bone marrow fails to make red blood cells, thereby leading to severe anemia.
Current therapeutic options...
Published: 3/26/2026
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Updated: 3/9/2023
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Inventor(s): Vijay Sankaran, Leif Ludwig, Richard Voit, David Williams
Keywords(s):
Category(s): Technology Classifications > Invention Type, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Disease Area, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Disease Area > Hematology, Technology Classifications > Invention Type > Therapeutics > Gene Therapy
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