|
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
|
Updated: 3/24/2026
|
Inventor(s):
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Disease Area > Rare Diseases
|
|
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
|
Updated: 12/8/2025
|
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
|
|
Enhanced BCL11A RNP / CRISPR delivery & editing using a 3xNLS-Cas9
Advancing Gene Therapy for Hemoglobinopathies: Enhanced CRISPR-Cas9 with Multi-NLS Integration
Background
Sickle cell disease (SCD) and β-thalassemia are severe β-globin disorders affecting millions globally, characterized by defective adult hemoglobin that fails to transport oxygen effectively, leading to significant morbidity. Existing...
Published: 3/25/2026
|
Updated: 10/13/2025
|
Inventor(s): Daniel Bauer, Pengpeng Liu, Scot Wolfe, Yuxuan Wu, Jing Zeng
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Gene Editing
|
|
Compounds Treating AP-4 Associated Hereditary Spastic Paraplegia
Compounds Treating AP-4 Associated Hereditary Spastic Paraplegia
Background
Hereditary Spastic Paraplegia (HSP) is a group of inherited neurological disorders characterized by progressive spasticity, weakness, and gait abnormalities and affects approximately 5-10 per 100,000 individuals worldwide. Among its subtypes, Adaptor Protein Complex 4 (AP-4)-related...
Published: 3/23/2026
|
Updated: 7/28/2025
|
Inventor(s): Darius Ebrahimi-Fakhari, Mustafa Sahin, Masato Satoh, Jed Hubbs
Keywords(s): Hereditary Spastic Parapeligia, Movement Disorder, Neuromuscular Disorder
Category(s): Technology Classifications > Disease Area > Neurology, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Therapeutics > Small Molecule
|
|
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
|
Updated: 3/10/2025
|
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
|
|
Genetic regulators of fetal hemoglobin
Genetic Regulators of fetal hemoglobin
Background
During human development, the major hemoglobin in red blood cells changes from fetal hemoglobin (HbF, α₂γ₂) to adult hemoglobin (HbA, α₂β₂) shortly after birth. This change, known as the fetal-to-adult hemoglobin switch, involves a transcriptional shift from the γ-globin...
Published: 3/26/2026
|
Updated: 11/20/2024
|
Inventor(s): Vijay Sankaran, Liam Cato, Rick Li, Yi-Chin Lu, Chun-Jie Guo
Keywords(s):
Category(s): Technology Classifications, Technology Classifications > Disease Area > Hematology, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Novel Targets
|
|
Therapeutic twin prime editing of hematopoietic stem cells for TINF2 dyskeratosis congenita
Therapeutic twin prime editing of hematopoietic stem cells for TINF2 dyskeratosis congenita
Background
Dyskeratosis congenita (DC) is a rare, genetic form of bone marrow (BM) failure. It is estimated that one in one million people has this condition. About 50% of the people who have DC have changes in the genes that are important in telomere maintenance,...
Published: 3/26/2026
|
Updated: 11/20/2024
|
Inventor(s): Daniel Bauer, Sebastien Levesque
Keywords(s):
Category(s): Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Disease Area > Hematology, Technology Classifications > Invention Type > Therapeutics > Gene Editing
|
|
Improving gene editing by modulating nucleotide metabolism
Improving gene editing by modulating nucleotide metabolism
Background
Currently, most treatments for genetic blood-related disorders (both non-cancerous and cancerous) aim to alleviate symptoms, manage complications, and slow disease progression, rather than completely correcting the underlying genetic defect. In some cases, patients may qualify...
Published: 3/26/2026
|
Updated: 11/20/2024
|
Inventor(s): Daniel Bauer, Sebastien Levesque
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics > Gene Editing, Technology Classifications > Disease Area > Rare Diseases
|
|
Manipulation of nucleotides for telomere diseases
Novel Drug Target for Regulating Telomeres in Disease
Background
Human chromosomes, which carry genetic information and play an essential role in inheritance and normal tissue regeneration, are capped in structures called telomeres, which protect the chromosomes from fraying during the cell replication process. With each cell replication, telomeres...
Published: 3/26/2026
|
Updated: 11/14/2024
|
Inventor(s): Suneet Agarwal, William Mannherz
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics > Small Molecule, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Disease Area > Oncology
|
|
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
|
Updated: 4/11/2024
|
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
|
