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Enhanced Delivery and Efficacy of Antisense Drugs via Covalently Linked Nanodisc Scaffolds
Application
A synthetic nanodisc (ND) scaffold for delivering antisense oligonucleotides (ASOs) for the treatment of disease.
Key Benefits
Improved cellular uptake and protein suppression using antisense oligos.
Longer ASO half-life compared to other ASO delivery methods.
Potential to overcome the delivery and efficacy challenges using ASOs for...
Published: 7/26/2024
Contributor(s): Khalid Salaita, Radhika Sharma
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Repurposing a Potent MDM2 Inhibitor for Leukemia Treatment
Application
VERU-111, also known as sabizabulin, is a potent microtubule inhibitor and, notably, an MDM2 inhibitor. This repurposed compound demonstrates remarkable efficacy in the treatment of Acute Myeloid Leukemia (AML).
Key Benefits
Unique dual-action functionality as a microtubule and MDM2 inhibitor.
Successful inhibition of MDM2, an oncogene...
Published: 7/26/2024
Contributor(s): Deborah DeRyckere, Muxiang Zhou, Lu Bing Gu, Douglas Graham, Tao Liu
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Advancing Lymphatic Endothelial Cell Generation from iPSCs
Application
The developed protocol offers a reproducible and scalable method for generating lymphatic endothelial cells (LECs) from induced pluripotent stem cells (iPSCs), addressing critical challenges in the field of lymphatic-related therapies.
Key Benefits
Reproducible and scalable protocol for generating LECs from iPSCs.
Utilizes specific...
Published: 7/26/2024
Contributor(s): Katherine Hekman, Katherine Owsiany
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Drug Platform for L-Asparaginases for the Treatment of Leukemias
Application
A novel humanized therapeutic L-asparaginase compound to treat acute lymphoblastic leukemia (ALL) and other cancers.
Key Benefits
A novel drug discovery platform for developing asparaginase-based compounds to treat leukemias and other cancers.
The derivatives could exhibit less immunogenicity and systemic toxicity than FDA-approved...
Published: 6/12/2024
Contributor(s): Sunil Raikar, Christopher Doering, H. (Harold) Trent Spencer, Kristopher Knight, Harrison Brown
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Drug Screening System for Cardioprotection
Application
A high throughput screening assay for developing therapies to protect from drug and alcohol-induced cardiotoxicity.
Key Benefits
A cardiovascular stem-cell-based screening assay that can identify drugs that protect the heart from toxic medications and alcohol.
Preliminary data in the laboratory show the system is clinically relevant...
Published: 7/26/2024
Contributor(s): Chunhui Xu, Antonio Rampoldi, Yuhong Du
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ONC201 for the Treatment of Vascular Aneurysms
Application
A repurposed therapeutic for the preventative and treatment of aneurysms.
Key Benefits
A repurposed cancer drug that targets cells known to be damaged during the progression of an aneurysm.
The drug has been tested within an animal model of aortic aneurysms, where it prevented the severe complications of the disease and protected the...
Published: 5/21/2024
Contributor(s): Alejandra Maria San Martin
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Small Molecules for Treating Cystic Fibrosis
ApplicationSmall molecule compounds to potentiate most common type of CFTR mutation in cystic fibrosis patients.Key BenefitsPotentiates ΔF508-hCFTR mutation, addressing more than 60 percent of CF patients. Market SummaryCystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and ion channel that malfunctions in the genetic...
Published: 7/28/2023
Contributor(s): Nael McCarty, Guiying Cui, Hanoch Senderowitz, Netaly Khazanov
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Microfluidic and Organotypic Tumor Slice Culture Drug Testing Platform for Individualized Cancer Therapy
Application
A drug combination screening/testing platform for individualized cancer therapy.
Key Benefits
Rapid screening of effective drug combination.
Integrated tumor microenvironment.
Identifying personalized therapy for individual cancer patient.
Market Summary
Traditional two-dimensional (2D) in vitro culture models for drug development...
Published: 6/21/2024
Contributor(s): Tobey John MacDonald, Anna Kenney, Abhinav Dey, YongTae (Tony) Kim
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Use of miRNA-483 and its Target Genes in Treatment of Cardiovascular and Inflammatory Diseases
Application
Therapeutic use of miR-483 mimic for treating aortic valve clarification.
Key Benefits
MiR-483 or mimics of miR-483 could be used as a therapeutic.
Could also be a target for additional drug development toward a non-invasive therapy for aortic valve calcification.
Market Summary
Aortic stenosis (AS) is the most common type of heart...
Published: 7/26/2024
Contributor(s): Hanjoong Jo, Joan Fernandez Esmerats, Nicolas (Nico) Villa-Roel
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HTiP: High-Throughput Immunomodulator Phenotypic Screening Platform
Application
High Throughput Screening for identifying effective immuno-modulators.
Key Benefits
HT screening platform replicating complex immune medicated response to facilitate discovery of small molecule immunomodulatory agents.
Simplicity and dual readouts of the design enable the screening of large chemical libraries to identify compounds...
Published: 4/23/2024
Contributor(s): Xiulei Mo, Haian Fu
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