Indolium 1 for Advanced Melanoma Treatment
Application
A small molecule compound for the treatment of melanoma.
Key Benefits
Novel small molecule compound for the treatment of resistant melanoma.
Well tolerated in vivo.
Market Summary
Malignant melanoma is a type of skin cancer that begins in melanocyte cells. Melanoma is the 19th most commonly occurring cancer. Exposure to UV radiation...
Published: 1/17/2024
Contributor(s): Jack Arbiser
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Human Brain Proteins with Evidence Consistent with Causality of Alzheimer’s and Depression
Application
Research and therapeutic targets for Alzheimer’s disease, depression, and other neurological conditions mediated by protein expression in the brain.
Key Benefits
These findings can be utilized as a research tool to better understand the genetic basis of Alzheimer’s disease.
Can also be utilized to develop therapeutics for...
Published: 12/20/2023
Contributor(s): Aliza Wingo, Thomas Wingo, Yue Liu, Allan Levey, Nicholas Seyfried, Duc Duong
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Combination Antibiotic Therapy to Exploit Multiple Heteroresistance
Application
Treatment with combinations of FDA-approved antibodies targeting small drug-resistant cell populations for infectious disease.
Key Benefits
Specific combinations of existing drugs that target small subpopulations of bacterial cells resistant to many standard of care treatment regimens.
Potential to overcome the drug resistance crisis...
Published: 12/1/2023
Contributor(s): David Weiss, David Hufnagel, Victor Band
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Vaccines and Antibody Therapeutics for HPV-related Cancers
Application
Treatment and prevention of HPV-induced malignancies.
Key Benefits
Identification of a protein expressed in a large number of HPV-induced cancers.
Antibodies have been generated that bind to the protein and potentially elicit an immune response to the cancer cells.
The antibodies can be used to develop new treatments and vaccines...
Published: 12/1/2023
Contributor(s): Rafi Ahmed, Andreas Wieland
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Antisense Restoration of ELAVL3 for Neurological Diseases
Application
Antisense therapeutics targeting ELAVL3 expression for neurodegenerative disorders.
Key Benefits
Antisense approach for restoring protein levels and function of ELAVL3 and TDP-43.
Preclinical data show the ability of several ELAVL3-targeting ASOs to restore protein expression via in vitro experiments.
Potential to be a new medicine...
Published: 3/1/2024
Contributor(s): Zachary McEachin
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Flumazenil for Treatment of CNS Symptoms in Myotonic Dystrophy
Application
A class of drugs that inhibit molecules responsible for the severe nervous system complications associated with myotonic dystrophy.
Key Benefits
Potentially the first medication for treating the severe nervous system symptoms associated with myotonic dystrophy by targeting GABRG2.
Transdermal and oral administration.
May drive the...
Published: 12/1/2023
Contributor(s): Gary Bassell, Andrew Jenkins, David Rye, Eric Wang, Maurice Swanson, Lyndon Lien
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Novel Therapeutic Target for Fragile X Syndrome and Neurodevelopmental Disorders
Application
Salvianolic acid C for treating Fragile X Syndrome (FXS).
Key Benefits
New therapeutic drug candidate for the treatment of FXS.
Preliminary data in the laboratory in organoids treated with the drug showed rescued cell phenotypes known to be associated with the disease in humans.
Potential to treat other neurodevelopmental disorders.
Market...
Published: 3/1/2024
Contributor(s): Peng Jin, Chuan He, Zhongyu Zou
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Cell and Gene Therapies for Hemophagocytic Lymphohistiocytosis
Application
Gene/cell therapy to treat hemophagocytic lymphohistiocytosis.
Key Benefits
Gene therapy approach for treating patients with UNC13D mutated hemophagocytic lymphohistiocytosis.
Preliminary in vitro and in vivo data show the ability of the gene therapy to increase UNC13D protein levels and decrease T cell activation.
It is administered...
Published: 1/17/2024
Contributor(s): H. (Harold) Trent Spencer, Christopher Doering, Shanmuganathan Chandrakasan, Sarah Takushi
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Therapeutic for Potential Elimination of Chronic Hepatitis B
Application
A therapeutic technique to eliminate infection of patients suffering from chronic Hepatitis B Virus infection.
Key Benefits
May provide a cure for chronic HBV infection rather than just simple management.
High efficiency of infection targeting may produce minimal side effects.
May be used in combination with other HBV inhibitors.
Market...
Published: 3/11/2024
Contributor(s): Raymond Schinazi, Leda Bassit, Ting Nie
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Repurposing an FDA-Approved Drug for Treating Age-Associated Lung Fibrosis
Application
New use of an FDA-approved therapeutic, diroximel fumarate (Vumerity), for treating Idiopathic Pulmonary Fibrosis.
Key Benefits
Novel use of an FDA-approved drug, diroximel fumarate - DRF (Vumerity), for treating age-dependent lung fibrosis.
Lung-targeted inhaled delivery of DRF is effective for boosting lung antioxidant responses...
Published: 1/10/2024
Contributor(s): Louise Hecker, Kosuke Kato
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