Targeted Therapies for Neurodegenerative and Infectious Diseases using Hydrophobic Auristatin F Compounds

The present inventive concept relates to the application of hydrophobic Auristatin F compounds to novel therapeutic areas, including neurodegenerative and infectious diseases, by conjugating these compounds to targeting moieties specific to disease-associated antigens.

Traditional antibody-auristatin drug conjugates have limitations in treating certain diseases, such as neurodegenerative and infectious diseases, due to their inability to effectively target and penetrate affected tissues. The original patent disclosed hydrophobic Auristatin F compounds and conjugates with activity against MDR+ cancer cells. However, these compounds were not explored for their potential in treating other diseases. The present inventive concept addresses this limitation by applying the core technology of the original patent to new therapeutic areas.

The inventive concept involves conjugating hydrophobic Auristatin F compounds to targeting moieties specific to disease-associated antigens in neurodegenerative and infectious diseases. These conjugates enable targeted delivery of the Auristatin F compound to affected tissues, enhancing efficacy and reducing off-target effects. The conjugates can be formulated for various administration routes, including ocular administration for retinal degenerative diseases. The inventive concept encompasses a range of diseases, including neurodegenerative diseases, such as Alzheimer's and Parkinson's, and infectious diseases, such as bacterial and viral infections.

The novelty of the present inventive concept lies in the application of hydrophobic Auristatin F compounds to novel therapeutic areas, including neurodegenerative and infectious diseases, and the use of targeting moieties specific to disease-associated antigens. This inventive step is non-obvious, as the original patent did not disclose or suggest the use of these compounds in these therapeutic areas.

Alternative embodiments of the inventive concept include the use of different targeting moieties, such as antibodies, peptides, or small molecules, and varying the linker chemistry to optimize conjugate stability and efficacy. Additionally, the inventive concept can be adapted for use in other diseases, such as cardiovascular diseases, by selecting targeting moieties specific to disease-associated antigens.

The present inventive concept has significant commercial potential in the treatment of neurodegenerative and infectious diseases, which are major unmet medical needs. The market for targeted therapies in these areas is substantial, with potential applications in both human and veterinary medicine.