University of California

Yitzhak Tor

Jeffrey D Esko

Nathan Luedtke

Murray Edmond Goodman

Wenyong Tong

Tracy Baker

Andrea Fin

Lisa McCoy

Yao Li

Kristina Hamill

The disclosure provides cyclic dinucleotides that are useful as STING agonists, pharmaceutical compositions and vaccines comprising the cyclic dinucleotides, and methods of treating diseases and disorders using the cyclic dinucleotides, pharmaceutical compositions, and vaccines.

Cyclic dinucleotide compounds as sting agonists

This disclosure relates to intranasal administration of conjugates comprising guanidinylated aminoglycosides ('guanidinoglycosides') and a polypeptide (e.g., an enzyme, antibody, or polypeptide growth factor). For example, such administration methods are useful for delivering a polypeptide to the brain and/or cerebrospinal fluid. Such methods are useful for treating a lysosomal storage disease through intranasal administration of a conjugate comprising one or more guanidinoglycosides and an enzyme useful for treating a lysosomal storage disease.

Intranasal administration of guanidinylated aminoglycosides

Provided are transporters based on polymyxin B. Also provided are methods of using the transporters for intracellular delivery of cargo and methods of enhancing intracellular uptake of cargo.

Polymyxin derived cell penetrating scaffolds

Reagents and methods useful for the synthesis of conjugates comprising guanidinylated cyclic acetals are provided. Also provided are methods for increasing the cellular uptake of various therapeutic compounds and treatment modalities using these conjugates.

Assisted enzyme replacement therapy

Guanidinium derivatives for improved cellular transport

Provided are reagents and methods useful for the synthesis of guanidinylated compounds. Also provided are methods for assaying molecules, including guanidinylated molecules that modulate viral infection and replication.

Growing community of inventors

San Diego, CA, United States of America

Yitzhak Tor