Sarepta Therapeutics, Inc. [PDF]
Sarepta Therapeutics, Inc. (SRPT) is a commercial-stage biopharmaceutical company focused on RNA targeted therapeutics, gene therapy and other genetic medicine approach for treating rare neuromuscular diseases. The company has been primarily focused on drug candidates for Duchenne Muscular Dystrophy (DMD), a rare, inherited disorder, primarily affecting young males and defined by progressive muscular weakness. It is caused by the absence of the protein dystrophin, which connects, strengthens and protects muscle fibers as they contract and relax. DMD is the most common type of muscular dystrophy. 20,000 children are diagnosed with DMD globally each year. The company has three commercial-stage DMD therapeutics, three clinical-stage DMD therapeutic candidates and four clinical-stage candidates for other indications.
Cidara Therapeutics, [PDF]
Cidara Therapeutics, Inc. (CDTX) is developing a new generation of therapeutics with a novel approach to treating solid tumor cancers made possible by the company’s Cloudbreak® Platform. The treatments link an anti-cancer drug with a fragment from the Fc region of an antibody. The resulting Drug Fc Conjugates (DFCs) interact with Fc cell surface receptors to mediate adenosine signaling, a portion of the innate immune system that suppresses immune response to a tumor and can hinder anti-tumor immunity. CDTX’s first oncology drug-Fc conjugate CBO-212, targeting CD73, contributes to immune evasion in solid cancers by flooding the tumor microenvironment with adenosine.