| Name | Title | Contact Details |
|---|
Enzyvant is also advancing the development of RVT-802, an investigational tissue-based biologic therapy for the potential treatment of primary immune deficiency associated with complete DiGeorge Syndrome. RVT-802 has been granted orphan drug designation, Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and pediatric rare disease designation by the U.S. Food and Drug Administration. Enzyvant anticipates a potential BLA filing for RVT-802 in the first half of 2018. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.
Lycera is a biopharmaceutical company that seeks to uncover new small molecule therapies for the treatment of autoimmune diseases and cancer. Our approach builds on an in-depth understanding of the orchestration of immune response in disease progression. When working correctly, the immune system can respond aggressively and with precise targeting to eliminate infectious pathogens or cancer cells. Incorrect regulation can cause the immune system to attack healthy tissue as foreign, supporting onset and progression of autoimmune disease while disruption of anti-tumor immune responses can shield cancer cells from the immune system and allow them to proliferate. Lycera is rapidly building a portfolio of selective immune modulators with mechanisms we believe will translate into improved outcomes for patients. With senior leadership and a team of scientists who represent the best of their fields, Lycera has the expertise and insight necessary to develop innovative therapies able to restore immune balance and significantly improve treatment for patients affected by these diseases around the world. Lycera product development programs are enhanced by our established global relationships with many leading chemists, scientists and clinical researchers. We are currently advancing novel small molecule compounds from distinct, yet complementary, areas of research, including immune metabolism, cell signaling and immune cell differentiation.
Rubius Therapeutics is a biopharmaceutical company pioneering a new era of cellular medicines. Our proprietary RED PLATFORM™ was designed to genetically engineer and culture Red Cell Therapeutics™ that are selective, potent and ready to-use cellular therapies for the potential treatment of several diseases across multiple therapeutic areas. Our initial focus is to advance RCT™ product candidates for the treatment cancer and autoimmune diseases by leveraging two distinct therapeutic modalities — potent cell-cell interaction and tolerance induction. We are currently enrolling patients in a Phase 1/2 clinical trial of RTX-240; the trial has two Phase 1 arms enrolling adult patients with advanced solid tumors: an ongoing monotherapy dose escalation arm in adults with relapsed/refractory or locally advanced solid tumors and a combination therapy dose escalation arm with pembrolizumab in adults with relapsed/refractory or locally advanced solid tumors. In April 2022, the combination arm with pembrolizumab was expanded to focus on non-small cell lung cancer and renal cell carcinoma patients. We are also enrolling patients in a Phase 1/2 clinical trial of RTX-224 for the treatment of patients with certain advanced solid tumors, including non-small cell lung cancer, cutaneous melanoma, head and neck squamous cell carcinoma, urothelial (bladder) carcinoma and triple-negative breast cancer.
Zynerba (NASDAQ: ZYNE) is dedicated to improving the lives of people with rare and near rare neuropsychiatric disorders where there is a high unmet medical need by pioneering the development and commercialization of next-generation pharmaceutically-produced cannabinoid therapeutics formulated for transdermal delivery. Cannabinoids interact with specific receptors throughout the body to produce pharmacologic effects, primarily in the CNS and immune systems. Transdermal delivery has the potential to reduce adverse effects associated with oral dosing because it allows the drug to be absorbed through the skin directly into the bloodstream, avoiding the gastrointestinal tract and lessening the opportunity for GI-related adverse events. It also avoids first-pass liver metabolism, potentially enabling lower dosage levels of active pharmaceutical ingredients with a higher bioavailability and an improved safety profile. Zynerba`s lead patent-protected product candidate in clinical development is ZYN002: ZYN002 is the first and only pharmaceutically-produced cannabidiol (CBD) formulated as a permeation-enhanced gel for transdermal delivery. Through a proprietary combination of our pharmaceutically-produced CBD and permeation enhancers, we believe we can effectively deliver CBD through the layers of the epidermis and into the circulatory system. The company is currently assessing ZYN002 in Fragile X syndrome and certain adult and pediatric refractory epilepsies. These include adult refractory focal epilepsy and developmental and epileptic encephalopathies (DEE), a heterogeneous group of rare and ultra-rare epilepsy syndromes associated with severe cognitive impairment and behavioral disturbances.
Mirus Bio is a Madison, WI-based company in the Healthcare, Pharmaceuticals, and Biotech sector.