Cristina Csimma

Cydan LLC operates as an orphan drug accelerator that identifies and de-risks programs. The firm's model evaluates programs for treating rare diseases with unmet medical need and is aimed at creating new companies to develop those therapies. The company was founded by James G. McArthur in 2013 and is headquartered in Cambridge, MA.

Vtesse, Inc. provides novel drugs for life-threatening rare diseases. It developed VTS-270, a modified form of cyclodextrin that has been evaluated in preclinical and clinical studies at institutes within the National Institutes of Health (NIH) to treat patients with Niemann-Pick Disease Type C (NPC). The firm is working with the NIH and other leading academic centers to advance clinical study of VTS-270 for NPC, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other LSDs. The company was founded by Ben Machielse in 2015 and is headquartered in Gaithersburg, MD.

Seneca Biopharma, Inc. operates as a biopharmaceutical company, which engages in the research, development, and commercialization of central nervous system therapies based on human neuronal stem cells and small molecule compounds. Its pipeline includes NSI-189 small molecule program, NSI-566 stem cell therapy program, and novel & proprietary chemical entity screening platform. The company was founded by I. Richard Garr, Karl Y. Johe, and Merrill Solomon in 1996 and is headquartered in Germantown, MD.

Exonics Therapeutics, Inc. develops therapies for neuromuscular diseases. It also develops gene editing technologies like CRISPR/Cas9 to permanently correct the majority of duchenne muscular dystrophy mutations. The company was founded by Eric N. Olson in February 2017 and is headquartered in Cambridge, MA.

T1D Exchange is a nonprofit organization that was established in 2010 with initial and ongoing support from the Leona M. and Harry B. Helmsley Charitable Trust. T1D Exchange is based in Boston, MA. The non-profit company is dedicated to improving outcomes for the entire T1D population through real-world evidence and clinical data collection and analysis. T1D Exchange is a leading provider of real-world evidence, identifying gaps in data and redefining best practices to improve the lives of those living with T1D. The company actively supports quality improvement and innovation through its quality improvement collaborative, patient registry, and data-oriented research services. David Walton has been the CEO of the company since 2018.

Idera Pharmaceuticals, Inc. is a clinical stage biopharmaceutical company, which focuses on the clinical development, and commercialization of drug candidates for both oncology and rare disease indications. The company was founded by Paul C. Zamecnik, Sudhir A. Agrawal, and James B. Wyngaarden on May 25, 1989 and is headquartered in Exton, PA.

Caraway Therapeutics, Inc. provides heathcare services. The company was founded by Timothy Harris, Wade Harper ,Ivan Dikic ,Tony Hyman and is headquartered in Cambridge, MA.

Forendo Pharma Oy provides novel pharmaceutical treatments for endometriosis and low testosterone levels. Its product fispemifene is the first selective estrogen receptor modulator designed only for men, which helps for the treatment of symptoms of low testosterone, in phase II clinical studies in men with secondary hypogonadism; fispemifene has normalized the low testosterone levels. Its 17HSD1-inhibitor focuses on endometriosis, is a potent and selective lead compounds demonstrated in vivo proof of efficacy in disease model of endometriosis. The company was founded by Risto Arvo Sakari Lammintausta in 2013 and is headquartered in Turku, Finland.

Syncona Ltd. is a closed-end investment fund. It engages in delivering returns from investments in long and alternative investment funds across multiple asset classes and targets. The company was founded on August 14, 2012, and is headquartered in St. Peter Port, the United Kingdom.