About Abeona Therapeutics
Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases. Developing therapies for rare disease requires new approaches and strong collaboration between researchers, industry, regulators and patient groups. With expertise in Research and Design, integrated manufacturing and novel gene and cell therapy development, Abeona is uniquely poised to bring new treatments to patients in need. Abeona strives to deliver gene and cell therapies to people affected by disease by harnessing the promise of genetic medicine.
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company’s portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates.
About Catalyst Pharmaceuticals
Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare, debilitating, chronic neuromuscular and neurological diseases. Catalyst Pharmaceuticals’ mission is to improve the lives of patients with rare and ultra-rare diseases by partnering with patients to empower them to take an active role in their health care.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
About Spark Therapeutics
At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We have successfully applied our technology in the first gene therapy approved in both the U.S. and EU for a genetic disease, and currently have four programs in clinical trials. At Spark, we see the path to a world where no life is limited by genetic disease.
About University of Rochester Center for Health + Technology (CHeT)
Over the past generation, CHeT has re-shaped the conduct of clinical research and advanced knowledge to improve health for thousands, if not millions, of individuals. CHeT is leading the field in evaluating how to harness the tools and technologies of the early 21st century to extend the reach and impact of research and clinical care. CHeT seeks to leverage Technology to Advance Care + Research by applying new tools and approaches in clinical trials to transform the design, assessment, and evaluation of novel therapies for neurological disorders. CHeT has been involved in over 111 clinical trials, enrolled over 40,000 research participants and has contributed to 7 FDA-approved treatments. CHeT collaborators and partners span the globe and include leading academic institutions, industry innovators in pharma, technology, not-for-profit foundations, advocacy groups, and the federal government.
About University of Rochester Clinical and Translational Science Institute (CTSI)
The University of Rochester Clinical and Translational Science Institute (CTSI) develops, demonstrates and disseminates methods and approaches to advance translational research, by providing education and training, supporting transdisciplinary teams, improving quality and efficiency, and engaging community stakeholders. Since its inception in 2006, the CTSI has provided pilot funding and training that helped researchers and students secure approximately $58 million in additional external funding to advance their studies.
About University of Rochester Del Monte Institute for Neuroscience
The Del Monte Institute for Neuroscience brings together faculty across the University of Rochester who are engaged in neuroscience research. The central purpose of the Del Monte Institute is to reduce the burden of neurological and neuropsychiatric disease in our society through development of basic scientific knowledge and of novel therapeutic approaches. The Institute exists to recognize new and emerging opportunities, to prosecute an aggressive innovative basic and translational research agenda, to build team science that tackles major problems from multi-methodological and multi-scalar perspectives, to tackle fundamental neuroscientific questions that simply cannot be broached at the level of an individual laboratory or investigator, questions that demand concerted multi-investigator teams.
About University of Rochester Department of Neurology
The University of Rochester Department of Neurology is committed to providing quality patient care, conducting research to generate knowledge that will alleviate human suffering through prevention and treatment of disease and educating physicians in accordance with highest professional standards. UR Neurology has an incredible team of neurologists, scientists, nurses, social workers, neuropsychologists, therapists, trainees and staff who’s focus is making a difference in the lives of people with a neurological disorder.
Erika Augustine MD, MS
Tauna Batiste MS
Alison Bateman-House PhD, MPH, MA
Sharon Begley BA
Derek Bowen BS
PJ Brooks PhD
Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences, NIH
Wilson Bryan MD
Mona Chitre PharmD
Danielle Edwards BA
Emily Farkas PA-C
Tanya Fischer MD, PhD
Jayne Gershkowitz BA
Martin Graham PhD
David Jacoby MD, PhD
Karl Kieburtz MD, MPH
Jinkuk Kim PhD
Nikki Marinsek PhD
Rachel McMinn PhD
Elissa Orlando MPA
Sean Nicholson PhD
Traci Schilling MD
Benjamin Schlatka MBA
Scott Steele PhD
Marshall Summar MD
Holly Tabor PhD
Nancy Yu BS
Dina Zand MD