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Designing a turnkey orphan drug program

The orphan drug designation is a vital regulatory pathway, responsible for a substantial portion of all new approvals at the FDA. Just how are today's drug developers navigating the incentives and challenges that come with following this route?

In this Endpoints News webinar, we talk to four experts who represent some of the key constituencies when developing a modern orphan program - from early research through approval. What are the latest approaches in achieving proof-of-concept in orphan populations? How will your program evolve between IND through NDA? What are regulators looking for? And how do we do all of this while keeping a laser focus on the patient?

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Cornell Stamoran

Cornell Stamoran

VP: Strategy & Corporate Development, Catalent

Cornell Stamoran is a life sciences industry strategist and has spent nearly 25 years engaged with the health care industry, including 20 years’ in advanced drug delivery. Cornell has directly participated in the development of drug design-enabled adherence and outcomes enhancement strategies for more than 150 branded drugs over the last 10 years. He holds several professional certifications and is a graduate of the University of Michigan.

Ethan Perlstein

Ethan Perlstein

Chief Scientific Officer, Christopher & Dana Reeve Foundation

Dr. Perlstein manages the Reeve Foundation’s basic and translational research portfolio and leads venture philanthropy investments in pursuit of the Foundation’s ultimate goal, curing spinal cord injury. Ethan has nearly two decades of experience in biomedical research and over five years in biotech company formation and executive leadership.
Ethan holds a doctorate in molecular and cell biology from Harvard University and was a Lewis-Sigler Fellow at Princeton University from 2007-2012. 

In 2014, Ethan founded Perlara PBC, the first biotech public benefit corporation with a legal mandate to co-develop drugs in collaboration with families and communities affected by genetic diseases. During that time, Ethan served as a member of the Medical and Scientific Advisory Board of Global Genes, the leading rare disease community-led advocacy organization. Prior to becoming a biotech entrepreneur, Ethan conducted and led cross-disciplinary academic research in genetics, cell biology, neuroscience, pharmacology, and drug discovery.

Terry Jo Bichell

Terry Jo Bichell

Founder and Director, COMBINEDBrain

Terry Jo Vetters Bichell is the Founder/Director of COMBINEDBrain, a patient-advocacy led organization formed in 2019 with a mission to identify outcome measures and biomarkers for neurodevelopmental disorders. Dr. Bichell has a B.A. from Johns Hopkins University and a bachelors in nursing from St. Louis University. Dr. Bichell earned a masters degree in public health and a certificate of nurse-midwifery from Boston University, where she was a Maternal and Child Health Leadership Scholar.

She worked as a nurse-midwife in San Diego, and was a scientific advisor to the CASA Midwifery School in San Miguel de Allende, Mexico, until her fifth child, Lou, was diagnosed with Angelman syndrome in April, 2000. Soon after her son’s diagnosis, she was inspired by Dr. Art Beaudet at the first international Angelman conference in Tampere, Finland in July, 2000 to help move bench research into clinical trials. Dr. Bichell was on the team of investigators who launched the first Angelman clinical trial (Folic acid in 2001), the NIH-funded Natural History trials, and the Levodopa trial. Dr. Bichell earned a PhD in neuroscience from Vanderbilt University in 2016, studying gene-environment interactions in Huntington’s disease rodent models, She was the founding director of the Angelman Biomarkers and Outcome Measures Alliance (A-BOM) from 2016-2018.

Dr. Bichell has published studies on clinical aspects of Angelman syndrome as well as Angelman rodent models concerning Gabrb3 mutation, Ube3a gene expression and circadian rhythms. In addition, Dr. Bichell originated Alphabet Therapy, a method to teach literacy skills to children with Angelman syndrome. Dr. Bichell, her husband, Dr. David Bichell, and their son, Lou, reside on a small horse farm in Nashville, TN along with several musician/caregivers who help with Lou. Their four adult daughters (and grandson) visit often, in person or on face-time.

Josh Lehrer

Josh Lehrer

Chief Medical Officer, Global Blood Therapeutics

Dr. Lehrer joined GBT in October 2013 to lead the clinical development program for Voxelotor. He was appointed senior vice president, development, in July 2017 and chief medical officer in October 2019. At GBT, he oversees the clinical development and regulatory affairs organizations. He has led program strategy and execution for voxelotor (GBT440), bringing it from IND through NDA filing. Prior to GBT, Dr. Lehrer led clinical development programs through proof-of-concept at Genentech in multiple indications and held key roles in business development and oversaw cardiovascular safety.

Dr. Lehrer has served as adjunct faculty in the Division of Cardiology at Stanford School of Medicine. Dr. Lehrer holds an A.B. from Harvard College in biochemistry and an M.Phil. in biological sciences from Cambridge University. Dr. Lehrer completed medical school and training in internal medicine at the University of California San Francisco (UCSF) and cardiology specialty training and postdoctoral research at Stanford University School of Medicine.

Arsalan Arif
moderator

Arsalan Arif

Founder & CEO, Endpoints News

Arsalan Arif is a news media entrepreneur who set out in 2015 to build his vision of an independent biotech news company at Endpoints News.