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Rare Disease Symposium

July 18, 2018, 8:30-5
Iona College, Romita Auditorium in Ryan Library
715 North Ave, New Rochelle, NY 10801
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In the News:
Rare Disease "Hub" Draws Support from Drug Developers (Genetic Engineering News)
Westchester Biotech Project Aims to Become Rare Disease "Hub" (ClinicalOMICs)
- July 18 Symposium At Iona College Brings Together Global Thought Leaders to Discuss Medical Possibilities, Potential Breakthroughs (White Plains Patch)

Co-Chairs:
Antonio Molina-Pachon, Ph.D., Founder and CEO
       GenCo Pharma
David Zuckerman, Ph.D., Assistant Professor of Biology
       Iona College

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Agenda

Welcome
Michael Welling, Founder and Co-Chair
Partner, Meridian Risk Management
Antonio Molina-Pachon, Ph.D., Founder and CEO, GenCo Pharma
Joanne Gere, Executive Director, Westchester Biotech Project
Joseph Stabile, Ph.D., Dean, School of Arts and Sciences
Iona College

Basic Research’s Role
David Zuckerman, Ph.D., Assistant Professor, Biology, Iona College

Regulatory Challenges and Opportunities
Bethany Hills, Esq., Partner, Chair FDA Practice, Mintz Levin
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Novel Approaches to Clinical Research
Jules Mitchel, Ph.D., President, Target Health
Click for Slides and Video

Addressing Friedreich’s Ataxia:
Research, Development, Impact

David Bettoun, Ph.D., Vice President, Preclinical Development
Chondrial Therapeutics
Click for Slides and Video
Cathleen Lutz, Ph.D., Director, Mouse Repository,
Rare and Orphan Disease Center
The Jackson Laboratory
Moderator: Rick Huntress, Director, Business Development, 
Clinical and In Vivo Services
The Jackson Laboratory
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Understanding Gestational Choriocarcinoma (GC)
Rajendra Gharbaran, Ph.D., Assistant Professor
Bronx Community College (CUNY)
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Update: NSF Big Data Progress
René Bastón, Executive Director, Northeast Big Data Innovation Hub
Columbia University / National Science Foundation
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Community Partner Updates
3Sixty Pharma Solutions

Dalfoni Banerjee, CEO, 3Sixty Pharma Solutions, represented by 
Sheila Bose, Life Sciences Consultant, 3Sixty Pharma Solutions
Micronic

Jackson Hyde, General Manager, Micronic

Motivating Principals: A Family Story
Michael Welling, Co-Founder and Chair
Westchester Biotech Project
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Innovative Data Science Approaches to Accelerate Rare Disease Clinical Drug Development
Scott Schliebner, Senior Vice President, Center for Rare Diseases
PRA Health Sciences

Research-Based Biotech:
Regeneron & Rare Disease
Scott Mellis, Ph.D., Vice President, Early Clinical Science, Rare Disease
Regeneron
Justin Grindley, Ph.D., Senior Staff Scientist, Regeneron
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Rare Disease Research Hub, Who’s in?
Antonio Molina-Pachon, Ph.D., Founder and CEO, GenCo Pharma
Joanne Gere, Executive Director, Westchester Biotech Project
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A View through a Different Lens: Applying the Principles of Pediatric Palliative Care to the Treatment of Rare Genetic Diseases
Alvin Moyer, MD, Attending Physician
Elizabeth Seton Pediatric Center
Varun Doddapaneni, MD, Attending Physician
Elizabeth Seton Pediatric Center
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Update: Collaborations that Are Working
Alan Belicha, Ph.D., CEO, MitoPath
Director, Business Development,
NY Center of Excellence, Bioinformatics & Life Science
University of Buffalo
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Investors’ Perspective
Sergei Petukhov, DVM, Partner, Enso Ventures

Final Comments, Adjourn
Michael Welling, 
Founder and Co-Chair
Partner, Meridian Risk Management


Speaker Bios

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Dalfoni Banerjee, Principal Consultant & CEO, 3Sixty Pharma Solutions

Dalfoni Banerjee is an award-winning, solution-focused entrepreneur. Beginning her career over 25 years ago in academic, pharmaceutical, and biotechnology research laboratories, she went on to lead inspection-readiness efforts, drive clinical research and clinical operations, manage relationships between pharma and key opinion leaders, develop marketing strategies/tactics, and launch new drug products – across 12+ therapeutic categories.

Dalfoni is an invited speaker and moderator on a variety of topics, has authored and co-authored several publications, and has received over 30 awards.

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René Bastón, Executive Director, Northeast Big Data Innovation Hub
Columbia University / National Science Foundation

René Bastón has over 20 years of experience in innovation, alliance building, public/private collaborations, entrepreneurship, technology transfer and commercialization. He has spent most of his time at the intersection of science and business. From academia to management consulting, technology transfer, non-profits, and startups - the transfer of knowledge across sectors fascinates him. He spent years as a management consultant for systems integrator, Ernst & Young; has created a number of public/private partnerships; and has founded startups based on the transfer of university, NIH, and NIST technologies.

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Alan Belicha, Ph.D., CEO, MitoPath
Director, Business Development, University of Buffalo

Dr. Belicha is responsible for engaging with industry and developing partnerships on behalf of the New York State Center of Excellence in Bioinformatics and Life Sciences, New York State Center of Excellence in Materials Informatics, and Buffalo Institute for Genomics and Data Analytics.

Alan joined the Business Development team at Mount Sinai Innovation Partners towards the end of 2012 where he was responsible for engaging with biotech and pharma industry to increase sponsored research activities and for the evaluation and licensing of early stage technologies in the life sciences. During his time at Mount Sinai Innovation Partners he contributed to the licensing of ~$850M of preclinical stage assets.

Prior to his transition into business development role, Alan obtained Ph.D. in molecular tumor immunology at Roswell Park Cancer Institute and completed a postdoctoral fellowship at the Icahn School of Medicine at Mount Sinai in New York City, where he also worked as a consultant in an early-stage venture fund for several years. 

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David Bettoun, Ph.D., Vice President, Discovery and Preclinical Development
Chondrial Therapeutics

Dr. Bettoun is a drug discovery and development expert, with extensive experience in preclinical research and development, global research strategy, and startup management. He has over 17 years of experience, and a proven track record of achieving quantifiable results implementing effective and highly successful internal or partnered R&D programs to further drug development for both small molecules and biologics. He is passionate about providing groundbreaking approaches and solutions for early and mid-stage programs from target validation to IND-enabling studies. 

Prior to his position at Chondrial Therapeutics, Dr. Bettoun was Chief Scientist at HaRo Pharmaceutical, working on pediatric oncology indications. As well, he has worked with Eli Lilly, Merck, and GSK in preclinical discovery and lead optimization in oncology, inflammation, and tissue remodeling.

Varun Doddapaneni, MD, Attending Physician, Elizabeth Seton Pediatric Center

Dr. Doddapaneni received a B.A. in Psychology and an M.S. in Biology from New York University. He then received his medical degree from St. George's University School of Medicine. He completed an Internal Medicine Residency at Englewood Hospital and Medical Center in Englewood, NJ and completed a Hospice and Palliative Medicine Fellowship at Lincoln Medical Center in Bronx, NY. He is currently a Palliative Medicine Attending at Lincoln Medical Center.

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Joanne Gere, Co-Founder and Executive Director, Westchester Biotech Project

Coming from a number of years working on programs for discovery scientists and entrepreneurs, Ms. Gere co-founded the Westchester Biotech Project to stimulate transformative resources for researchers, engineers, and data scientists. By providing a flexible and enjoyable platform for collaborations and partnerships, the WBP listens for emerging challenges and opportunities affecting preclinical, translational, and clinical professionals. Planning is in the works for 2019 activities, including a dynamic asset mapping initiative, the Rare Disease Research Hub, and our newest Roundtable: Translating Data Science.

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Rajendra Gharbaran, Ph.D., Assistant Professor, Bronx Community College (CUNY)

Dr. Gharbaran is a broadly trained biologist with background in basic and biomedical research in developmental neurobiology and cancer biology. His previous research was in hematology oncology, specifically identifying biomarkers for high risk Hodgkin’s lymphoma. He has a broad range of biology research skill-set including molecular biology and gene cloning, mammalian cell and tissue culture, drug targeting and in-vitro drug assays, immunohistochemistry and immunofluorescence, live-cell imaging, confocal microscopy, among others. He also has expertise in systems pharmacology and bioinformatics.

His current research interest is in the identification of biomarkers for early detection of rare but aggressive cancers and developing better drug targets for improve patient outcome.

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Justin Grindley, Ph.D., Senior Staff Scientist, Regeneron

Dr. Grindley has a strong track record of research and an in-depth knowledge of developmental signaling pathways, stem cells and oncology. He has pharmaceutical industry experience in managing preclinical development of oncology bio-therapeutics, and a broad biology research skill-set encompassing molecular biology, tissue and organ culture, in-vivo tumor models, histology, imaging and cell-based assay development. As well, he has bioinformatics and data mining knowledge encompassing transcriptomics, proteomics, and comparative genomics. 
 

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Bethany Hills, Esq., Chair FDA Practice, Partner, Mintz Levin

Bethany Hills has dual specialties in FDA and health care law making her uniquely qualified to shepherd FDA-regulated companies through complex domestic and foreign compliance matters, and also to provide invaluable counsel regarding positioning, reimbursement, pricing and other strategic decisions. Bethany’s solid understanding of the current landscape and the relevant issues impacting the health care delivery system (collaborations, incentives, legal limitations, reimbursements, etc.) enable her to provide her diverse portfolio of clients with top-tier strategic advice regarding product launches and, thereby, boost their value proposition and ROI.

Dynamic and passionate about her work and clients,—who include American and foreign medical device manufacturers, life science and pharmaceutical companies, and hospitals, physicians, and collaborative delivery system organizations—Bethany provides excellent service with a great spirit of enthusiasm, energy, and collaboration to ensure successful results.

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Rick Huntress, Director, Business Development, Clinical and In Vivo Services
The Jackson Laboratory

At the nonprofit Jackson Laboratory, Rick Huntress works directly with researchers, sponsors, engineers, and data scientists to design and execute early stage drug efficacy evaluations. The Jackson Laboratory's mission is to discover precise genomic solutions for disease and empower the global biomedical community in the shared quest to improve human health.

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Cathleen M. Lutz, Ph.D., Senior Director, Mouse Repository and Orphan Disease Center
The Jackson Laboratory

Dr. Lutz has over 25 years of experience in the field of mouse genetics, and works with patient foundations to develop mouse models for the research community. The program at The Jackson Laboratory includes a growing collection of more than 10,000 unique strains, including over 1,700 live colonies for distribution to the scientific community.

Efforts to improve mouse resources for rare diseases include the generation of precise patient mutations and the development of strains for preclinical testing of therapeutics. Dr Lutz also oversees the In Vivo Pharmacology program which provide services in preclinical drug delivery and efficacy testing for biotech, pharmaceutical, and industry partners. The program specializes in multiple routes of drug delivery and in life and postmortem readouts, especially in the areas of physiology and histological readouts. 

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Scott Mellis, Ph.D., VP, Early Clinical Science, Rare Disease
Regeneron

Dr. Mellis received medical scientist training at Washington University School of Medicine and postgraduate training in Internal Medicine and Rheumatology at Columbia University College of Physicians and Surgeons. He joined Pfizer Pharmaceuticals Group in 1990 with a focus on Zithromax® (azithromycin), including research on B. burgdorferi in collaboration with academic colleagues.  Dr. Mellis joined Regeneron Pharmaceuticals in 2001, and led clinical development for ARCALYST® (rilonacept), a medication for an ultra-rare auto-inflammatory disease.  He then led Regeneron’s translational and precision medicine initiatives across the general medicine portfolio.  He is a co-founder of the Regeneron Genetics Center.  Dr. Mellis is now leading an effort to optimize Regeneron’s development of new medications for patients with rare diseases.

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Jules Mitchel, Ph.D., President, Target Health

Dr. Mitchel is President of  Dr. Mitchel brings over 25 years of experience both in the pharmaceutical industry and basic research. He has broad based pharmaceutical experience in drugs, biologics, devices and diagnostics including strategic planning, multiple IND, IDE, NDA, PMA and 510(k) submissions; participation in FDA meetings; management of multicenter clinical trials; data management; biostatistics and clinical study reports. Dr. Mitchel leads the team at Target Health to develop software tools to enable the “Paperless Clinical Trial.”

Target Health is a privately held New York City based full service e*CRO with staff dedicated to all aspects of Regulatory Affairs, Chemistry, Manufacturing and controls, Clinical Research, Biostatistics, Data Management and Strategic Planning. Target Health is committed to bridging Internet-based technology with the drug and device development.

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Antonio Molina-Pachon, Ph.D., Founder and CEO, GenCo Pharma

Dr. Molina-Pachon is an entrepreneur developing solutions for rare diseases and cancer. He is collaborating with the Westchester Biotech Project and international partners to develop a regional hub for researchers who are addressing the most complex, and promising, discoveries.

Since 1997 he has held many executive positions, including Director of Discovery, Director of Business Development, CSO, and CEO positions. He has been the founder of 6 companies, all of which are still running today. 

Alvin Moyer, MD, Attending Physician, Elizabeth Seton Pediatric Center

Dr. Moyer received a B.A in Neuroscience from Colgate University in Hamilton, NY and received his medical degree from Vanderbilt University in Nashville, TN.  He completed his pediatric training at the University of Rochester in Rochester, NY and hospice and palliative medicine training at Elizabeth Seton Pediatric Center and St. Vincent’s Hospital in New York City.  Since 2008, He has been an attending pediatrician and Director of palliative care at Elizabeth Seton Pediatric Center, now located in Yonkers, New York.

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Sergei Petukhov, DVM, Partner, Enso Ventures

Dr. Petukhov leads Enso's biotechnology investments and holds several board positions in Enso portfolio companies including Seres Therapeutics, Galera Therapeutics, Cavion, NeuroVia and Cellectar Biosciences. He has been the Managing Director at CLS Capital, worked at RUSNANO investment fund, and was an advisor at Kaye Scholer LLP for number of US pharmaceutical companies.

He has worked and studied at The Rockefeller University, The University of Edinburgh, The University of Cambridge, Moscow 1st Medical Academy, and Moscow State Academy of Veterinary Medicine and Biotechnology.

Scott Schliebner, Senior Vice President, Center for Rare Diseases
PRA Health Sciences

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Scott Schliebner is an innovative drug development executive with 25 years of clinical leadership experience across the CRO, biotech, and non-profit sectors. He is interested in disruptive and innovative strategies to accelerate clinical drug development, with a particular focus on orphan drugs and rare diseases. He strongly believes the current drug development paradigm is archaic and unsustainable and seeks to implement strategies to ensure clinical trials become an integrated healthcare option for all. 

He has specialties in clinical drug development, rare diseases, orphan drugs, patient-focused approaches, precision and personalized medicine, patient advocacy, clinical trials, and study design, among others.

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David Zuckerman, Ph.D., Assistant Professor, Biology, Iona College

Dr. Zuckerman's research focus is on the cell biology of the soil bacterium Myxococcus xanthusM. xanthus is a model social bacterium, and communities of these cells carry out  many complex functions that require coordinated activities. A better understanding of these features will help inform us how individual cells coordinate their behaviors within larger communities, and the overall cellular organization.

His primary research interests include examining cell movement across surfaces and determining of how protein structures within the cell contribute to the shape and behavior of the bacteria.


Event Highlights: Rare Disease Symposium

  • Learn about the latest research and its implications for new diagnosis and treatments
  • Meet with experts involved in the Rare Disease field
  • Gain insights into the future of Rare Disease diagnosis and treatment
  • Learn about the regional organizations that have commitments to the field

We bring together the diverse players involved in the Rare Disease space in a collaborative and educational spirit. Discussions will be forward-thinking, focused on the continued development of new treatment and diagnostic options for the many individuals in the world living with a rare disease. Westchester County's research capacity is primed for continued growth in the coming years, and it is important that Rare Disease research continues to be an area of focus.

Come share your research and its potential impact, discuss current collaborative endeavors in the space, and meet like-minded professionals working toward a common goal. Above all, just bring your energy and a desire to contribute to furthering an important cause.