Monthly Gene Therapy Business Review - Special Issue

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BIO International Convention, Boston MA, June 4-7, 2018

BIO is back in Boston to celebrate 25 years of innovation.  RxC International's Rich Tinsley and Jim Hoyes will be in attendance.  We hope to meet you there. It's a beautiful time of year in Boston, MA.

This year's conference is sure to be appealing to the gene therapy community for a host of reasons.  We identified more than 20 different sessions dedicated to gene therapy with over 300 attending organizations focused on gene therapy. Given individual time constraints and tight schedules please find a few gene therapy session recommendations. Note the dates and times, unfortunately there is some overlap between sessions so please prioritize. 

1) Gene Therapy Regulation

US Cell & Gene Therapy Regulatory Policy Update 

Tuesday 1:45 PM 2:45 PM in Regulatory Innovation Room 207, Level 2

Speakers: * Wilson Bryan, Director, Office of Compliance and Biologics Quality Center for Biologics Evaluation and Research at FDA * Manal Morsy MD,PhD,MBA, Senior Vice President, Head of Regulatory Affairs at Athersys, Inc * Anne-Virginie Eggimann, MSc, Vice President, Regulatory Science at bluebird bio, Inc. * Dr. Snehal Naik, PhD Associate Director, Global Regulatory Affairs at J&J * Dr. Melody Eble, PharmD, Director, Global Regulatory Affairs at J&J

Recent or planned updates to the regulatory framework for cell and gene therapy products and experience with the new RMAT (Regenerative Medicine Therapy Designation) will be reviewed and discussed.

Advancements and regulatory framework in human genome editingWednesday 4:15 PM 5:15 PM in Genome Editing Room 210B, Level 2

Speakers: * Carrie Wolinetz PhD, Acting Chief of Staff and Associate Director, Science Policy at National Institutes of Health * Dr. Jakob Reiser, Office of Tissues and Advanced Therapies Division of Cellular and Gene Therapies at FDA/CBER * Sandy Macrae, MB, PhD, CEO at Sangamo Therapeutics * Gideon Rose, Editor at Foreign Affairs

Human genome editing is an exciting technology making history by leveraging advanced science to bring forward new classes of innovative medicines to patients. We have seen many breakthroughs in recent times, and significant advancements in the development of genome editing treatments for patients. With this advancement, we have seen a heightened interest from around the globe in ways we can use responsibly genome editing technology to improve human health. This session will tackle the regulatory frameworks for therapies that make use of genome editing. A panel of distinguished experts will share their perspectives on navigating these regulatory requirements.

2) Gene Therapy Commercialization

Delivering on the Promise of Gene Therapy

Tuesday 2:30 PM 3:45 PM in Room 253ABC, Level 2

Speakers: * Michael Dombeck, Vice President, Business Development at Precision Biosciences * Alison Finger, Chief Commercial Officer at bluebird bio, Inc * John Pieracci PhD, Director, Gene Therapy Process Development at Biogen Corporation  * Bob Smith Senior Vice President, Global Gene Therapy Business at Pfizer, Inc. * Sandy Macrae MB, PhD, CEO at Sangamo Therapeutics * Steve Bagshaw, CEO at FUJIFILM Diosynth Biotechnologies

Gene therapies are transforming patients' lives in ways that were unimaginable twenty years ago. With recent scientific advances interest in the ways that gene therapy can improve human health are exploding, with the potential to treat and cure disease. With these advances come challenges and opportunities in scientific discovery and new approaches in delivering drugs. Panelists will tackle these issues and share what it takes to bring new types of medicines to patients.

Advances in Genome Editing - The Next Frontier in Human Health 

Wednesday 1:45 PM 2:45 PM in Genome Editing Room 210B, Level 2

Speakers: * Samarth Kulkarni, Ph,D CEO at Crispr Therapeutics * Lakiea Bailey Executive Director, WWBD at Sickle Cell Community Consortium * Ms. Katrine Bosley, CEO at Editas Medicine * Mike Certo, PhD, Head, Genome Editing at bluebird bio, Inc. 

There are thousands of genetic diseases for which there is no treatment. Genome editing is a groundbreaking technology which offers a solution for the development of therapies for said diseases. Scientific advances in human genome editing have proven that somatic cell genome editing has the potential to treat and cure genetic diseases for which there is no cure. Leading experts in the field of genome editing will discuss the most recent breakthroughs and the dynamic opportunities on the horizon.

Making Genetic Therapies a Reality for Patients 

Wednesday 4:15 PM 5:15 PM in Orphan & Rare Diseases Room 209, Level 2

Speakers: * David Williams, Chief Scientific Officer and Senior Vice President for Research at Boston Choldren's Hospital * Dr. Emmanuel Dulac, Chief Commercial Officer at Alnylam Pharmaceuticals * Jeffrey Walsh, Chief Financial and Strategy Officer at bluebird bio, Inc. * Dr. Kathy High PhD, President and Head of R&D at Spark Therapeutics * Sam Rasty PhD, Chief Operating Officer at Homology Medicines, Inc. 

Gene-based therapies are increasingly being sought for patients in part due to advanced technologies that can regulate, edit or correct genetic components. As these approaches are relatively new to drug development and even newer to treating patients, many questions still exist beyond scientific translation from the lab to the clinic. Attendees will have the opportunity to hear from a panel of experts as they discuss the importance of commercial, scientific and clinical working together, every step of the way, from development to commercialization, to bring a new treatment to patients.

3) Public Private Partnership

The Changing Face of Academic Technology Transfer 

Tuesday 4:15 PM 5:15 PM in Translational Research Room 206AB, Level 2

Speakers: * Howard Fillit, MD, Founding Executive Director and Chief Science Officer at The Alzheimer's Drug Discovery Foundation * Rubén D. Flores-Saaib, PhD, Dorector of Commercialization at UCSD * Wendi Yagnik PhD, Global Head of Academic Partnerships and External Innovation at Novartis Institutes for BioMedical Research, NIBR * Dr. Sadhana Chitale MBA,PhD, Director of Life Sciences/Technology Transfer at Office of Industrial Liaison at NYU  

With support and recognition from senior leadership, academic institutions are revamping how they conduct technology transfer and lowering barriers for the movement of new technologies to the market. Technology Transfer offices (TTOs) are willing to commit their own resources and put skin in the game to create more robust early stage assets especially in the area of drug development. Many TTOs now work much early in the process with their partners from biotech, pharma and foundations to raise non-dilutive capital and validate assets. In this session you will hear from representatives of leading academic institution, disease foundation, pharma and biotech on how working with TTOs has been a win-win for both sides.

4) Pricing, Access & Value-Based Payments

Developing a Path to Value-Based Payment for Transformative TherapiesTuesday 11:00 AM 12:00 PM in Patient Access & Reimbursement Room 210A, Level 2

Speakers: * Paul Levesque, Global PResident, Rare Disease at Pfizer * Marianne Hamilton, Research Director at Duke-Robert J. Margolis, MD, Center for Health Policy * Dr. Michael Sherman, Chief Medical Officer & Senior Vice President, Health Services at Harvard Pilgrim Healthcare * Ms. Katrine Bosley CEO at Editas Medicine, Inc. * Dr. Gregory Daniel, Deputy Director and Clinical Professor at Duke-Robert J. Margolis MD, Center for Health Policy, Duke University

Value-based payment (VBP) arrangements, which align payments to observed value and outcome in a population, can be critical tools to improve patient outcomes and lower overall healthcare costs. VBP creates a unique approach to financing curative or transformative therapies, which are highly effective but could also impose considerable costs and uncertainties on the U.S. healthcare system. Specifically, this panel will focus on special issues in applying VBP arrangements in the transformative therapy space, with a focus on distinct issues in "single administration" therapies, distinctive barriers to implementing new payment models within the single-administration therapy context, design elements of new payment models to address these needs and identify system, policy, and cultural changes needed to facilitate new payment models in this context.

About RxC International

Understanding the subtleties of gene therapies is critical for bringing these treatments to market. RxC International has extensive experience successfully commercializing and launching new drug products, developing innovative solutions, realizing a product's best potential, and working across organizations to achieve common goals.

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