15th ICORD Conference 6th – 7th February 2023

(online conference – CEST Time Zone)

Gene and Cell Therapies: Hope and Realities of Advances in Regenerative Medicine
A Community Partnership to Expand Access to Gene and Cell Therapies
Day 1:  February 6, 2023:  Experiences of Patients and Health Care Providers
13:45 – 14:00 Welcome and Introduction to the Conference
Domenica Taruscio. President of ICORD and National Centre for Rare Diseases, Istituto Superiore di Sanità, Italy
Session 1
14:00 – 15:00
Managing Expectations and Education on Gene Therapy Treatments
Session Chairs: Cynthia Tifft and Manuel Posada
14:00-14:15 The Clinical Researcher Investigator – Fears, Feelings, and Concerns Prior to Initiating Therapy – Cynthia Tifft. Deputy Clinical Director. National Human Genome Research Institute. Director, Pediatric Undiagnosed Diseases Program. National Institutes of Health 
14:15-14:30 Patients and Family Perspectives and Experiences with Before, During and After Receiving Gene Therapy.  Niclas Flysjo and Jessica Lindqvist. Family
14:30-14:45 Clinical Nurse Experiences Before and After Treatment. Precilla D’Souza. National Institutes of Health Undiagnosed Diseases Program, Common Fund, National Institutes of Health
14:45-15:00 Panel Questions and Answers
Session 2
15:00 – 16:00
Novel Approaches to Gene Therapy and Scalability to Entire Population
Session Chairs: Emilio Roldan and Stephen Groft
15:00-15:15 Platform Vector Gene Therapy Pilot Project (PaVe GT). PJ Brooks, Acting Director, Division of Rare Diseases Research Innovation, NCATS NIH
15:15-15:30 Bespoke Gene Therapies and Shared Molecular Etiologies. PJ Brooks, Acting Director, Division of Rare Diseases Research Innovation, NCATS NIH
15:30-15:45 Strategies for advancing the story for families with n=1 disorders. David Adams, Co-Director NIH Undiagnosed Diseases Program, NHGRI, NIH
15:45-16:00 Panel Questions and Answers
16:00 – 16:15 Short Break
Session 3
16:15 – 17:15
Ethic issues and advances therapies.
Session Chairs: Mireia del Toro and Domenica Taruscio
16:15-16:30 South American vision of access and ethics on gene therapies. Graciela Sara Moya Catholic University of Buenos Aires
16:30-16:45 Ethical Issues Surrounding Expanded Access to Cell & Gene Therapy. Mats G. Hansson. Senior Professor of Biomedical Ethics. Centre for Research Ethics and Bioethics at Uppsala University, Sweden
16:45-17:00 Access to Therapies: A Family and Non-profit Perspective—Andrew McFadyen, Executive Director. The Isaac Foundation
17:00-17:15 Panel Questions and Answers
Session 4
17:15 – 18:15
Education and Training to Meet Community Needs and Managing Outcome Expectations.
Session Chair: Helene Cederroth and UDNI  and Domenica Taruscio
17:15-17:30 Staff who Treat Patients and Monitor Outcomes Some from Japan – Yukiko Nishimura. President, NPO ASrid, Japan
17:30-17:45 Perspectives and Needs of Patients/Families and Patient Advocacy Groups – Dr. Jesús Navarro Torres. Vicepresidente de la Asociación Iberoamericana de Enfermedades Raras o poco Frecuentes (ALIBER)
17:45-18:00 Perspectives and Needs of undiagnosed Patients/Families. Helene Cederroth Wilhelm Foundation and UDNI
18:00-18:15 Panel Questions and Answers
End of the day
Scientific and Regulatory Issues to Increase Community Access to Gene and Cell Therapies
Day 2:  February 7, 2023:  Regulatory Issues and Requirements
Session 5
14:00 – 15:00
Perspectives on Meeting Patient and Medical Needs for Gene and Cell Therapies
Session Chairs: Simon Day and Wendy White
14:00-14:15 Global Access to Orphan Products for Rare Diseases focused on AT – W. Gahl. Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, USA.
14:15-14:30 Research Advances and Successful Efforts; Future Research and Development Activities – Samantha Parker. Vice-chair of IRDiRC
14:30-14:45 Industry Perspectives on Regulatory Issues and Requirements – Kinnari Patel. President and COO of Rocket Pharmaceuticals
14:45-15:00 Panel Questions and Answers
Session 6
15:00 – 16:00
Increasing Global Access to Interventions
Session Chairs: David Pearce and Luis Alejandro Barrera
15:00-15:15 Moving from Clinical Trials to Treatment and Global Access – Marc Dooms. Senior orphan drug pharmacist, University Hospitals Leuven
15:15-15:30 Country/Regional Approaches to Manufacturing – Ramaiah Muthyala.  President, Indian Organization for Rare Diseases, Hyderabad, Telangana INDIA. Associate Professor. Experimental Clinical Pharmacology, University of Minnesota
15:30-15:45 Global Distribution, Supply, and Storage Systems – David Pearce. President of Innovation, Research & World Clinics, Sanford Health Scientist, Pediatrics and Rare Diseases Group. Professor, Department of Pediatrics, Sanford School of Medicine of the University of South Dakota
15:45-16:00 Panel Questions and Answers
16:00 – 16:15 Short Break
16:15 – 16:30 Summary of the IX International HTA Symposium “IMPACT OF EU HTA REGULATION ON RARE DISEASES AND ORPHAN DRUGS”
Rumen Stefanov. President-Elect of ICORD, Director of Institute for Rare Diseases, Bulgaria
16:30 – 16:40 Summary of the ICORD webinar “HOW CAN ONE HEALTH APPROACHES HELP RARE DISEASES?”.
Domenica Taruscio, President of ICORD and National Centre for Rare Diseases, Istituto Superiore di Sanità, Italy
16:40 Wrap up and summary
Stephen Groft, Cynthia Tifft, and Simon Day
17:00 End of the conference