SPEAKERS

 

 

 

 

The Advisory Board

 

We’re proud to work alongside a stellar line up of Orphan Drug and Gene Therapy Professionals who have helped us curate the most up to date and relevant program for you.

World Leading Speakers

 

Bringing together industry leaders from across the orphan drug and cell and gene therapy industries.

 

Iolanda Arbiol, Directora, Malalties Minoritàries

Iolanda Arbiol | Directora | Malalties Minoritàries » speaking at Orphan Drug Congress

Diego Ardigo, R&D Rare Diseases Unit Head, Chiesi Farmaceutici SpA

Diego Ardigo | R&D Rare Diseases Unit Head | Chiesi Farmaceutici SpA » speaking at Orphan Drug Congress

Francis Arickx, Head Of The Directorate Reimbursement Of Medicines And Pharmaceutical Policy, NIHDI - INAMI Belgium

Francis Arickx | Head Of The Directorate Reimbursement Of Medicines And Pharmaceutical Policy | NIHDI - INAMI Belgium » speaking at Orphan Drug Congress

Annette Bakker, President, Childrens Tumor Foundation

Annette Bakker | President | Childrens Tumor Foundation » speaking at Orphan Drug Congress

David Baram, Chief Executive Officer, Emendo Biotherapeutics

David Baram | Chief Executive Officer | Emendo Biotherapeutics » speaking at Orphan Drug Congress

Oswald Bentinck, Vice President And Head Of Market Access EMEA, AveXis Inc

Oswald Bentinck | Vice President And Head Of Market Access EMEA | AveXis Inc » speaking at Orphan Drug Congress

Carol Blount, NHS Partnerships Director, British Generic Manufacturers Association (BGMA)

Carol Blount | NHS Partnerships Director | British Generic Manufacturers Association (BGMA) » speaking at Orphan Drug Congress

Axel Boehnke, Director Market Access Eu North, PTC Therapeutics

Axel Boehnke | Director Market Access Eu North | PTC Therapeutics » speaking at Orphan Drug Congress

Thomas Bols, Head of Government Affairs and Public Policy, EMEA & APAC, PTC Therapeutics

Thomas Bols | Head of Government Affairs and Public Policy, EMEA & APAC | PTC Therapeutics » speaking at Orphan Drug Congress

Philip J Brooks, Program Director, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)

Philip J Brooks | Program Director, National Center For Advancing Translational Sciences - Ncats | National Institutes of Health (NIH) » speaking at Orphan Drug Congress

Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)

Anna Bucsics | Project Advisor | Mechanism of Coordinated Access to orphan medicinal products (MoCA) » speaking at Orphan Drug Congress

Gorana Capkun, Head Rwe Enablement, Novartis Oncology

Gorana Capkun | Head Rwe Enablement | Novartis Oncology » speaking at Orphan Drug Congress

Vittoria Carraro, Senior Government Affairs Manager, EUCOPE

Vittoria Carraro | Senior Government Affairs Manager | EUCOPE » speaking at Orphan Drug Congress

Manuel Carrondo, Director, iBET: Instituto de Biologia Experimental e Tecnológica

Manuel Carrondo | Director | iBET: Instituto de Biologia Experimental e Tecnológica » speaking at Orphan Drug Congress

Nathalie Cartier-Lacave, Research Director, Inserm

Nathalie Cartier-Lacave | Research Director | Inserm » speaking at Orphan Drug Congress

Giacomo Chiesi, Head of Global Rare Diseases, Chiesi Farmacutici, S.p.A.

Angela Columbano, Head Of Business Development And Partnership, Genethon

Angela Columbano | Head Of Business Development And Partnership | Genethon » speaking at Orphan Drug Congress

Denis Costello, Executive Director, CML Advocates Network

Denis Costello | Executive Director | CML Advocates Network » speaking at Orphan Drug Congress

John Crowley, Chief Executive Officer, Amicus Therapeutics Inc

John Crowley | Chief Executive Officer | Amicus Therapeutics Inc » speaking at Orphan Drug Congress

Omar Dabbous, Vice President, Health Economics And Outcomes Research, Novartis Gene Therapies

Omar Dabbous | Vice President, Health Economics And Outcomes Research | Novartis Gene Therapies » speaking at Orphan Drug Congress

Andeleeb Dahy-Al Mobaraq, Regulatory Affairs Director, Astellas Pharma

Andeleeb Dahy-Al Mobaraq | Regulatory Affairs Director | Astellas Pharma » speaking at Orphan Drug Congress

Doug Danison, VP, Access Value and Evidence Strategy, bluebird bio

Doug Danison | VP, Access Value and Evidence Strategy | bluebird bio » speaking at Orphan Drug Congress

Carla Deakin, Programme Director - Commercial And Managed Access, NICE

Carla Deakin | Programme Director - Commercial And Managed Access | NICE » speaking at Orphan Drug Congress

Graciana Diez-Roux, Chief Scientific Officer, Telethon Institute of Genetics and Medicine

Graciana Diez-Roux | Chief Scientific Officer | Telethon Institute of Genetics and Medicine » speaking at Orphan Drug Congress

Toon Digneffe, Head of Public Affairs And Public Policy, Takeda

Toon Digneffe | Head of Public Affairs And Public Policy | Takeda » speaking at Orphan Drug Congress

Scott Dorfman, CEO, Odylia Therapeutics

Scott Dorfman | CEO | Odylia Therapeutics » speaking at Orphan Drug Congress

Karen Facey, Senior Research Fellow, University of Edinburgh

Karen Facey | Senior Research Fellow | University of Edinburgh » speaking at Orphan Drug Congress

Rute Fernandes, Head Of Rare Diseases Of Europe And Canada, Takeda

Rute Fernandes | Head Of Rare Diseases Of Europe And Canada | Takeda » speaking at Orphan Drug Congress

Vivian Fernandez, Director Of Patient Advocacy, Regenxbio Inc

Vivian Fernandez | Director Of Patient Advocacy | Regenxbio Inc » speaking at Orphan Drug Congress

Alison Finger, Chief Commercial Officer, bluebird bio

Alison Finger | Chief Commercial Officer | bluebird bio » speaking at Orphan Drug Congress

Lucilla Franchetta Ortelli, Global Patient Services Lead Multiple Sclerosis, Biogen

Lucilla Franchetta Ortelli | Global Patient Services Lead Multiple Sclerosis | Biogen » speaking at Orphan Drug Congress

Hans-Juergen Fuelle, Vice President, Head Global Regulatory Strategy, Novartis Gene Therapies

Hans-Juergen Fuelle | Vice President, Head Global Regulatory Strategy | Novartis Gene Therapies » speaking at Orphan Drug Congress

Traci Fulton, Senior Director, Project Management, Precision For Medicine

Traci Fulton | Senior Director, Project Management | Precision For Medicine » speaking at Orphan Drug Congress

Michela Gabaldo, Head Of Alliance Management, Fondazione Telethon

Michela Gabaldo | Head Of Alliance Management | Fondazione Telethon » speaking at Orphan Drug Congress

Maciej Gajewski, Executive Director, Head of International Government Affairs and Policy, Alexion Pharma GmBH, Switzerland

Maciej Gajewski | Executive Director, Head of International Government Affairs and Policy | Alexion Pharma GmBH, Switzerland » speaking at Orphan Drug Congress

Josie Godfrey, Independent, Duchenne

Josie Godfrey | Independent | Duchenne » speaking at Orphan Drug Congress

Wim Goettsch, Special Hta-Advisor, Zorginstituut

Wim Goettsch | Special Hta-Advisor | Zorginstituut » speaking at Orphan Drug Congress

Bernard Grimm, EuropaBio

Bernard Grimm |  | EuropaBio » speaking at Orphan Drug Congress

Marcus Guardian, Chief Operating Officer, EUnetHTA

Marcus Guardian | Chief Operating Officer | EUnetHTA » speaking at Orphan Drug Congress

Anthony Hall, Chief Medical Officer, Healx Limited

Anthony Hall | Chief Medical Officer | Healx Limited » speaking at Orphan Drug Congress

Rachel Haurwitz, President And Chief Executive Officer, Caribou Biosciences

Rachel Haurwitz | President And Chief Executive Officer | Caribou Biosciences » speaking at Orphan Drug Congress

Niklas Hedberg, Head Of Pharmacy, Tandvards och Lakemedelsformansverket

Niklas Hedberg | Head Of Pharmacy | Tandvards och Lakemedelsformansverket » speaking at Orphan Drug Congress

Christopher R Heery, Chief Medical Officer, Precision BioSciences

Christopher R Heery | Chief Medical Officer | Precision BioSciences » speaking at Orphan Drug Congress

Ramon Hernandez Vecino, Head Of Real World Evidence Data And Analytics Engagement Lead Randd, Sanofi

Ramon Hernandez Vecino | Head Of Real World Evidence Data And Analytics Engagement Lead Randd | Sanofi » speaking at Orphan Drug Congress

Christophe Hilbert, Senior Manager, Access, Value and Evidence Strategy, Europe, bluebird bio

Annie Hubert, Senior Director, European Public Policy, Alliance for Regenerative Medicine

Annie Hubert | Senior Director, European Public Policy | Alliance for Regenerative Medicine » speaking at Orphan Drug Congress

Wills Hughes-Wilson, Head Of Patient Access And Commercial Planning, Mereo Biopharma

Wills Hughes-Wilson | Head Of Patient Access And Commercial Planning | Mereo Biopharma » speaking at Orphan Drug Congress

Paul Humphrey, EUMEA Head for Patient Advocacy and Engagement, BioMarin

Paul Humphrey | EUMEA Head for Patient Advocacy and Engagement | BioMarin » speaking at Orphan Drug Congress

Carlo Incerti, Chairman of the Board, Genzyme, A Sanofi Company

Carlo Incerti | Chairman of the Board | Genzyme, A Sanofi Company » speaking at Orphan Drug Congress

Julian Isla, Data And Artificial Intelligence Resource Manager, Microsoft Corp

Julian Isla | Data And Artificial Intelligence Resource Manager | Microsoft Corp » speaking at Orphan Drug Congress

Adrian Jonas, Associate Director for Data & Analytics, NICE

Adrian Jonas | Associate Director for Data & Analytics | NICE » speaking at Orphan Drug Congress

Rachel Jones, Medical Director, swiich health

Thomas Kassberg, Chief Business Officer And Executive Vice President, Ultragenyx Pharmaceutical

Thomas Kassberg | Chief Business Officer And Executive Vice President | Ultragenyx Pharmaceutical » speaking at Orphan Drug Congress

Gregory Keenan, Senior Vice President, Head of Medical & Drug Safety, Avrobio

Gregory Keenan | Senior Vice President, Head of Medical & Drug Safety | Avrobio » speaking at Orphan Drug Congress

Ken Kengatharan, Managing Partner, Atheneos Ventures And Chief Executive Officer, Atheneos Ventures

Ken Kengatharan | Managing Partner, Atheneos Ventures And Chief Executive Officer | Atheneos Ventures » speaking at Orphan Drug Congress

Ken Kengatharan, Managing Partner, Atheneos Ventures And Chief Executive Officer, Atheneos Ventures

Ken Kengatharan | Managing Partner, Atheneos Ventures And Chief Executive Officer | Atheneos Ventures » speaking at Orphan Drug Congress

Robin Kenselaar, Senior Vice President And General Manager EMEA, Orchard Therapeutics

Robin Kenselaar | Senior Vice President And General Manager EMEA | Orchard Therapeutics » speaking at Orphan Drug Congress

Diane Kleinermans, Advisor To The Ministry Of Health And Social Affairs, Belgian Federal Government

Diane Kleinermans | Advisor To The Ministry Of Health And Social Affairs | Belgian Federal Government » speaking at Orphan Drug Congress

Mr Daan Kranenburg, Vice President, Expansion Markets and Commercial Operations, bluebird bio

Mr Daan Kranenburg | Vice President, Expansion Markets and Commercial Operations | bluebird bio » speaking at Orphan Drug Congress

Michelle Krishnan, Senior Medical Director, AveXis Inc

Michelle Krishnan | Senior Medical Director | AveXis Inc » speaking at Orphan Drug Congress

Akshay Kumar, Partner, Partners4Access

Akshay Kumar | Partner | Partners4Access » speaking at Orphan Drug Congress

Yann Le Cam, EURORDIS

Yann Le Cam |  | EURORDIS » speaking at Orphan Drug Congress

Friedhelm Leverkus, Director Health Technology Assessment And Outcomes Research, Pfizer

Friedhelm Leverkus | Director Health Technology Assessment And Outcomes Research | Pfizer » speaking at Orphan Drug Congress

Farzana Malik, Managing Partner, Cogience Sarl

Farzana Malik | Managing Partner | Cogience Sarl » speaking at Orphan Drug Congress

Matthew May, Patient Partnership Director, F. Hoffmann-La Roche Ltd.

Matthew May | Patient Partnership Director | F. Hoffmann-La Roche Ltd. » speaking at Orphan Drug Congress

Christeen Moburg, Head Of Patient Advocacy, Sangamo Therapeutics

Christeen Moburg | Head Of Patient Advocacy | Sangamo Therapeutics » speaking at Orphan Drug Congress

Lucia Monaco, Head Of Research Impact And Strategic Analysis, Fondazione Telethon

Lucia Monaco | Head Of Research Impact And Strategic Analysis | Fondazione Telethon » speaking at Orphan Drug Congress

Allison Morgan, Vice President, Clinical Development, Auxesia Orion

Allison Morgan | Vice President, Clinical Development | Auxesia Orion » speaking at Orphan Drug Congress

Paolo Morgese, EU Director Market Access & Member Relations, Alliance for Regenerative Medicine

Paolo Morgese | EU Director Market Access & Member Relations | Alliance for Regenerative Medicine » speaking at Orphan Drug Congress

Krishnan Nandabalan, President And Chief Executive Officer, InveniAI Corporation

Krishnan Nandabalan | President And Chief Executive Officer | InveniAI Corporation » speaking at Orphan Drug Congress

Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)

Alexander Natz | Secretary General | European Confederation of Pharmaceutical Entrepreneurs (Belgium) » speaking at Orphan Drug Congress

Elena Nicod, Research Fellow, Bocconi University

Elena Nicod | Research Fellow | Bocconi University » speaking at Orphan Drug Congress

Amer Omar, Head of Rare Disease Access & Reimbursement, Lupin

Amer Omar | Head of Rare Disease Access & Reimbursement | Lupin » speaking at Orphan Drug Congress

Dr Emanuele Ostuni, Head Of Europe, Cell And Gene Therapy, Novartis

Dr Emanuele Ostuni | Head Of Europe, Cell And Gene Therapy | Novartis » speaking at Orphan Drug Congress

Ana Palma, Senior Director Global Hta And Patient Access Lead, Sobi

Ana Palma | Senior Director Global Hta And Patient Access Lead | Sobi » speaking at Orphan Drug Congress

Dr Pan Pantziarka, Program Director, Anticancer Fund

Dr Pan Pantziarka | Program Director | Anticancer Fund » speaking at Orphan Drug Congress

Samantha Parker, Chief Patient Access Officer, Lysogene

Samantha Parker | Chief Patient Access Officer | Lysogene » speaking at Orphan Drug Congress

Detlev Parow, Head Pharmaceutical Department, D.A.K.

Detlev Parow | Head Pharmaceutical Department | D.A.K. » speaking at Orphan Drug Congress

Nina Pinwill, Acting Commercial Medicines Director and Head of Commercial Operations, NHS England

Nina Pinwill | Acting Commercial Medicines Director and Head of Commercial Operations | NHS England » speaking at Orphan Drug Congress

Caridad Pontes, Manager Of Pharmacotherapeutic Harmonization, Catalan Health Service

Caridad Pontes | Manager Of Pharmacotherapeutic Harmonization | Catalan Health Service » speaking at Orphan Drug Congress

Tom Pulles, Vice President And Head Of Medical Affairs And Patient Advocacy, Ultragenyx

Tom Pulles | Vice President And Head Of Medical Affairs And Patient Advocacy | Ultragenyx » speaking at Orphan Drug Congress

Ana Rath, Director, INSERM & Investigator of research project - Manager of technological platform - Coordinator of research network, Orphanet Romania

Ana Rath | Director, INSERM & Investigator of research project - Manager of technological platform - Coordinator of research network | Orphanet Romania » speaking at Orphan Drug Congress

Lior Raviv, Vice President Of Development, Pluristem Therapeutics

Frederic Revah, Chief Executive Officer, Genethon

Frederic Revah | Chief Executive Officer | Genethon » speaking at Orphan Drug Congress

Kieran Rooney, Vice President, Strategic Alliances And Licensing, Amryt Pharmaceuticals

Kieran Rooney | Vice President, Strategic Alliances And Licensing | Amryt Pharmaceuticals » speaking at Orphan Drug Congress

Mark Rothera, President And Chief Executive Officer, Orchard Therapeutics

Mark Rothera | President And Chief Executive Officer | Orchard Therapeutics » speaking at Orphan Drug Congress

Christopher M de M Rudolf, CEO, volv global

Christopher M de M Rudolf | CEO | volv global » speaking at Orphan Drug Congress

Sophie Schmitz, Managing Partner, Partners4Access

Sophie Schmitz | Managing Partner | Partners4Access » speaking at Orphan Drug Congress

Tamir Singer, Head Of Commercial Development, Specialised Commissioning, NHS England

Tamir Singer | Head Of Commercial Development, Specialised Commissioning | NHS England » speaking at Orphan Drug Congress

Owen Smith, Investment Director, 4Bio Capital

Owen Smith | Investment Director | 4Bio Capital » speaking at Orphan Drug Congress

Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer

Bob Smith | Senior Vice President, Global Gene Therapy Business | Pfizer » speaking at Orphan Drug Congress

Jayne Spink, Chief Executive Officer, Genetic Alliance U.K.

Jayne Spink | Chief Executive Officer | Genetic Alliance U.K. » speaking at Orphan Drug Congress

Katy Spink, Chief Operating Officer And Managing Partner, Dark Horse Consulting

Katy Spink | Chief Operating Officer And Managing Partner | Dark Horse Consulting » speaking at Orphan Drug Congress

Pietro Sternini, Head Of Market Access Europe, Atara Biotherapeutics

Magali Taiel, CMO, GenSight Biologics

Magali Taiel | CMO | GenSight Biologics » speaking at Orphan Drug Congress

Rick Thompson, Chief Executive Officer, Findacure

Rick Thompson | Chief Executive Officer | Findacure » speaking at Orphan Drug Congress

David Thomson, Associate Director, NICE

David Thomson | Associate Director | NICE » speaking at Orphan Drug Congress

David Thomson, Associate Director, NICE

David Thomson | Associate Director | NICE » speaking at Orphan Drug Congress

Lonneke Timmers, Expert, Secretary Scientific Advisory Board, Zorginstituut

Lonneke Timmers | Expert, Secretary Scientific Advisory Board | Zorginstituut » speaking at Orphan Drug Congress

Dr Josep Torrent-Farnell, Medicines Department, Catalan Health Service

Dr Josep Torrent-Farnell | Medicines Department | Catalan Health Service » speaking at Orphan Drug Congress

Sheela Upadhyaya, Rare Disease Expert, National Institute for Health and Care Excellence

Sheela Upadhyaya | Rare Disease Expert | National Institute for Health and Care Excellence » speaking at Orphan Drug Congress

Inneke Van De Vijver, Strategic Advisor - Reimbursement Pharmaceuticals, N.I.H.D.I.

Inneke Van De Vijver | Strategic Advisor - Reimbursement Pharmaceuticals | N.I.H.D.I. » speaking at Orphan Drug Congress

Sander Van Deventer, Executive Vice President, Research And Product Development, uniQure

Sander Van Deventer | Executive Vice President, Research And Product Development | uniQure » speaking at Orphan Drug Congress

Birgitte Volck, President, AVROBIO

Birgitte Volck | President | AVROBIO » speaking at Orphan Drug Congress

Lance Weed, Former VP US Operations, uniQure

Lance Weed | Former VP US Operations | uniQure » speaking at Orphan Drug Congress

Robert wyer, Innovation Director, swiich health

Loukas Xaplanteris, Global Value & Access Head Neuromuscular Diseases, Biogen

Loukas Xaplanteris | Global Value & Access Head Neuromuscular Diseases | Biogen » speaking at Orphan Drug Congress

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World Orphan Drug Congress

 

The World Orphan Drug Congress provides you with a one stop progressive scientific and strategic solution to the orphan drugs industry.

 

 

Contact us


To sponsor or exhibit contact:
Marc Rhys-Evans

marc.rhys-evans@terrapinn.com

To speak:
Wing-Yun Cheung

wing-yun.cheung@terrapinn.com