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EVENT DATES
Apr 2018
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Venue

Gaylord National Resort & Convention Center 201 Waterfront Street National Harbor , Maryland 20745
Tel: (301) 965-4000
Website
Google Map

Orphan Drug Congress USA 2018

Attendees

1,000

Exhibitors

17

Register

$2,600 - $3,530

Overview

Event Overview:

World Orphan Drug Congress USA focuses on the most pressing challenges and opportunities to bring rare disease therapies to patients faster.

World Orphan Drug Congress USA has evolved from a one track conference focused on regulatory pathways, policy and incentives, to a global gathering of 1,000 leaders in orphan drugs from 38 countries, and over 135 presentations covering all aspects of orphan drug development and rare disease research.

World Orphan Drug Congress USA is truly a global gathering with representatives from the United States, Canada, Mexico, Ireland, England, Brazil, Italy, France Finland, South Africa, Japan, Sweden, India and Australia. 

Exhibitor Information:

Who Should Sponsor & Exhibit?

  • Pharma/Biotech 
  • Market Research consultants 
  • Specialty pharma distributors 
  • Data management
  • Equipment and tools vendors 
  • Technology providers
  • IT and software solutions 
  • CROs 
  • CMOs
  • Investment banks
  • Law firms
  • Risk advisers
  • Marketing & Communications Consultants 
  • Strategic Consultants 
  • Market Access Consultants

Who Will You Meet?

PHARMA

Head, SVP, VP, Director of:

  • Rare Disease Unit
  • R&D
  • Medical Affairs
  • Commercial
  • Marketing

BIOTECH

  • CEO
  • CSO VP of Business Development

UNIVERSITIES

  • Professors and Heads
  • Academic medical center chiefs
  • Payers & regulators

Exhibitor Prospectus and Kit

Agenda

Apr 26   

Apr 2608:20

Conference pass

Opening remarks

Keynotes

Scott Gray,Chief Executive Officer,Clincierge

Apr 2608:30

Conference pass

Keynote address: A new era in medicine- introducing gene therapy

Keynotes

What we’ve learned – lessons science has taught us as we create the path for the first gene therapy for an inherited disease in the U.S.

Staying true to our roots while preparing the patient, medical and policy maker communities for anew chapter in the retinal disorders textbook

Assessing the impact of potential one-time treatments like gene therapies for patients with rare diseases

Katherine High,President and Head, Research & Development,Spark Therapeutics

Apr 2608:55

Conference pass

Keynote address: Cell and gene therapy innovation and their role in the future of rare disease research

Keynotes

Current state of cell and gene therapies and why this field is becoming more and more significant

Looking into scalability and centralized approaches for cell and gene therapy development and commercialization

Novel approaches and how these therapies benefit the continuum of research for rare diseases

Hans Bishop,President and Chief Executive Officer,Juno Therapeutics

Apr 2609:20

Conference pass

Keynote address: Next- generation CAR T-cells to cure cancer - paving a new way for R&D, value-assessment and pricing and reimbursement of treatments for genetic diseases

Keynotes

Learnings from CAR-T development that can be applied to rare disease drug development

Will CAR therapy pricing impact pricing schemes of other orphan drugs and gene therapies?

Exploring aspects of these different therapeutic areas that share common goals, personalization and pricing challenges

Andre Choulika,Chairman & Chief Executive Officer,Cellectis

Apr 2609:45

Conference pass

Keynote CEO Panel: Gene therapy - a new paradigm for patients, new challenges for industry

Keynotes

Looking back at the commercial performance of the first generation of gene therapies- from proof of concept to the road to approval

How will gene therapy innovation look in the next 10 years?

Considering potential new value propositions for patients, payers and the health care system

Strengthening clinical development, exploring novel technologies while maintaining safety- what needs to be improved?

Moderator:Jude Samulski,Vice President, Gene Therapy,Pfizer

Tim Miller,President and Chief Executive Officer,Abeona Therapeutics

Karen Aiach,Chief Executive Officer,Lysogene

Frederic Revah,Chief Executive Officer,Genethon

Ken Mills,Chief Executive Officer,ReGenX Biosciences Llc

Mark Rothera,President and Chief Executive Officer,Orchard Therapeutics

Apr 2610:30

Conference pass

CANADA: Is there a roadmap to rare diseases and orphan drugs in Canada?

Patient Groups Seminar

Durhane Wong-Rieger,President and Chief Executive Officer,Canadian Organization For Rare Disorders

Apr 2610:50

Conference pass

SOUTH AFRICA: Overcoming the lack of medical infrastructure, research and support to improve the quality of life of rare disease patients in South Africa

Patient Groups Seminar

Kelly Du Plessis,Chief Executive Officer,Rare Diseases South Africa

Apr 2611:10

Conference pass

COLOMBIA: Reviewing dynamics of public policies for orphan drugs in Latin America – perspectives of patients and access situation

Patient Groups Seminar

Angela Chavez,President,Colombia Rare Disorders Society

Apr 2611:25

Conference pass

The evolution of a partnership to help decision making and improve patient access to investigational medicines

Clinical Development & Regulatory Track

Exploring ethical issues surrounding access to investigational products before FDA approval

Working with patient groups to help demystify pre-approval access and create community-specific resources

Developing a group approach to ethical decision medicine – pharma and ethicists working together

Alison Bateman House,Professor,New York University Langone Medical Center

Beverly Harrison,Head, Patient Support, Janssen Office of the Chief Medical Officer,Janssen Research and Development Llc

Apr 2611:25

Conference pass

Collaborative mission-driven approach to developing innovative potential new treatments for unserved and underserved patient communities

Commercial Track

Addressing industry challenges of too much good science and insufficient resources to fully advance pipelines

Aligning biopharma innovators with investigators, patient groups, and funders to advance promising investigational therapies

Concept in Action: Big Pharma expertise and capabilities leveraged to enable company launch pipeline of four mid- and late -stage clinical assets, enabling strong Series A funding

Lara Sullivan,President and Founder,SpringWorks Therapeutics

Apr 2611:25

Conference pass

CANADA: Orphan drug evaluation and decision making in Canada in the context of international HTA decisions

Global Market Access Track

Orphan drug evaluation at CADTH to reimburse, reimburse with clinical criteria/conditions and not reimburse

HTA challenges when there is no regulatory framework for orphan drugs in Canada and no international consensus for OD

Balancing evidence requirements with treatment’s cost

Trevor Richter,Director, Common Drug Review and Optimal Use,CADTH

Apr 2611:25

Conference pass

Genetic testing for patient engagement – shortening the diagnostic odyssey and connecting patients to research and treatments

Gene Track

No-cost genetic testing programs support patients and clinicians to gain an accurate diagnosis quicker by removing barriers to genetic testing

Drive patient identification, expedite trial enrollment and accelerate time to market for new therapeutics

Create engaged patient communities that partner biopharma and patient advocacy groups through Patient Insights Networks (PIN)

Moderator:Daniel Anderson,Head of Commercial Partnerships,Invitae

Mark Dant,Executive Director,The Ryan Foundation

Jordanna Mora,Associate Director of Patient Advocacy and Engagement,Alnylam Pharmaceuticals

Catherine Pajak,Senior Director, Marketing, North America, Lysosomal Storage Disorders,BioMarin Pharmaceutical Inc.

Apr 2611:25

Conference pass

Empowered educated patients influencing pre-clinical development and working with industry to optimize drug discovery

Rare Disease Advocacy World

Patient groups role guiding research, disease understanding, supporting pre-clinical development and follow up experiments

Driving translational research and the influence on formulation, routes to delivery and overall clinical development

Effective partnerships between advocacy and industry to expand collaborations that can support enrollment and physician engagement

Christopher Missling,President and CEO,Anavex Life Sciences

Steven Kaminsky,Chief Science Officer,International Rett Syndrome Foundation

Apr 2611:25

Conference pass

VC Panel: Investing in the orphan drug space – derisking commercial models and driving biotechs to successful launches

Pitch & Partner

Assessing advanced therapies commercial opportunities – and risks – as the new frontier of investment in rare disease companies

Type of work and diligence to obtain comfort with the ultimate market opportunity

Assessing barriers to entry including therapeutic modality and IP

Regulators role in incentivizing or impeding the ability to develop products both domestically and abroad. What can be different?

Moderator:Brian Bronk,Head of External Innovation - Rare Diseases,Sanofi

Kush Parmar,Managing Partner,5A.M. Ventures

David Bonita,Private Equity Partner,Orbimed Advisors

David Mott,General Partner,N.E.A.

Apr 2611:30

Conference pass

BRAZIL: The challenges of Brazil’s third sector in the participation of health promotion, access and regulation of public policies in rare and chronic diseases

Patient Groups Seminar

Gustavo San Martin,AME - Amigos Múltiplos pela Esclerose

Apr 2611:50

Conference pass

The virtual opportunity in rare disease trials

Clinical Development & Regulatory Track

Solving specific challenges with virtual trials and their supporting technologies

Reducing patient burden through siteless data capture and evidence generation

Exploring the promise and pitfalls of telemedicine

Karen Kaucic,Senior Vice President, Early Development, Rare Disease and Pediatric Center of Excellence,PPD® Consulting

Apr 2611:50

Conference pass

Navigating the rare disease innovation ecosystem and partnering for the long-term

Commercial Track

Looking into new technology platforms and transformative new products

Nurturing relationships with academia, incubators, startups and biotechs to develop fruitful partnerships

From due diligence to alliance management, realizing the potential of scientific innovation, and going beyond ROI

Gregory Fond,Director, External Innovation, Rare Diseases,Sanofi

Apr 2611:50

Conference pass

ITALY: The Italian experience on market access for rare disease drugs

Global Market Access Track

What is needed to achieve regional access? How does Italy compare to other European countries?

Looking into new rules to gain ‘innovative status’

Opportunities to market access through a more formalized process for early access and compassionate use requests

Martine Zimmermann,SVP, Head of Global Regulatory Affairs,Alexion Pharmaceuticals

Apr 2611:50

Conference pass

Patient advocates taking a real stand in drug development: how the Cystic Fibrosis Foundation worked with biotech and pharma to find a cure

Rare Disease Advocacy World

Research funds and initiatives coming from grassroots advocacy and support, not from pharma

Uniting the CF community with drug developers through transparency and the pursuit of a common goal

Fueling motivation from all involved parties to continue with the co-development of drugs

What is the applicability of this model to other rare diseases?

Preston Campbell,President and Chief Executive Officer,Cystic Fibrosis Foundation

Apr 2611:50

Conference pass

INDIA: Challenges of patients with rare diseases in India and recent progress

Patient Groups Seminar

Harsha Rajasimha,Co Founder,Organization for Rare Diseases India

Apr 2612:10

Conference pass

CHINA: The global challenges of rare diseases and the role of China

Patient Groups Seminar

Kevin Huang,President,Chinese Organization for Rare Disorders

Apr 2612:15

Conference pass

Early development planning for orphan drugs: tasks, timelines, and takeaways

Clinical Development & Regulatory Track

Organizing orphan drug designations: Where do they fit in and how long do they take?

Understanding natural history studies: purpose, design, and obtaining meaningful data

Preparing for an IND/IMPD and related clinical studies

Angi Robinson,Executive Director, Strategic Development, Rare Disease & Pediatrics,Premier Research

Apr 2612:15

Conference pass

Development and execution of commercial strategies for clinical and commercial stage RNAi products for serious, life-threatening diseases

Commercial Track

Pre-launch strategies and building a rare disease commercial team from scratch

Launching successfully: different challenges, different approaches, same result

Achieving market access by placing a focus on prescribers, patients and payers

Global vs. local strategies

Mark Baglin,Vice President of Global Marketing,Alnylam Pharmaceuticals

Apr 2612:15

Conference pass

BENELUXA Belgium, Netherlands, Luxembourg and Austria joining forces to improve access and to orphan drugs and paving the way for more collaborations

Global Market Access Track

Joint collaboration to negotiate with pharmaceutical companies the price of orphan drugs

Looking into data exchange, sharing of registries and coordinated assessment of new technologies and innovation

Advantages in patient access and dossier submission for pharmaceutical companies

Diane Kleinermans,Dr,Belgian Federal Government

Apr 2612:15

Conference pass

Using patients’ genomic information to uncover pathogenic mutations linked to rare diseases

Gene Track

Leveraging next generation sequencing and genome interpretation for rare disease research

Combining data science and systems biology to accelerate rare disease diagnosis and therapeutic discovery

Searching for and decoding rare disease “genomic shields”

Fostering partnerships to advance rare disease therapies

Apr 2612:15

Conference pass

The post market patient and patient advocacy- Acknowledging that with approval, the work is not done

Rare Disease Advocacy World

How can patient groups keep encouraging orphan disease research and investments after approval?

Rare disease clinical development when approved treatments are available.

Opportunities to reduce delays to access prior to approvals

Post-marketing engagement to improve and expand patient access – engaging with multiple new stakeholders.

Working synergistically with industry counterparts to educate the physician community, delivery centers, and payers.

Apr 2612:15

Conference pass

Case study: Enzyvant’s approach to developing RVT-802, a novel tissue-based therapeutic for complete DiGeorge Syndrome

Pitch & Partner

Leveraging expedited regulatory pathways, including breakthrough designation and Regenerative Medicine Advanced Therapy (RMAT) designation

Collaborating with academic partners

Fostering advocacy relationships and aligning corporate and patient priorities

Developing a commercial model for a transformative, one-time therapy in the pre-launch phase

Alvin Shih,CEO,Enzyvant

Apr 2612:30

Conference pass

JAPAN: Collaboration with/among multi-stakeholders as an intermediary organization in Japan

Patient Groups Seminar

Yukiko Nishimura,President, NPO,Japan Patients Association, ASrid

Apr 2612:40

Conference pass

IRDiRC goals 2017-2027 – new research goals to accelerate rare disease therapies and diagnostics globally

Clinical Development & Regulatory Track

Spearheading patient diagnosis by having currently undiagnosable individuals enter a globally coordinated diagnostic and research pipeline

Working toward the approval of 1000 new rare diseases therapies focused mainly on diseases without approved options

Development of methodologies to assess the impact of diagnoses and therapies on rare disease patients

Christopher Austin,Director, National Center for Advancing Translational Sciences (NCATS),National Institutes of Health

Apr 2612:40

Conference pass

The role of commercial in early development through launch

Commercial Track

Leading market access strategies, KOL development, engagement and mapping out all scenarios for a drug’s entire life cycle prior to launch

Driving lifecycle expansion into other indications and geographies

Managing and growing global commercial teams in rare disease

Alex La Croix,Senior Director, Rare Genetic Disease Marketing,Agios Pharmaceuticals

Apr 2612:40

Conference pass

Market access challenges and opportunities for orphan and ultra-orphan drugs

Global Market Access Track

Looking into the global demand for an evidence-based approach toward orphan drug pricing and reimbursement

Current landscape on pharmaceutical practices: undertaking burden of Illness studies, focusing on baseline quality of life, and direct and indirect costs to evaluate orphan drugs and assist with reimbursement

Tools to overcome the challenges faced by industry when identifying relevant data sources to document the cost and burden of orphan diseases

Strengths and limitations of available methods of data capture

Thomas Goss,Senior Vice President,Boston Healthcare Associates Inc

Apr 2612:40

Conference pass

Validating and optimizing NGS-based diagnostics to improve diagnostic yield and clinical utility

Gene Track

Challenges in the field of genetic diagnostics and current technologies used to address them

Introducing the concept and importance of transparent, traceable, and comprehensive analytic validation of an NGS platform for clinical diagnostics

Patient examples which demonstrate the importance of high quality and comprehensive genetic diagnostics for improved outcomes for rare disease patients

Controlling costs by utilizing high quality WES derived sequencing data for rare disease patients

Tero-Pekka Alastalo,President, Chief Medical Officer,Blueprint Genetics

Apr 2612:40

Conference pass

Inclusion of patient reported outcomes (PROs) in rare disease trial design

Rare Disease Advocacy World

Capturing patients’ bona-fide needs, preferences, and value drivers through systematic investigation

Adoption of end- to-end patient evidence generation pathways to increase the probability of success

Enabling payers and providers to better understand patients’ values

Developing meaningful patient reported outcomes and producing further evidence to support approval

Apr 2612:40

Conference pass

Validive for the treatment of severe oral mucositis

Pitch & Partner

Chandler Robinson,CEO,Monopar Therapeutics

Apr 2612:50

Conference pass

JOTROL™ for treatment of MELAS syndrome, Friedreich's ataxia and MPS I

Pitch & Partner

Christer Rosén,Chairman & CEO,Jupiter Orphan Therapeutics, Inc.

Apr 2613:30

Conference pass

Lunch keynote address: Breakthrough therapies call for breakthrough processes to ensure sustainable patient access

Keynotes

Yann Le Cam,Chief Executive Officer,EURORDIS

Apr 2614:30

Conference pass

Temodex, a locally acting form of temozolomide, for treatment of glioblastoma

Pitch & Partner

Igor Lokot,Chief Executive Officer,Double Bond Pharmaceutical A.B.

Apr 2614:30

Conference pass

FINLAND: Rare diseases in a universal healthcare system – experiences in medical treatments and rehabilitation from patients’ perspective

Patient Groups Seminar

Katri Asikainen,Chairperson,Finnish Rare Diseases and Disabilities Organization (HARSO)

Apr 2614:31

Conference pass

ROUNDTABLE 1: Pediatric trials - reducing the financial, logistical and emotional burden for patients and families participating in pediatric trials

Keynotes

Apr 2614:32

Conference pass

ROUNDTABLE 2: Compassionate Use Programs - helping patients get access to medication, educating the community, physicians and investors to ensure safe access to experimental treatments

Keynotes

Andrew McFadyen,Executive Director,The Isaac Foundation

Apr 2614:33

Conference pass

ROUNDTABLE 3: Virtual Trials - enabling clinical trials to be more virtual, reducing distance times, overcoming geographic barriers and increasing data collection through the use of wearables

Keynotes

Scott Schliebner,Vice President, Scientific Affairs, Rare Diseases,PRA

Apr 2614:34

Conference pass

ROUNDTABLE 4: Expanded access - incorporating the patient advocacy perspective to develop a review process and a formalized procedure on expanded access

Keynotes

Jayne Gershkowitz,Chief Patient Advocate,Amicus Therapeutics

Apr 2614:35

Conference pass

ROUNDTABLE 5: BD & Forecasting - pricing and access considerations and understanding how to build the patient forecast based on available epidemiological information

Keynotes

Joseph Musumeci,Senior Advisor,BluePrint Orphan

Apr 2614:36

Conference pass

ROUNDTABLE 6: Strategy - patient engagement and pipeline building for the commercialization and access of rare disease products

Keynotes

Yvette Venable,International Public Affairs Lead,Kyowa Kirin International

Apr 2614:37

Conference pass

ROUNDTABLE 7: Gene therapy – new payment and financing models are needed to facilitate access

Keynotes

Francesca Cook,Director, Pricing and Market Access,RegenxBio

Apr 2614:38

Conference pass

ROUNDTABLE 8: Clinical development – When the sum is greater than the parts through academic, pharmaceutical and CRO collaboration in orphan disease drug development

Keynotes

Michael Murphy,Chief Medical & Scientific Officer,Worldwide Clinical Trials

Apr 2614:39

Conference pass

ROUNDTABLE 9: Managed Access Programs - How MAPs (pre-commercial supply to patients with unmet medical needs) can be a valuable component of launch strategy for orphan drugs

Keynotes

Robert Donnell,Director of Business Development,Durbin

Apr 2614:40

Conference pass

ROUNDTABLE 10: Gene therapy - Optimizing gene therapy clinical development through education, collaboration, patient input and involvement in advisory boards

Keynotes

Samantha Parker,Chief Patient Access Officer,Lysogene

Apr 2614:40

Conference pass

SFX-01 for the treatment of subarachnoid haemorrhage

Pitch & Partner

Stephen Franklin,Chief Executive,Evgen Pharma

Apr 2614:41

Conference pass

ROUNDTABLE 11: Scientific innovation - platform approaches to evaluate multiple genetic diseases at the same time

Keynotes

Philip J. Brooks,Program Director, Office of Rare Diseases Research,NCATS, NIH

Apr 2614:42

Conference pass

ROUNDTABLE 12: Epidemiology and forecasting - using epidemiological data to determine market viability, niche populations and commercial differentiation

Keynotes

David Lapidus,President,Lapidus Data

Apr 2614:43

Conference pass

ROUNDTABLE 13: Government affairs - developing a company’s footprint and contributions to impact policy, reimbursement and patient engagement

Keynotes

Betsy Ricketts,Senior Director, Government Affairs,Ultragenyx Pharmaceutical

Apr 2614:44

Conference pass

Roundtable 14: Patient identification - realizing the full potential of machine learning algorithms to search for undiagnosed patients with rare diseases

Keynotes

Nadea Leavitt,Predictive Analytics Principal, Real-World Insights,IQVIA

Stephanie Roy,Principal, Real-World Evidence Solutions,IQVIA

Apr 2614:45

Conference pass

Roundtable 15: Expanded Access - drug development strategy considerations for open label extension or expanded access protocols

Keynotes

Anne Cropp,Chief Scientific Officer,Early Access Care

Apr 2614:46

Conference pass

ROUNDTABLE 16: VC funding – successfully addressing investors interests and challenges when assessing companies of all sizes to guarantee funding of orphan drug biotechs

Keynotes

Ari Brettman,Principal,Clarus Ventures

Apr 2614:50

Conference pass

A bis-benzylisoquinoline alkaloid for use with chemotherapy for refractory and relapsed acute myeloid leukemia (AML)

Pitch & Partner

Saira Bates,Co Founder and Chief Executive Officer,Escend Pharmaceuticals Inc

Apr 2614:50

Conference pass

DENMARK: State of the art for the rare people in Denmark - Europa

Patient Groups Seminar

Birthe Byskov Holm,President,Rare Diseases Denmark

Apr 2615:10

Conference pass

IFB-088 a potential new therapeutic option to treat demyelinating charcot-marie-tooth diseases

Pitch & Partner

Philippe Guedat,Managing Director,In Flectis Bio Science

Apr 2615:10

Conference pass

SWEDEN: Rare Diseases Sweden – Challenges and opportunities

Patient Groups Seminar

Beata Ferencz,Project Manager,Rare Diseases Sweden

Apr 2615:20

Conference pass

5-(4-carboxymethyl-phenylazo)-2-hydroxybenzoic acid disodium salt (INN-108) for treatment of ulcerative colitis in pediatric/adult patients

Pitch & Partner

Jay Madan,President,Innovate Biopharmaceuticals Inc

Apr 2615:30

Conference pass

RP5063: A novel investigational drug to treat pulmonary arterial hypertension (PAH)

Pitch & Partner

Laxminarayan Bhat,Founder and Chief Executive Officer,Reviva Pharmaceuticals Inc

Apr 2615:30

Conference pass

UK: Resetting the clock – giving patients and families a voice in developing innovative therapies for rare diseases

Patient Groups Seminar

Alastair Kent OBE,Ambassador,Genetic Alliance U.K.

Apr 2615:40

Conference pass

From real world data to real world evidence: FDA’s perspective on the enhanced use of RWE

Clinical Development & Regulatory Track

RWE goals to have regulatory decisions incorporate data/evidence from settings that more closely reflect clinical practice

RWE expectations within 21st Century Cures and PDUFA VI

FDA’S experience with RWE, collaborations and the assessment of data fitness and standards

Impact of the inclusion of RWE in regulatory decision-making and the assessment of orphan drugs

Jacqueline Corrigan-Curay,Director, Office of Medical Policy, Center for Drug Evaluation and Research,FDA

Apr 2615:40

Conference pass

Value assessment is different for Rare Diseases, requiring new thinking to build consensus around access decisions

Commercial Track

Addressing divergent priorities from stakeholders in response to potentially curative gene and cell therapies, including physicians, regulators, patients, payers and manufacturers

Unblocking the affordability conundrum by bringing stakeholders together to rethink the measurement of value and how this impacts wider societal and economic factors

The need for tailored planning that differs from more mainstream disease areas given the very distinct role that patients and advocacy groups play in rare diseases

Walter Colasante,C.R.A. Charles River Associates

Apr 2615:40

Conference pass

AUSTRALIA: Orphan drug program reforms: changes in the eligibility criteria of orphan drug designations

Global Market Access Track

Expansion of the orphan disease prevalence threshold to allow more conditions to classify as orphan

Changes including the validity period of orphan drug designation before seeking approval, fee waivers, and additional criteria

Transition period considerations for sponsors

Kaye Robertson,Senior Medical Officer and Medicines evaluator, Prescription Medicines Assessment Branch,Therapeutic Goods Administration

Apr 2615:40

Conference pass

Creating value in gene therapy clinical development programs, achieving trial optimization and supporting the path to commercialization

Gene Track

Investigating novel vector technologies that can improve efficacy while reducing immunogenicity and potential re-dosing

Thinking about end to end clinical development including trial design, enrollment, execution, supply chain and manufacturing

How can clinical development be optimized to support scalability? What are the current limitations faced by this field despite a decade of improvements?

Olivier Danos,Chief Scientific Officer,Regenxbio Inc

Apr 2615:40

Conference pass

Beyond “patient focused drug development” buzzwords - let’s get practical

Rare Disease Advocacy World

Industry and patient partnerships for clinical trial design and execution, funding discussions and community outreach

Patients as board members through the product lifecycle to impact the needs of the community

Partnering with patients in a compliant way and achieving support from government and academic institutions to drive scientific research

Ron Bartek,Co-Founder/Founding President,Friedreich's Ataxia Research Alliance

Carole Ben Maimon,Chief Executive Officer,Chondrial Therapeutics

Apr 2615:40

Conference pass

BXQ-350, a novel approach for the potential treatment of glioblastoma multiforme

Pitch & Partner

Ray Takigiku,President and Chief Executive Officer,Bexion Pharmaceuticals Llc

Apr 2615:50

Conference pass

ALS205 for the treatment of amyotrophic lateral sclerosis (ALS)

Pitch & Partner

Alan Robertson,Chief Executive Officer and Managing Director,Alsonex

Apr 2615:50

Conference pass

BULGARIA: Bulgarian patients with rare diseases in focus of World and EC orphan drug development

Patient Groups Seminar

Vladimir Tomov,Chairman,National Alliance of People with Rare Diseases - Bulgaria

Apr 2616:00

Conference pass

Filling post market evidence gaps to support approval decisions and reimbursement

Clinical Development & Regulatory Track

Thinking about post approval data collection before approval

Designing new studies and looking into real world evidence, patient reported outcomes and natural history studies

Developing an evidence strategy that assesses the current reimbursement, regulatory and commercial landscape

Ashish Dugar,Vice President, Global Medical Affairs,Sarepta Therapeutics

Apr 2616:00

Conference pass

Spotlight on ultra-rare: commercializing therapies for very small patient populations

Commercial Track

Lessons from the development and launch of Mepsevii for a disease affecting <200 patients

Accelerating approval, finding the patients, building a value story, and assuring access

Commercialization challenges posed in the ultra-rare- versus rare-disease space.

Gary Geipel,Senior Director, Global Brand Communications,Ultragenyx Pharmaceutical

Apr 2616:00

Conference pass

BRAZIL: Accelerating the review and approval of orphan drugs in Latin America’s largest market

Global Market Access Track

Resolution to establish a procedure for clinical trials consent and good manufacturing practices

Guidelines for registration of new drugs for treatment, diagnosis or prevention of rare diseases

What this means for patients, drug manufacturers and the healthcare system

Jarbas Barbosa da Silva Jr,Director-President,ANVISA

Apr 2616:00

Conference pass

Gene editing approaches in rare blood disorders

Gene Track

Unmet need for transformative therapies in hemoglobinopathies

Disrupting an erythroid specific BCL11A enhancer with zinc finger nucleases for the treatment of sickle cell disease and beta-thalassemia

The potential to reactivate fetal hemoglobin to treat patients with SCD and beta-thalassemia

Dana Levasseur,Leader and Associate Director,Bioverativ

Apr 2616:00

Conference pass

The holy grail of rare disease research – Framework for effective engagement with patient groups in clinical trials

Rare Disease Advocacy World

Understanding current practices in patient groups-sponsors collaborations

Filling knowledge gaps regarding best practices for patient groups engagement in every phase of drug development

From fundraising to trial operations to helping with access- understanding the ecosystem

Pamela Tenaerts,Director,Clinical Trials Transformation Initiative

Apr 2616:00

Conference pass

TZ 101/TZ 102: Transforming cancer patient survival and quality of life outcomes by improving the delivery of therapeutic cells to tumors and sites of inflammation

Pitch & Partner

Lynnet Koh,Chairman and Chief Executive Officer and Founder,Targazyme

Apr 2616:10

Conference pass

Human rhodospin optogenetics for treatment of retinitis pigmentosa

Pitch & Partner

Erik Richardson,Project Leader of Gene Therapy,Acucela

Apr 2616:10

Conference pass

RUSSIA: The situation with rare diseases and patients’ organizations in Russia. Local legislation and it pitfalls, sharing experience

Patient Groups Seminar

Denis Belyakov,Executive Director,Union of Patients’ And Patients’ Organizations With Rare Diseases

Apr 2616:20

Conference pass

Real World Evidence 2.0 – from basic evidence collection to its inclusion in drug review applications

Clinical Development & Regulatory Track

What is usable Real World Evidence?

Simplifying collection, incorporating data into drug applications, and leveraging its potential to drive drug effectiveness

What are the necessary steps to incorporate RWE in the regulatory approval process? How can these be incorporated early on?

Apr 2616:20

Conference pass

Orphan drug launches – what drives success? How to best prepare for an orphan drug launch.

Commercial Track

Launch excellence: how orphan drugs performance is associated with pre-launch preparedness and developed commercial awareness

Performance: the impact of multiple levels of orphan drug differentiation and unmet medical needs

Not all orphan drugs are the same – how to leverage pre-approval initiatives, forecasting and epidemiology for commercial success

 

Adam Sohn,Vice President, Global Consulting Services,IQVIA

Simone Seiter,Vice President Global Lead Launch Excellence,IQVIA

Apr 2616:20

Conference pass

TURKEY: Access to orphan drugs in a large market outside the European Union

Global Market Access Track

Legislative efforts to define rare diseases and promote the development and commercialization of orphan drugs

Channels available to obtain unapproved drugs in Turkey but available in the EU (off-label use, named- patient imports, compassionate use)

Working towards establishing national networks for the prevention, surveillance, treatment of rare diseases

 

Ayse Deniz Ozger,Vice President, Healthcare Division,Eczacibasi Healthcare

Apr 2616:20

Conference pass

Achieving patient retention in gene therapy follow up

Gene Track

Strategies for optimizing patient retention throughout gene therapy programs (Phase 1-3, follow-up studies, registries)

New ways to better understand and navigate patient challenges first hand (logistical hurdles presented by moving from the initial “treatment” site/country (HCP) to potentially new follow-up sites/HCPs)

Gene therapy trials vs. other trials and what needs to be considered

 

Mariah Baltezegar,Executive Director Clinical Development,Syneos Health

Apr 2616:20

Conference pass

Collaboration of board members & rare disease non-profits

Rare Disease Advocacy World

NDF strategic partnerships to expand research Successful completion of International Symposium 2017

Certification of NDF Patient Advocates

Growing advocacy initiatives globally Improving education and awareness

Best practices of Board Members

 

Tricia Mullins,Board Member,Neuromuscular Disease Foundation

Apr 2616:20

Conference pass

A novel mechanism for treating ipf using small molecules

Pitch & Partner

Mehran F. Moghaddam,CEO,Orox Biosciences

Apr 2616:30

Conference pass

Hepcidin for treatment of iron overload disorders

Pitch & Partner

Matt Ritter,Head of Business Development,La Jolla Pharmaceutical Company

Apr 2616:30

Conference pass

MALAYSIA: #thinkSMALLactBIG – getting people to think about SMA and rare diseases, the SMALL ones who are affected by it, and to do their part to make a BIG difference

Patient Groups Seminar

Sook Yee Lim,Vice President,Malaysian Rare Disorders Society

Apr 2617:20

Conference pass

Keynote panel: How rare disease companies are ramping up innovation to boost orphan drug development and patient centricity

Keynotes

Incorporating patients in the drug development process and working towards commercial viability- how to fully engage?

How do you view disruptive therapies such as (gene therapy/gene editing) impacting the broader orphan disease development paradigm? How are you innovating within your company?

Responding to the changes in the political, regulatory and reimbursement landscape while guiding the direction of rare disease companies

How to achieve industry collaboration and idea exchanges that can better serve the patient community?

Francois Nader,Chairman,Acceleron Pharma

Timothy Walbert,Chairman, President, and Chief Executive Officer,Horizon Pharma

Arthur Tzianabos,President and Chief Executive Officer,Homology Medicines

Douglas Ingram,Chief Executive Officer,Sarepta Therapeutics

Daniel De Boer,Chief Executive Officer,Proqr

Apr 27   

Apr 2708:35

Conference pass

Keynote address: Developing genomic medicines

Keynotes

Gene therapy, genome editing, cell therapy, gene regulation: choosing the best therapeutic platform to address patients’ needs

Editing liver cells with ZFNs to potentially cure rare diseases, including MPS I, MPS II and hemophilia B

Zinc finger nuclease technology: Precision, Efficiency, Specificity -- what’s important when developing genomic medicines

Zinc finger nucleases as a best-in-class T-cell editing tool for cell-based therapies

The next frontier: what does the future of genome editing look like?

Sandy Macrae,President and Chief Executive Officer,Sangamo Therapeutics

Apr 2709:00

Conference pass

Keynote address: Advancing innovative therapies to treat genetically-defined diseases using CRISPR-based genome editing

Keynotes

Momentum toward making medicines – a potentially transformative new category of medicines that aim to repair “broken” genes

Strategy focused on diseases with few or no available treatments and exploring a Regulatory framework for CRISPR-based medicines

New data demonstrating advancements for translating CRISPR technologies into medicines

Clinical natural history study of Leber Congenital Amaurosis to understand the disease course and potential clinical endpoints

Advancing toward the clinic with potential to transform therapy for retinal disease LCA10-CEP290

Charlie Albright,Chief Scientific Officer,Editas Medicine

Apr 2709:25

Conference pass

Keynote address: Translating CRISPR/Cas9 technology into transformative gene-based medicines for patients with serious diseases

Keynotes

In vivo and ex vivo treatments using CRISPR/Cas9 for different therapeutic indications

CRISPR/Cas9 potential to correct DNA changes in somatic (non germ line) cells in patients with serious disease and the road to clinical trials

Partnerships with other pharmaceutical companies to develop gene-editing-based therapeutics

Rodger Novak,President CRISPR Therapeutics AG and Director of the Board,CRISPR Therapeutics

Apr 2709:50

Conference pass

The NIH Common Fund Somatic Cell Genome Editing Program: Developing tools and technologies to enable safe and effective genome editing in humans

Keynotes

Overview of the NIH Common Fund Somatic Cell Genome Editing Program

Program components including creating better animal models, tools to detect adverse events, technologies to deliver genome editing and developing capabilities for knowledge sharing

Looking into the future of genome editing technologies and strengthening academic and industry collaborations to achieve wider adoption

Christopher Austin,Director, National Center for Advancing Translational Sciences (NCATS),National Institutes of Health

Apr 2710:50

Conference pass

Advancing Choroideremia research and therapy development through industry partnerships

Patient Groups Seminar

Chris Moen,President,Choroideremia Research Foundation Inc

Apr 2711:10

Conference pass

Roadmap for collaboration – accelerating the development of and access to new and innovative treatments for systemic amyloidosis

Patient Groups Seminar

Isabelle Lousada,CEO,Amyloidosis Research Consortium

Apr 2711:15

Conference pass

TAK-celeratorTM - An incubator-like model to accelerate the development of transformative therapies in rare diseases

Commercial Track

Taking assets through translation into novel therapies utilising externalisation paths such as creation of NewCo’s or partner collaborations to increase the chance of reaching patients

Internal and external funding opportunities and the benefits of a global pharma company

Building mutually beneficial collaborations across academia, patient organisations and industry through a flexible working and agile style

Christine Charman,External Asset Lead,Takeda Development Centre Europe

Apr 2711:15

Conference pass

THE PHILIPPINES: How a newly passed Rare Diseases Act is unlocking an emerging market’s commercial potential

Global Market Access Track

Two years after the introduction of a Rare Disease Act covering registries, patient care, R&D, patient care and screenings

Introduction of a national strategy specifically for rare disease management facilitating coverage and access

How the Philippine society for orphan disorders pushed for the approval of this key legislation

Carmencita D Padilla,Founding Chairman,Philippine Society for Orphan Disorders

Apr 2711:15

Conference pass

Orphan drug designations in an era of novel technologies including CAR-T-cell, stem cells and gene therapy

Gene Track

Case studies of recent FDA approvals with orphan drug designation

Challenges for the newer modality of therapies such as CAR-T intended to treat diseases with higher prevalence including cancers

Different requirements and challenges faced by pharmaceutical companies to obtain global orphan drug designations

Bob Desai,Vice President, Technical,PAREXEL International Corp

Apr 2711:15

Conference pass

Community-based patient advocacy

Rare Disease Advocacy World

Different approaches to patient advocacy

Health disparities and the role of stakeholders in complex healthcare ecosystems

Interaction with patient advocacy groups and patient advocates

Mariana Fagnilli,Head of Global Patient Advocacy,bluebird bio

Apr 2711:15

Conference pass

Maximizing the impact of public investment to accelerate rare disease therapeutic development

Pitch & Partner

Private public partnerships with NCATS to bridge translational gaps – funding and technical expertise

Establishing platform technologies at TRND to catalyze rare disease therapeutic development

Exploring innovative approaches to unblock regulatory bottlenecks

Nora Yang,Director, Portfolio and Project Management, Strategic Operations, Division of Pre-Clinical Innovation,National Institutes of Health

Apr 2711:15

Conference pass

Proposed Value based frameworks for pricing and reimbursement of orphan drugs - can a new value framework help ease friction over orphan drug prices?

Pricing & Reimbursement Track

Methods to recommend fair prices to allow for broader insurance coverage for innovative new treatments

Different proposals for gene therapy pricing and possible routes for implementation

Impact of willingness to pay thresholds in the US and the orphan market space

Rick Chapman,Director of Health Economics,Institute for Clinical and Economic Review

Apr 2711:30

Conference pass

Using the power of community to advance treatments in Usher syndrome

Patient Groups Seminar

Mark Dunning,Chairman,Coalition for Usher Syndrome Research

Apr 2711:40

Conference pass

Ensuring commercial success through lifelong innovation: evolving alongside patients to meet their changing needs through the product lifecycle

Commercial Track

Reviewing opportunities for biopharma companies to continue listening to patients long after a drug has launched, particularly as pediatric patients become adults and their needs evolve

Turning patient and physician insights into new formulations, new doses, and new storage options

Going beyond scientific innovation to address changing patient needs through ambassador and other programs

Holly May,Vice President, Sales and Marketing,Sobi

Apr 2711:40

Conference pass

BRAZIL: Qualifying and standardizing the legal process for access to non-approved orphan drugs in Brazil

Global Market Access Track

Working towards better access for orphan drugs to surpass the common route of legal action by patients

Navigating Brazil’s complex payer landscape and leveraging SUS’ guidelines for improved diagnosis and treatment

Strategies for launching orphan drugs in LatAm’s most promising orphan drug market

Arnaldo Hossepian,Member,Conselho Nacional de Justiça

Apr 2711:40

Conference pass

Pioneering partnerships between industry and patient groups to advance gene therapy research to treat inherited retinal diseases

Gene Track

Supporting research initiatives like My Retina Tracker registry and studies to identify barriers that prevent patients use of genetic testing

Leveraging partnerships and funding opportunities to increase gene therapy clinical trial enrollment

Working toward common objectives and maximizing patient groups’ and industry’s capabilities

Sue Washer,President/CEO,AGTC

Apr 2711:40

Conference pass

Cross-functional education of internal stakeholders while maximizing patients’ opportunities through the product lifecycle

Rare Disease Advocacy World

Creating a strategic plan to address patient advocacy across the spectrum of pharmaceutical functions

Presenting opportunities for patients to engage with regulatoryagencies at preclinical and clinical stages

Incorporating the patient perspective to influence endpoints, recruitment, retention, etc.

Cristina Klafehn,Associate Director,Development Sciences Patient Advocacy,BioMarin Pharmaceutical Inc.

Apr 2711:40

Conference pass

Case study: What Sucampo’s acquisition by Mallinckrodt Pharmaceuticals tells about investing in ultra-rare diseases with unmet medical needs

Pitch & Partner

Hugh O'Neill,President, Autoimmune & Rare Disease,Mallinckrodt Pharmaceuticals

Apr 2711:40

Conference pass

Drug pricing in Europe, improving the role of HTA agencies and looking into the sustainability of managed access agreements

Pricing & Reimbursement Track

Looking into voluntary price joint price negotiations as a means to assess highly priced drugs and increase access

Can HTA assessment be improved across European countries?

What will be the future of managed access agreements as more drugs require them and more resources are needed? Can this model be replicated around Europe?

Edmund Jessop,Medical Adviser,National Health Service

Apr 2711:50

Conference pass

Achieving our goals together – caregivers and patients’ partnerships with industry to advance Huntington’s disease therapies

Patient Groups Seminar

Catherine Martin,Executive Director,The Huntington’s Disease Youth Organization

Bryan Viau,Chairperson,The Huntington’s Disease Youth Organization

Apr 2712:05

Conference pass

Designing your distribution channel to optimize your orphan drug commercialization strategy

Commercial Track

Taking the right steps to maintain inventory control

Ensuring access to patient site of care

Increase speed to market through strong channel footprint

Gain valuable business insights through data and analytics

Kevin Kissling,Vice President, 3PL Services,McKesson Specialty Health

Chad Pulliam,Director, Channel Operations,,Jazz Pharmaceuticals

Apr 2712:05

Conference pass

Tailoring a market access strategy to include the patient voice and effectively engage with key stakeholders locally and globally

Global Market Access Track

Implementing market access strategies earlier in drug development to work towards reimbursable label and access, not just approval

Including the patient and payer perspective early on in clinical development and understanding the different levels of unmet medical need

Health economics and outcomes research- capturing the PROs around quality of life and linking that to the disease, patients and caregivers

Raquel Cabo,Vice President, Global Market Access,Ovid Therapeutics

Apr 2712:05

Conference pass

Increasing process yields in AAV production for gene therapies

Gene Track

Tackling the industry’s AAV demand

Managing the variation in volume needed in AAV material to treat various therapeutic indications

Improving the triple transfection manufacturing platform in AAV production

Next evolution of the AAV production platform

Alain Lamproye,Chief Executive Officer,YposKesi

Apr 2712:05

Conference pass

Patient-centered qualitative research to inform potential outcome measures for Angelman syndrome clinical trials

Rare Disease Advocacy World

Identifying and developing the best outcome measures and biomarkers for future clinical trials

Working with doctors and patients globally to determine what to measure, how to measure it, and how to interpret findings

The Angelman Biomarkers and Outcome Measures Alliance (A-BOM)

Meghan Miller,Project Leader/Discovery Scientist, Rare Diseases,Roche

Apr 2712:05

Conference pass

Developing new payment and alternative financing models to ensure higher value and better outcomes for the money spent in highly priced medical products

Pricing & Reimbursement Track

Adapting health system reimbursement models to encourage innovation in curative gene therapies and other innovative therapies

Obstacles and opportunities for the increased implementation of value-based payment arrangements in the United States

Gene therapies vs. other highly priced treatments- looking into the sustainability of the healthcare system

Gregory Daniel,Deputy Director,Duke University

Apr 2712:10

Conference pass

Collaboration between patient advocacy groups and industry - creating a lasting partnership

Patient Groups Seminar

Anne Bruns,Director of Outreach for the Rare and Undiagnosed Network,The Atypical HUS Foundation

Apr 2712:20

Conference pass

Glutamate modulating medicines for the treatment of rare neurologic diseases

Pitch & Partner

Donnie McGrath,Head of Corporate Strategy & Business Development,Biohaven Pharmaceuticals

Apr 2712:30

Conference pass

PP-001 a novel small molecule for the treatment of noninfectious uveitis

Pitch & Partner

Franz Obermayr,Chief Executive Officer,Panoptes Pharma

Apr 2712:40

Conference pass

Better treatments for cancer including GBM and metastatic brain cancer

Pitch & Partner

Bill Hornung,Chief Business Officer,Diffusion Pharmaceuticals LLC

Apr 2712:50

Conference pass

Active checkpoint control therapeutics will supersede most monoclonal antibodies

Pitch & Partner

Fred Jacobs,Chief Executive Officer and Co Founder,TYG

Apr 2713:00

Conference pass

Lunch Keynote address: Evaluating the role of the Orphan Drug Act and looking into incentives, orphan drug usage and costs to ensure sustainability

Keynotes

Peter Saltonstall,CEO,National Organization for Rare Disorders

Apr 2714:00

Conference pass

Building community advocacy and patient engagement for individuals with bleeding disorders to educate legislators, care providers and other stakeholders

Patient Groups Seminar

Susan Fenters Lerch,Executive Director,The Hemophilia Foundation of Michigan

Apr 2714:01

Conference pass

ROUNDTABLE 1: Incentives ecosystem - current political, regulatory and legislative climate in the EU and US, around orphan product incentives

Keynotes

Rylan Hanks,Director, Regulatory and R&D Policy,Amgen Inc

Apr 2714:02

Conference pass

ROUNDTABLE 2:Cell and gene therapy- post-approval challenges for orphan advanced therapies, taking into account the patient journey, manufacturing and distribution

Keynotes

Diego Ardigo,Project Lead,Chiesi Farmaceutici SpA

Apr 2714:03

Conference pass

ROUNDTABLE 3: Gene therapy - patient advocacy in gene therapy vs. traditional programs, achieving adherence and long-term engagement

Keynotes

Barbara Wuebbels,Vice President of Patient Advocacy,Audentes Therapeutics

Apr 2714:04

Conference pass

ROUNDTABLE 4: Gene Therapy trials- Overcoming the challenges of conducting gene therapy studies and navigating the regulatory landscape

Keynotes

Scott Schliebner,Vice President, Scientific Affairs, Rare Diseases,PRA

Apr 2714:05

Conference pass

ROUNDTABLE 5: Startup building - building and leading start-up biotechs into successful companies and building the necessary platforms to support commercial goals

Keynotes

Dawn Bir,Chief Commercial Officer,Reata Pharmaceuticals

Apr 2714:06

Conference pass

ROUNDTABLE 6: Clinical development- incorporating the patient voice in endpoint design and designing trials with all stakeholders in mind

Keynotes

Anita Appius,Global Development Team Leader,Roche

Apr 2714:07

Conference pass

ROUNDTABLE 7: Cognitive endpoints – designing trials for success and strategies for rescuing a failed study

Keynotes

Melissa Hogan,Founder and President,Project Alive

Apr 2714:08

Conference pass

ROUNDTABLE 8: RWD – utilizing real-world data throughout the product life-span for rare disease treatments

Keynotes

Stella Blackburn,Vice President, Global Head of Early Access and Risk Management,IQVIA

Apr 2714:09

Conference pass

ROUNDTABLE 9: Unorthodox funding strategies - alternate but complimentary paths to funding orphan drug companies

Keynotes

M Ken Kengatharan,Managing Partner, Atheneos Ventures and CEO,Auxesia Orion

Apr 2714:11

Conference pass

ROUNDTABLE 11: PROs in clinical trials – what have we accomplished and what is the new frontier in orphan drug treatments?

Keynotes

Kathryn Lasch,Executive Director of Patient-Reported Outcomes,Pharmerit International

Apr 2714:15

Conference pass

Retinol palmitate for prevention of bronchopulmonary dysplasia (BPD) and retinopathy of prematurity (ROP) in preterm babies

Pitch & Partner

Philipp Novak,Founder and Chief Executive Officer,ORPHANIX

Apr 2714:20

Conference pass

Driving treatments in Hyperoxaluria through collaborations with industry, FDA and patients

Patient Groups Seminar

Kim Hollander,Executive Director,The Oxalosis and Hyperoxaluria Foundation

Apr 2714:25

Conference pass

Orally Inhaled Ondansetro for the prevention of chemotherapy-induced nausea and vomiting due to highly emetogenic chemotherapy in pediatric patients

Pitch & Partner

Eric Li,Chief Scientific Officer,Luxena Pharmaceuticals

Apr 2714:35

Conference pass

Enabling a New Era of RNA medicines

Pitch & Partner

Mark Herbert,Interim President,Arcturus Therapeutics

Apr 2714:40

Conference pass

Using a private digital platform to accelerate R&D - enabling patients to collect, share and monetize their data

Patient Groups Seminar

Jeanne Barnett,Founder and President,Cysticfibrosis.com

Apr 2714:45

Conference pass

The Use of AKT Inhibitors in rare overgrowth syndromes

Pitch & Partner

Brian Schwartz,Chief Medical Officer,Arqule, Inc.

Apr 2714:55

Conference pass

Multiple clinical-staged programs for the treatment of rare diseases

Pitch & Partner

David Cory,President, Chief Executive Officer,Eiger Biopharmaceuticals

Apr 2715:00

Conference pass

Inspiring the rare disease community with hope through the power of sport – an athlete’s rare disease story

Patient Groups Seminar

Robert Long,Director of Strategic Development,Uplifting Athletes

Apr 2715:05

Conference pass

Cancer and thyroid hormone deprivation - a novel oncological approach

Pitch & Partner

Offer Fabian,Chief Executive Officer and Co Founder,Musli Thyropeutics

Apr 2715:10

Conference pass

Evidence generation in rare diseases – thinking creatively to generate evidence that effectively supports products

Clinical Development & Regulatory Track

Evidence requirements throughout the product lifecycle – where to look during each stage

Partnering with patients, caregivers and physicians to support evidence generation, trial success and reimbursement

Understanding of the breadth of real-world evidence options

Apr 2715:10

Conference pass

35 Years: Where have we come and where are we going

Rare Disease Advocacy World

A look at the progress the rare disease community has made over the last 35 years, since the founding of NORD and the passage of the Orphan Drug Act

NORD's POV on the current state of affairs in the United States in health care, policy, research, and awareness

Where are science and medicine heading, and where does the rare disease community need to focus its attention and efforts to ensure sustainability for the next 35 years

Pamela Gavin,Chief Strategy Officer,National Organization for Rare Disorders

Apr 2715:10

Conference pass

The value of computer vision, machine learning and robotic automation to expedite rare disease research

Discovery Track

Running experiments and performing analysis on hundreds of diseases simultaneously using powerful software and analytics

Finding new indications for existing drugs using augmented high-throughput drug research and technology

Creating high-resolution, comprehensive biological data sets

Academia-industry partnerships to foster rare disease research

Ron Alfa,Vice President of Discovery and Product,Recursion Pharmaceuticals

Apr 2715:15

Conference pass

Safety, tolerability and efficacy of a novel sustained release analog of vasoactive intestinal peptide, PB1046, in patients with pulmonary arterial hypertension

Pitch & Partner

John Lee,Chief Medical Officer,Phasebio Pharmaceuticals, Inc.

Apr 2715:20

Conference pass

Engaging the patient community to drive innovative and solution based collaborations to cure Alpha-1 Antitrypsin Deficiency

Patient Groups Seminar

Henry Moehring,President and CEO,Alpha-1 Foundation

Apr 2715:25

Conference pass

Oral, centrally acting TNF-α inhibitors for CNS inflammatory diseases including Frontotemporal Dementia (FTD)

Pitch & Partner

Prasad Gabbita,Chief Executive Officer,P2d Inc

Apr 2715:35

Conference pass

Scaling up rare disease patient engagement in Europe

Rare Disease Advocacy World

The EURORDIS new structured approach on patients' engagement in research, drug development and life cycle and healthcare

Creating value for patients and working through partnership to create value to all stakeholders

Yann Le Cam,Chief Executive Officer,EURORDIS

Apr 2715:35

Conference pass

BIOGENERA: A new era of DNA personalized drugs

Pitch & Partner

Roberto Tonelli,President, Chairman of the Board, Co-founder,BIOGENERA

Apr 2715:35

Conference pass

Using artificial intelligence to match rare disease treatments

Discovery Track

Leveraging drug repurposing and combination therapies to accelerate therapeutic outcomes

Applying genomic drug-matching

Partnering effectively with patient foundations

Tim Guilliams,CEO,Healx

Apr 2715:40

Conference pass

New born screening and registries, a foundation’s approach to change the future

Patient Groups Seminar

Terri Klein,Interim President and CEO,National M.P.S. Society

Apr 2715:45

Conference pass

Acidified Sodium Nitrite and EDTA for the Treatment of Pseudomonas aeruginosa infections in the lungs of cystic fibrosis patients

Pitch & Partner

Richard Muruve,Chief Executive Officer and President,Arch Biopartners

Apr 2715:55

Conference pass

Treatment of hepatocellular carcinoma (HCC) and tuberculosis (TB) with oral tableted immunotherapeutics, hepcortespenlisimut-L and tubimod

Pitch & Partner

Aldar Bourinbaiar,Chief Executive Officer,Immunitor Inc

Apr 2716:00

Conference pass

HIStories: sharing the patient experience to advance the scientific understanding of congenital hyperinsulinism

Patient Groups Seminar

Julie Raskin,Executive Director,Congenital Hyperinsulinism International

Keynote Speakers

Scott Gray

Chief Executive Officer, Clincierge

Scott Gray is an acknowledged leader and innovator in the world of medical meetings, patient-centered clinical trial support and healthcare transportation. In 2013, while hearing of patients’ challenges with logistics to participate in a global clinical trial, the Clincierge® concept was born as a means to improve patient-centricity in clinical trials and general medical treatments. He co-founded Gray Consulting International in 1994 and has served as CEO ever after. Since 2007, Gray Consulting International has been named 6 times to the “Top 25 Full-Service Event Management Companies in North America.”  Scott is also a popular speaker at major event management conferences has made numerous media appearances and sits on several industry advisory boards. 

Katherine High

President and Head, Research & Development, Spark Therapeutics

Dr. Kathy High, an accomplished hematologist with a longstanding interest in gene therapy for genetic disease, began her career studying the molecular basis of blood coagulation and the development of novel therapeutics for the treatment of bleeding disorders. Her pioneering bench-to-bedside studies of gene therapy for hemophilia led to a series of studies that characterized the human immune response to AAV vectors in a variety of target tissues. Kathy’s work has evolved to encompass clinical translation of potential genetic therapies for multiple inherited disorders. As the director of the Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia, Kathy assembled a multidisciplinary team of scientists and researchers working to discover new gene and cell therapies for genetic diseases and to facilitate rapid translation of preclinical discoveries into clinical application.

Kathy was a long-time member of the faculty at the University of Pennsylvania and of the medical staff at CHOP, where she was also an Investigator of the Howard Hughes Medical Institute. She served a five-year term on the FDA Advisory Committee on Cell, Tissue and Gene Therapies and is a past president of the American Society of Gene & Cell Therapy (ASGCT). She received her A.B. in chemistry from Harvard University, an M.D. from the University of North Carolina School of Medicine, a business certification from the University of North Carolina Business School Management Institute for Hospital Administrators and an M.A. from the University of Pennsylvania.

Tim Miller

President and Chief Executive Officer, Abeona Therapeutics

Timothy J. Miller, PhD, became our President and Chief Executive Officer and Director on May 15, 2015. Dr. Miller was President & CEO of Abeona Therapeutics LLC from 2013 to 2015. He has 16 years of scientific research, product development, regulatory and clinical operations expertise, with a focus on transitioning novel biotherapeutics through pre-clinical phases and into Phase 1 and 2 human clinical trials. Dr. Miller was President & CEO of Red5 Pharmaceuticals from 2013 until 2015 and was Vice President, Business Development of BioEnterprise Inc in 2015. He was Senior Director of Product Development at SironRX Therapeutics from 2010 to 2013. Between 1996 and 2010 Dr. Miller held various positions at several companies focusing on gene therapy and regenerative medicine. Dr. Miller earned his PhD in Pharmacology with a focus on Gene therapy/Cystic Fibrosis from Case Western University. He also holds a B.S. in Biology and M.S. in Molecular Biology from John Carroll University (Cleveland, OH).

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