Clinical Trials in Rare Diseases 2023

We are pleased to announce the launch of a brand new event: Clinical Trials in Rare Diseases will take place on 13th-14th September 2023 in Princeton!

13 - 14

September

2023
  • Princeton Marriott at Forrestal, New Jersey, USA
  • Complimentary
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Why attend?

A FANTASTIC NETWORK OF YOUR PEERS AND COLLEAGUES

With over 40 solution providers this will be a perfect platform to reconnect with current business partners, meet new vendors and discover which new technologies are being employed to improve your Rare Disease clinical trials.

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Agenda

  • 13 Sep 2023
  • 14 Sep 2023
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8 AM

Registration and refreshments

8:50 AM

Chairperson’s opening remarks

Speakers

Chris Adams
Chief Executive Officer, Andarix

9 AM

OPENING KEYNOTE: Clinical Study Participation: Rare Disease Patient Perspective

  • Understanding what it’s like to live with a rare disease (~750 cases reported worldwide)
  • How living with a rare disease can impact the decision to enroll and stay in a clinical study
  • Some solutions to ease the burden on patients and improve access to studies

Speakers

Michele Rhee
Rare Disease Patient and Head of Global Advocacy Relations and Patient Engagement, Savara Inc

9:30 AM

Moving the needle in rare disease trials: how to optimize study design without breaking the bank

It’s no secret that rare disease trials encounter many obstacles from start to finish. With small patient populations, limited data on the disease, and patient enrollment and retention challenges, every component of the study protocol can significantly influence the success of the trial. These challenges, paired with ever-increasing clinical trial costs, can leave sponsors feeling overwhelming pressure to stretch their budgets without sacrificing the data they need for regulatory approval. During this session, you will learn:

  • Tangible tactics for reducing patient burden
  • Centralized monitoring strategies to contain biotech spend without compromising data quality
  • Ways to infuse technology and decentralized approaches to enhance the patient experience

Speakers

Leslie Wetherell
Executive Director, Program Strategy, Rare Disease and Pediatrics, Premier Research

10 AM

PANEL DISCUSSION: How decentralization and direct-to-patient shipping opens up possibilities for patients with rare diseases

  • Improving enrolment, retention and access to clinical trials through decentralized and hybrid clinical trial models
  • Incorporating direct-to-patient shipping in order to make participating trials easier for patients and challenges associated with DTP
  • DCT and DTP as a way to open up trial participation to pools of patients who would otherwise have been unable to access trials
  • Assessing home nursing and hybrid options for when full decentralization is not possible due to the nature of your drug

Speakers

Keith Chiasson
Vice President, Drug Development
Laurie Taraborrelli
Clinical Programs and Compliance Specialist
Peter Ciszewski
Founder and Publisher, CheckRare

10:30 AM

Morning refreshments and networking

11:15 AM

Improving ROI by working with true disease experts: patients, and how working with advocacy groups can positively impact a trial

  • Methods used to collaborate with rare disease advocacy groups from trial design forward
  • Working with advocacy groups to understand challenges and barriers patients face, and how you can support participants throughout the clinical trial in order to increase retention rates and improve experiences
  • Developing a case for alternate trial endpoints that positively impact how patients feel, function, and survive

Speakers

Julie Breneiser
Executive Director, Gorlin Syndrome Alliance

11:45 AM

Biostatistical considerations in rare disease product development

What are the study design considerations for a rare disease study?

  • Often there are no formal regulatory guidance
  • How to address sample size requirements with small patient population
  • What are the accepted assessments from clinical practice
  • Subgroup or prognostic factor considerations

Speakers

David Ng
Vice President, Global Biometrics, Novotech

12:15 PM

An update on regulations and policy surrounding rare disease and orphan drug trials: what do you need to know?

  • FDA, NIH and CMS issues impacting rare disease and orphan drug trials
  • Utilizing real world evidence effectively and key considerations when using this kind of data
  • Understanding regulatory flexibility for clinical trial diversity and decentralization in orphan drug trials

Speakers

Karin Hoelzer
Director, Policy and Regulatory Affairs, National Organization for Rare Disorders (NORD)

12:45 PM

Lunch and networking

2 PM

How can you improve diversity and inclusion in rare disease trials?

  • FDA guidance and legislation around diversity in clinical trials and how to apply this in a rare disease trial
  • How to increase awareness of your trial in order to reach more patients including those in more diverse settings
  • The importance of building trust with groups and communities who may not traditionally participate in clinical trials in order to increase trial diversity and ultimately improve the effectiveness of your drug

Speakers

Laurie Taraborrelli
Clinical Programs and Compliance Specialist

2:30 PM

CASE STUDY: A site perspective on running a clinical trial for a rare disease

  • Overcoming challenges involved when in a trial with an aggressive disease
  • Learnings and outcome of running clinical trials on glioblastoma tumors
  • How sponsors can best work with sites effectively to ensure rare disease trials run as smoothly as possible

Speakers

Ravipal Luthra
Clinical Research Lead, Sylvester Comprehensive Cancer Center, University of Miami

3 PM

CASE STUDY: Developing a cell therapy for patients with telomere biology disorders: challenges and lessons learned

  • An overview of Elixirgen’s lead EXG-34217 program from a clinical operations perspective
  • Running a clinical trial with patients with a rare disease: what do you need to consider?
  • Key challenges faced during Phase I/II trials on EXG-34217 and future opportunities uncovered

Speakers

Akihiro Ko
CEO, Co-Founder and Board Chairman, Elixirgen Therapeutics

3:30 PM

Afternoon refreshments and networking

4:15 PM

Clinical trials for rare diseases: challenges and opportunities

  • Best practice in ensuring your rare disease trial is a success
  • Using precision medicine in clinical trials to advancing therapeutics for rare diseases
  • Assessing clinical data with biomarker-correlated outcomes in Rett Syndrom

Speakers

Daniel Klamer
Vice President, Business Development & Scientific Strategy, Anavex Life Sciences

4:45 PM

Chairperson’s closing remarks

Speakers

Chris Adams
Chief Executive Officer, Andarix

8 AM

Registration and refreshments

8:50 AM

Chairperson’s opening remarks

Speakers

Chris Adams
Chief Executive Officer, Andarix

9 AM

PROBLEM-SOLVING ROUNDTABLE DISCUSSIONS

During the roundtable discussion session, the conference hall will be divided into four ‘zones’. Delegates can choose which zone they would like to join. Each zone will be led by a table moderator and will focus on a different challenge within rare disease and orphan drug clinical trials. After 45 minutes, delegates will have the opportunity to swap and choose a different table, and each roundtable will run twice.

ROUNDTABLE 1: Considerations when designing a clinical trial for a rare disease: challenges and solutions

 

 

ROUNDTABLE 2: Creating a patient friendly clinical trial: working with advocacy groups effectively

Julie Breneiser, Executive Director, Gorlin Syndrome Alliance

 

ROUNDTABLE 3: Identifying partners and vendors to maximize efficiency of your rare disease trial

Laurie Taraborrelli, Clinical Programs and Compliance Specialist, Fujirebio Diagnostics

 

ROUNDTABLE 4: Building strong academic partnerships to make your rare disease trial a success

 

 

Speakers

Laurie Taraborrelli
Clinical Programs and Compliance Specialist
Julie Breneiser
Executive Director, Gorlin Syndrome Alliance
Gurdyal Kalsi
Chief Medical Officer, Asklepion Pharmaceuticals
Jeff R. Swarz
Chief Executive Officer, ATED Therapeutics

10:30 AM

Morning refreshments and networking

11:15 AM

Discussing intersections with rare diseases, health disparities and clinical trial research

  • Diversity and health equity: the importance of considering the underserved populations
  • Understanding how global changing demographics impact clinical trial diversity and inclusion
  • Recognising systemic disparities in clinical trials and how to address these

Speakers

Jenifer Waldrop
Executive Director, Rare Disease Diversity Coalition

11:45 AM

CASE STUDY: From bench to bedside: taking an orphan drug from the lab to a clinic

  • Enhancing Diversity of Clinical Trial Populations – Inclusive practices and methodological approaches to facilitate enrolment of a broader population when preparing for a (rare disease) trial
  • Using qualitative systems pharmacology to offer hypothesis generating opportunities and fill natural history and real-world evidence (RWE) gaps
  • Assessing unmet needs and meaningful outcomes for orphan drugs in indications with limited precedence or pre-existing approved treatment options

Speakers

Noopur Singh
Director, Medical Affairs, Xentria

12:15 PM

Lunch, networking and prize draw!

1:30 PM

New solutions and tactics for enrolling patients in rare disease trials

  • Finding patients who are ‘off the grid’: how can you identify potential trial participants who have given up on finding a treatment for their rare disease?
  • Leveraging the power of social media in your patient enrolment campaign: using tools such as Facebook groups and other platforms in order to reach out to higher numbers of patients
  • What new technology is available to support patient recruitment and how can you employ this effectively to reduce timelines for enrolment, and therefore the overall length of your trial?

Speakers

Jay Russak
Senior Director, Clinical Operations, Keros Therapeutics

2 PM

CLOSING KEYNOTE PANEL: Looking to 2024 and beyond: what’s on the horizon for rare disease and orphan drug trials?

  • With this year as the 40th anniversary of the Orphan Drug Act, how have clinical trials evolved over that period?
  • New regulations and guidance for running orphan drug trials in the USA: how will these impact trials?
  • Are we still seeing long term changes as a result of COVID? How the pandemic made trials become more flexible: are these changes here to stay?
  • Developments in patient recruitment: leveraging technology to shorten timelines and achieve as diverse a patient population as possible
  • Should decentralized trials be the norm, particularly when working with rare diseases?

Speakers

Peter Ciszewski
Founder and Publisher, CheckRare
Karin Hoelzer
Director, Policy and Regulatory Affairs, National Organization for Rare Disorders (NORD)
Akihiro Ko
CEO, Co-Founder and Board Chairman, Elixirgen Therapeutics
Kristy Galante
Senior Director, Janssen R&D

2:30 PM

Chairperson’s closing remarks

Speakers

Chris Adams
Chief Executive Officer, Andarix

Speakers

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Chris Adams
Chief Executive Officer, Andarix

Mr. Adams has been a founder or co-founder of life science and technology based companies in Massachusetts for the past several years. Mr. Adams has been instrumental in developing new proprietary therapeutic and medical device technologies and bringing the technologies to commercialization. The companies he has founded include, Compellis Pharmaceuticals, a developer of therapeutics to treat obesity and its co-morbidities, diabetes and hypertension and Mosaic Technologies, a venture backed MIT spin-out genomics company that developed patented technologies for genetic analysis and detection. Chris is the holder of more than 20 issued US and international patents for medical devices and pharmaceuticals.

Session Details:

Chairperson’s opening remarks

2023-09-13, 8:50 AM

Session Details:

Chairperson’s closing remarks

2023-09-13, 4:45 PM

Session Details:

Chairperson’s opening remarks

2023-09-14, 8:50 AM

Session Details:

Chairperson’s closing remarks

2023-09-14, 2:30 PM

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Michele Rhee
Rare Disease Patient and Head of Global Advocacy Relations and Patient Engagement, Savara Inc

Michele Rhee is Head of Global Advocacy Relations and Patient Engagement at Savara Inc. She has nearly two decades of experience engaging with patient communities and leading patient advocacy across biopharmaceutical and nonprofit industries. Michele has built the patient advocacy function as Vice President of Patient Affairs and Advocacy at X4 Pharmaceuticals and Head of Patient Affairs at Enzyvant. Prior to her work at Enzyvant, Michele served as Global and R&D Patient Advocacy Lead at Takeda Oncology, Head of Patient Advocacy at bluebird bio, and Director of both Strategic Initiatives and Program Initiatives at the National Brain Tumor Society. In addition, Michele was appointed to the Massachusetts Rare Disease Advisory Council in 2021 and is a founding board member for Costs of Care, an organization dedicated to helping clinicians and health systems deliver better care at lower cost. Michele received her MPH from Yale School of Public Health and MBA from Yale School of Management.

Session Details:

OPENING KEYNOTE: Clinical Study Participation: Rare Disease Patient Perspective

2023-09-13, 9:00 AM

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Leslie Wetherell
Executive Director, Program Strategy, Rare Disease and Pediatrics, Premier Research

Leslie Wetherell is a clinical research professional with over 25 years of global drug development experience, with a focus in rare disease. In her role, Wetherell’s therapeutic and operational expertise supports biotech and pharmaceutical companies achieve key results in rare disease and pediatric studies. Her focus is producing strategic efficiencies that support patient-centric and timely program delivery.

 

Wetherell has a depth of experience overseeing operational strategy, delivery, and efficiency in phase 1-3 trials in rare disease, oncology, hematology, and general medicine. Her past roles include data management, project management, procurement, and global strategy development positions. She holds a bachelor’s degree from San Diego State University.

Session Details:

Moving the needle in rare disease trials: how to optimize study design without breaking the bank

2023-09-13, 9:30 AM

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Keith Chiasson
Vice President, Drug Development

Battle-ready project oversight aficionado, with a proven history of working in both the Pharmaceutical and Medical Device landscapes. Innovator, trail blazer and process improvement advocate and guru. Skilled in Electronic Data Capture (EDC), Good Laboratory Practice (GLP), Medical Devices, Good Clinical Practice (GCP), and Clinical Trial Management System (CTMS). VERY detail-oriented, loves to do things outside-the-box, run on unpaved trails, and flip burgers differently from everybody else. Motto: "Comfort zone... remind me what this is again?"

Session Details:

PANEL DISCUSSION: How decentralization and direct-to-patient shipping opens up possibilities for patients with rare diseases

2023-09-13, 10:00 AM

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Laurie Taraborrelli
Clinical Programs and Compliance Specialist

Session Details:

PANEL DISCUSSION: How decentralization and direct-to-patient shipping opens up possibilities for patients with rare diseases

2023-09-13, 10:00 AM

Session Details:

How can you improve diversity and inclusion in rare disease trials?

2023-09-13, 2:00 PM

Session Details:

PROBLEM-SOLVING ROUNDTABLE DISCUSSIONS

2023-09-14, 9:00 AM

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Peter Ciszewski
Founder and Publisher, CheckRare

Session Details:

PANEL DISCUSSION: How decentralization and direct-to-patient shipping opens up possibilities for patients with rare diseases

2023-09-13, 10:00 AM

Session Details:

CLOSING KEYNOTE PANEL: Looking to 2024 and beyond: what’s on the horizon for rare disease and orphan drug trials?

2023-09-14, 2:00 PM

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Julie Breneiser
Executive Director, Gorlin Syndrome Alliance

Julie Breneiser is the Executive Director of the Gorlin Syndrome Alliance (GSA). Prior to her position as director, Julie served as volunteer Board President for the GSA. She and her two young adult children are affected with Gorlin syndrome, a rare genetic disorder caused by a tumor suppressant mutation that can affect every organ system. Her early career began as a Physician Assistant followed by teaching preschoolers with disabilities.

The work that Julie does with the GSA focuses on collaboration with industry to bring new and/or potential treatments to clinical trial. She also provides individual support to affected patients and their caregivers along with educating health care providers about this rare disease. In November of 2021, Julie was honored with an appointment to the Patient Engagement Collaborative at the FDA. This group of patients and advocates works to achieve more meaningful patient engagement in medical product development and other regulatory discussions at the FDA. Julie previously served as a consumer reviewer for the National Institutes of Health and the Department of Defense’s Congressionally Directed Medical Research Program. She speaks nationally and internationally raising awareness of Gorlin syndrome.

Session Details:

Improving ROI by working with true disease experts: patients, and how working with advocacy groups can positively impact a trial

2023-09-13, 11:15 AM

Session Details:

PROBLEM-SOLVING ROUNDTABLE DISCUSSIONS

2023-09-14, 9:00 AM

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David Ng
Vice President, Global Biometrics, Novotech

David Ng is Vice President, Global Biometrics at Novotech, a global CRO.   David has over 30 years of experience in global data management, statistics, and regulatory interactions; and has worked on numerous treatments for rare diseases, from proof of concept to submission and ultimate regulatory approval.   David has also collaborated with various organization and vendors to optimize data access and collection, from early EDC and ePRO systems to the use of EMR in clinical development.

Session Details:

Biostatistical considerations in rare disease product development

2023-09-13, 11:45 AM

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Karin Hoelzer
Director, Policy and Regulatory Affairs, National Organization for Rare Disorders (NORD)

Dr. Karin Hoelzer directs Policy and Regulatory Affairs for the National Organization for Rare Disorders (NORD®). In this role, Karin provides strategic direction to advance NORDs federal policy and regulatory priorities. She works closely with key rare disease partners across the pharmaceutical and biological space to ensure the policy landscape supports innovative approaches and new treatments to help rare disease patients, and adequately incorporates patient preferences and perspectives in therapy development. Her role also involves extensive legislative and communication engagements to advance rare disease policy priorities.

Dr. Hoelzer is a health policy, risk analysis, and biomedical research expert, with extensive intellectual property and regulatory expertise across most FDA-regulated products. Most recently, she worked at Maximus, Inc. where she established and led a new health data analytics division to provide more timely data and better insights to government clients in support of the public health response to the COVID-19 pandemic.

Prior to working at Maximus, Inc., Dr. Hoelzer served as Senior Officer for Health Programs at The Pew Charitable Trusts where she led policy and regulatory efforts to improve the federal oversight of a variety of FDA-regulated products. Dr. Hoelzer joined Pew from the Food and Drug Administration (FDA), where she served as Risk Analyst. In this role, she assessed and quantified the expected impact of changes to FDA policy and regulatory practice.

Prior to joining FDA in 2010, Dr. Hoelzer worked as Biomedical Researcher for Cornell University. She received a PhD in comparative biomedical sciences from Cornell University and a Doctor of Veterinary Medicine degree from the University of Veterinary Medicine in Hannover (Germany).  Since 2014, she is also a  registered patent agent with the U.S. Patent and Trademark Office, and remains active in the biomedical research space. This includes serving as Editor in Chief for a major peer-reviewed international biomedical research journal and as the Executive Director for the professional association representing veterinarians in DC.

Session Details:

An update on regulations and policy surrounding rare disease and orphan drug trials: what do you need to know?

2023-09-13, 12:15 PM

Session Details:

CLOSING KEYNOTE PANEL: Looking to 2024 and beyond: what’s on the horizon for rare disease and orphan drug trials?

2023-09-14, 2:00 PM

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Ravipal Luthra
Clinical Research Lead, Sylvester Comprehensive Cancer Center, University of Miami

Session Details:

CASE STUDY: A site perspective on running a clinical trial for a rare disease

2023-09-13, 2:30 PM

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Akihiro Ko
CEO, Co-Founder and Board Chairman, Elixirgen Therapeutics

Akihiro Ko has served as Chief Executive Officer and Chair of the board of directors since May 2017, when he co-founded the company. Previously, Mr. Ko served as Chief Operating Officer for Elixirgen, LLC, the parent company of Elixirgen Therapeutics, and President of Elixirgen Scientific, LLC, a provider of stem cell reagent kits and related services. In his previous roles, Mr. Ko was responsible for operations and business development at Elixirgen, LLC and Elixirgen Scientific. Mr. Ko earned a Bachelor of Science degree from University of Virginia, where he cultivated his interest in biology.

Session Details:

CASE STUDY: Developing a cell therapy for patients with telomere biology disorders: challenges and lessons learned

2023-09-13, 3:00 PM

Session Details:

CLOSING KEYNOTE PANEL: Looking to 2024 and beyond: what’s on the horizon for rare disease and orphan drug trials?

2023-09-14, 2:00 PM

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Daniel Klamer
Vice President, Business Development & Scientific Strategy, Anavex Life Sciences

Dr Daniel Klamer, Vice President of Business Development and Scientific Strategy of Anavex, has more than 15 years of experience in neuroscience and the orphan disease space, with acquisition, partnering and R&D experience in Europe and the USA. Prior to Anavex he worked at Retrophin and Neurosearch Sweden. At Neurosearch Sweden, Dr Klamer led and evaluated multiple discovery-phase neuropharmacological research products with an emphasis on strategic evaluation of preclinical and clinical development.

Dr. Klamer earned his PhD in Pharmacology at The Sahlgrenska Academy at University of Gothenburg, Sweden, his MBA at Fordham Gabelli School of Business, and his Post-Doctoral training at the Department of Psychiatry, Yale University School of Medicine. In addition, Dr. Klamer holds a position as an Associate Professor at the Department of Pharmacology at The Sahlgrenska Academy at the University of Gothenburg.

Session Details:

Clinical trials for rare diseases: challenges and opportunities

2023-09-13, 4:15 PM

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Gurdyal Kalsi
Chief Medical Officer, Asklepion Pharmaceuticals

Session Details:

PROBLEM-SOLVING ROUNDTABLE DISCUSSIONS

2023-09-14, 9:00 AM

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Jeff R. Swarz
Chief Executive Officer, ATED Therapeutics

Dr. Swarz co-founded and is CEO of the Israeli biotech firm ATED Therapeutics. He is also a Partner and Managing Director at M.M. Dillon & Company. Dr. Swarz had served as a Senior Managing Director at Caris & Company. Dr. Swarz brings 35 years of experience in product development and marketing, equity analysis, capital raising, and investment analysis for companies in various segments of the healthcare industry. Prior to joining Caris, Dr. Swarz was a Managing Director at FBR and Life Sciences Group where he was responsible for corporate finance, mergers and acquisitions and private financing. Before that, he was a partner at EGS Healthcare Capital Partners, a healthcare private equity fund investing in biotechnology, specialty pharmaceuticals, and medical device companies. As an equity analyst in biotechnology research at Credit Suisse First Boston and Goldman Sachs, Dr. Swarz was rated among Wall Street’s top ten analysts in biotechnology by Institutional Investor Magazine for ten years. Dr. Swarz was a National Institutes of Health Research Fellow in Neurovirology, completed a Postdoctoral Fellowship (Neurovirology) at Johns Hopkins School of Medicine’s Department of Neurology and earned a Ph.D. in Neuroscience from the University of Rochester, and a B.Sc. degree in Biological Sciences (with honors) from the University of California at Irvine.

Session Details:

PROBLEM-SOLVING ROUNDTABLE DISCUSSIONS

2023-09-14, 9:00 AM

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Jenifer Waldrop
Executive Director, Rare Disease Diversity Coalition

Jenifer Ngo Waldrop joined the Black Women’s Health Imperative (BWHI) as Executive Director of the Rare Disease Diversity Coalition (RDDC) in October 2022. Before joining the leadership team of RDDC, she developed models and programs for diversity, equity, and inclusion initiatives to support multiple business units with Fortune 500 companies and organizations in Asia, Canada, and the US.

Jenifer has led initiatives at non-profit organizations where she recruited local community members to participate in numerous health-related fundraising events and developed programs to target disparities and underrepresented groups. In addition to facilitating development and fundraising, Jenifer developed a reputation for successful coalition building.

Jenifer received her undergraduate degree from Colby College and her Master of Human Resources Development from Villanova. As the Professional Development Director of the National Association of Asian-American Professionals (NAAAP) of Colorado, Jenifer continues her outreach in society. Through this organization, she mentors formally and informally.

Session Details:

Discussing intersections with rare diseases, health disparities and clinical trial research

2023-09-14, 11:15 AM

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Noopur Singh
Director, Medical Affairs, Xentria

Noopur’s motivation stems from her desire to help mitigate treatment access challenges seen across indications. She is focused on strategic patient-centered drug development that bridges scientific gaps and cuts through red tape across the drug supply chain. She is determined to increase access to treatments to help fulfill current unmet needs. Her approach stems from the patient journey, ensuring it is integrated throughout the drug development life cycle. In her role as Director of Medical Affairs, Noopur nurtures Xentria’s key relationships with patient experts, key opinion leaders and clinical investigators.

Prior to her role at Xentria, Noopur was the Research Director at the Foundation for Sarcoidosis Research where she played a key role in the development and execution of the organization’s scientific research agenda. Noopur has also worked as Clinical Site Manager for Synexus Clinical Research in Chicago, where she was responsible for operationalizing clinical trials. Noopur held several roles at Lurie’s Children’s Hospital Chicago where she managed over 25 industry-sponsored trials. She has also been an active participant in the Therapeutics Development Network with the Cystic Fibrosis Foundation. Noopur earned a B.S.in Kinesiology from the University of Michigan.

Session Details:

CASE STUDY: From bench to bedside: taking an orphan drug from the lab to a clinic

2023-09-14, 11:45 AM

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Jay Russak
Senior Director, Clinical Operations, Keros Therapeutics

Session Details:

New solutions and tactics for enrolling patients in rare disease trials

2023-09-14, 1:30 PM

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Kristy Galante
Senior Director, Janssen R&D

Kristy Galante is a Senior Director at Janssen R&D focusing on Rare Disease in Immunology.  Kristy has over 20 years of clinical trial experience where she has focused on both prevalent disease and now primarily on rare disease.  Kristy is seen as an expert in supporting complex clinical trial operations and advocates for the site and patient voice in trial development.  With a strong passion for innovation, Kristy has been able to support new clinical trial solutions with successful launch across the Immunology portfolio.  Kristy has also had leadership roles in Janssen Strategic Partnerships focusing on CRO Governance, Process and Performance Metrics.  Kristy supported the Johnson and Johnson COVID vaccine program ensuring supplier alignment and deploying many novel innovative tactics.  Kristy has her MS from Temple University School of Pharmacy.

Session Details:

CLOSING KEYNOTE PANEL: Looking to 2024 and beyond: what’s on the horizon for rare disease and orphan drug trials?

2023-09-14, 2:00 PM

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Plan Your Visit

Venue

Princeton Marriott at Forrestal, 100 College Road East Princeton, New Jersey 08540

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A FANTASTIC NETWORK OF YOUR PEERS AND COLLEAGUES!

With over 40 solution providers, this conference will be a perfect platform to reconnect with current business partners, meet new vendors and discover which new technologies are being employed to improve your Rare Disease clinical trials.

 

See What It's All About

Enquiry

Contact Us

SPONSORSHIP ENQUIRIES

Ben Lloyd-Davies

Sponsorship Manager

+44 207866 9497

SPEAKING OPPORTUNITIES

Louisa Manning

Programme Director

+44 (0) 207 661 0906

MARKETING ENQUIRIES

Moona Popal

Marketing Manager

SPONSORSHIP ENQUIRIES

To enquire about sponsorship opportunities for the conference, please contact:

Ben Lloyd-Davies

Sponsorship Manager


+44 207866 9497

SPEAKER ENQUIRIES

To enquire about speaking opportunities for the conference, please contact:

Louisa Manning

Programme Director


+44 (0) 207 661 0906