3rd Annual Clinical Trials in Rare Diseases 2025

Clinical Trials in Rare Diseases will take place on 17th-18th September 2025!

17 - 18

September

2025
  • Marriott Princeton at Forrestal, Princeton, USA
  • Complimentary
  • Why attend?
  • Agenda
  • Speakers
  • Plan Your Visit
  • Event Gallery
  • Sponsors
  • Media Centre
  • Why partner?
  • Contact Us
close

2025 Sponsors Included:

Back

Company Information

Founded in 1997, Ergomed is a full service, global Phase I-IV clinical development and trial management service partner with leading expertise in developing oncology and rare disease drugs.  Our portfolio includes experience working with novel therapies, early phase development, immunotherapy, and adaptive trial designs. Ergomed embodies a patient-centric approach to recruitment and retention with access to global patients in vital geographical areas.  Ergomed’s proven delivery record, expertise, and true partnership means our sponsors can trust us to deliver their oncology trials whatever the challenge. We also offer an industry-leading suite of specialist pharmacovigilance (PV) solutions under the PrimeVigilance brand.

Ergomed has supported and managed clinical development, trial management, pharmacovigilance, and medical information services for over 300 clients in more than 100 countries.

We deliver a complete solution for oncology trials of all sizes and complexity. For more information about Ergomed, please visit: www.ergomedcro.com

Company Website

To learn more, please visit our website - www.ergomedcro.com

Next Sponsor
Back

Company Information

Greenphire is the industry leader of clinical payment technology, designed to improve the way research professionals work. We leverage our proprietary payments platform and workflow automation to help clients improve operational efficiency, reduce costs, mitigate regulatory risks, improve the patient and site experience and produce quantifiable results that improve clinical operations and strategic planning.

Company Website

To learn more, please visit our website - http://greenphire.com/

Next Sponsor
Back

Company Information

Biorasi is a global contract research organization (CRO) delivering fast and flexible solutions across clinical trials to maximize speed-to-market for its sponsors. As the leader in clinical trials for dermatology, neurology, and oncology, Biorasi sets new benchmarks for speed, agility, and quality in patient enrollment, decentralized trials, and data sciences.

Next Sponsor
Back

Company Information

LabConnect is the world’s most agile central laboratory solutions partner supporting clinical trials of all sizes and complexity. With modern operations and strategic alliances with world-leading laboratories, LabConnect is uniquely positioned to provide access to the latest innovation and automation. LabConnect is the trusted partner for biotechnology, pharmaceutical, and clinical research organizations.

 

Follow LabConnect on LinkedIn

Learn more at labconnect.com

Company Website

To learn more, please visit our website - http://www.labconnectllc.com/

Next Sponsor
Back

Company Information

Prevail InfoWorks improves the probability of clinical trial success by providing stakeholders real-time actionable intelligence to effectively manage endpoints and key deliverables with the Single Interface™, its patented cloud-based tool that aggregates, reconciles, analyzes and single-view reports/visualizes all clinical, operational and project accounting data, regardless of the source or format.

Company Website

To learn more, please visit our website - www.prevailinfoworks.com

Next Sponsor
Back

Company Information

Saama Technologies is the advanced clinical data and analytics company, unleashing wisdom from data to deliver better actionable business outcomes for the life sciences industry. Saama’s unified, AI-driven clinical data analytics cloud platform seamlessly integrates, curates, and animates unlimited sources of structured, unstructured, and real-world data to deliver actionable insights across all therapeutic areas. The award-winning platform gives unprecedented real-time visibility into clinical data, enabling sponsors to file New Drug Applications (NDAs) more efficiently to bring drugs to market faster and at lower costs. For more information, visit http://www.saama.com.

Company Website

To learn more, please visit our website - www.saama.com

Next Sponsor
Back

Company Information

eClinical Solutions is a leading global provider of cloud-based enterprise software and software-driven clinical data services. The elluminate® Clinical Data Cloud provides life sciences companies with greater control of their clinical trial data with one platform for all data sources, from aggregation through to standardization, visualization, and preparation for submission. elluminate software and data driven services have been used by more than 100 life sciences companies on over 500 clinical trials to reduce cycle time and improve data quality. For more information, visit www.eclinicalsol.com, or contact us at info@clinicalsol.com.

Company Website

To learn more, please visit our website - www.eclinicalsol.com

Next Sponsor
Back

Company Information

At Viedoc, we design engaging software that modernizes clinical research so that necessary treatments can reach the people who need them faster. We believe in life and science, in people, and in our collective power to change the world and build a healthier future. That unmatched driving force is what pushes us to innovate, accelerate and improve every aspect of modern clinical studies.

Company Website

To learn more, please visit our website - www.viedoc.com

Next Sponsor
Back

Company Information

When it comes to transforming the clinical trials landscape, Replior does it by offering a full suite of tools designed specifically for all stakeholders but with a special focus on the patient. From an established EDC to an engaging best-in-class ePRO connected to Virtual Visits and eConsent, Trial Online Suite supports all phases of clinical trials in most therapeutical areas, collaborating tightly through trust and transparency with large pharmaceuticals to smaller biotech companies.

Company Website

To learn more, please visit our website - https://www.replior.com/

Next Sponsor
Back

Company Information

Egnyte combines the power of cloud content management, data security, and AI into one intelligent content platform. More than 22,000 customers trust Egnyte to improve employee productivity, automate business processes, and safeguard critical data, in addition to offering specialized content intelligence and automation solutions across industries, including architecture, engineering, and construction (AEC), life sciences, and financial services.

Company Website

To learn more, please visit our website - https://www.egnyte.com/solutions/life-sciences

Next Sponsor
Back

Company Information

MMS Holdings (MMS) is an innovative, data focused CRO that supports the pharmaceutical and biotech industries with a proven, scientific approach to complex trial data and regulatory submission challenges. Strong industry experience, technology-enabled services, and a data-driven approach to drug development make MMS a valuable CRO partner, creating compelling submissions that meet rigorous regulatory standards. With a global footprint across four continents, MMS maintains a 97 percent customer satisfaction rating. For more information, visit www.mmsholdings.com

Company Website

To learn more, please visit our website - www.mmsholdings.com

Next Sponsor

    Why attend?

    Our 3rd Annual Clinical Trials in Rare Diseases will take place on 17th-18th September 2025.

    This event will explore the challenges in clinical trials for orphan drugs and rare diseases, bringing together clinical development and clinical operations professionals to discuss common obstacles and solutions. 

    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.

    100+

    Attendees

    		30+

    Exhibitors
    100+

    Attendees

    		30+

    Exhibitors

    Testimonials

    See What It's All About

    Agenda

    • 17 Sep 2025
    • 18 Sep 2025
    Expand All

    8 AM

    Registration and refreshments

    8:50 AM

    Chairperson’s opening remarks

    Speakers

    John Seman
    Chief Executive Officer, REVITALE Pharma

    9 AM

    Case Study: Cutting edge technology – using AI in rare diseases

    • Sharing insights on how to use AI to bring drugs to rare disease patients
    • Running through step by step processes including pre IND, FDA work, organising and conducting trial
    • Discussing challenges, successes and real-world experience

    Speakers

    Bruce Bloom
    Chief Collaboration Officer, Healx

    9:30 AM

    Reserved for event sponsor

    10 AM

    Case study: Tackling patient recruitment to ensure your trial is delivered on time

    • Considering ways to boost recruitment through investigator meetings and recruitment workshops
    • Communicating effectively and working together with sites to assist in recruitment
    • Sharing recruitment challenges and successes from a rare disease trial that started during Covid-19 pandemic

    Speakers

    Jamie Switzer
    Associate Director Clinical Operations, Insmed

    10:30 AM

    Reserved for event sponsor

    11 AM

    Morning refreshments and networking

    11:30 AM

    Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?

    • Finding innovative solutions for patient engagement
    • Collaborating with advocacy groups, patient organizations and care givers to extend patient reach
    • Utilizing decentralized trials with remote visits and monitoring to reduce patient burden where possible

    Moderator: Anka Ehrhardt, Director Cell-Based Assays / Analytical Research & Development, Merck

    Speakers

    Anka Ehrhardt
    Director Cell-Based Assays / Analytical Research & Development, Merck
    Peter Covitz
    Executive Director, Head Digital Innovation, Data Generation & Managed Access, Alexion
    Jamie Switzer
    Associate Director Clinical Operations, Insmed
    Gurdyal Kalsi
    Chief Medical Officer, Asklepion Pharmaceuticals
    Tiara Johnson
    Associate Director Global Patient Affairs Program Lead and Regulatory Patient Engagement, BioMarin

    12:15 PM

    Reserved for event sponsor

    12:45 PM

    Running decentralized rare disease trials: challenges and benefits

    • Comparing outcomes of a DCT model versus non DCT model
    • Sharing benefits and lessons learned from experiences within rare disease DCTs

    Speakers

    Maria Dunaway-Bryant
    Director Clinical Operations - Neuroscience & Oncology, Jazz Pharmaceuticals
    Jennifer Oertli
    Senior Clinical Trials Manager, Jazz Pharmaceuticals
    Kasia Wajer
    Senior Clinical Trial Manager, Jazz Pharmaceuticals

    1:15 PM

    Lunch and networking

    2:15 PM

    Navigating clinical trials for rare diseases: hurdles and opportunities

    • Best practice in ensuring your rare disease trial is a success
    • Using precision medicine in clinical trials to advance therapeutics for rare diseases
    • Overcoming limited data sets and patient pools

    Speakers

    Gurdyal Kalsi
    Chief Medical Officer, Asklepion Pharmaceuticals

    2:45 PM

    Engaging with advocacy groups to aid with trial design, patient enrolment and patient engagement

    • Leveraging advocacy experience when designing trials with patient centric focus
    • Partnering with patience advocacy organisations to educate regarding value of trial participation
    • Sharing best practice in engaging advocacy groups early to drive trial awareness and ensure trial success

    Speakers

    Eve Dryer
    Board Member, NephCure

    3:15 PM

    Afternoon refreshments and networking

    3:45 PM

    Case Study: Discussing an approach to quality and clinical operations oversight in a rare disease trial at a small biotech

    • Deciding the right level of oversight: key considerations
    • Addressing communication hurdles and how to overcome them
    • Supporting sites for operational improvement and inspection readiness

    Speakers

    Amy DeWerth
    Senior Director Regional Clinical Lead, Pharvaris
    Vinay Edwin
    Director Clinical Quality, Pharvaris

    4:15 PM

    Case Study: Sharing success story of rare brain tumor clinical trial

    • Running the trial: innovative approaches and overcoming challenges
    • Treating with oncolytic virus intra-tumoral therapy
    • Reaching clinical success: findings and future plans

    Speakers

    Ashok Srivastava
    Co-Founder & Chief Medical Officer, rTR Technovation Corporation

    4:45 PM

    Panel Discussion: Looking at the future of clinical trials in rare diseases: where are we now and where are we headed

    • Considering advances in trial designs and master protocols
    • Innovating through technology, digital health tools, AI and machine learning
    • Expanding collaborations and partnerships
    • Addressing ethical and diversity challenges
    • Discussing long-term vision in rare disease therapy development

     

    Speakers

    Anka Ehrhardt
    Director Cell-Based Assays / Analytical Research & Development, Merck
    Bruce Bloom
    Chief Collaboration Officer, Healx

    5:30 PM

    Chairperson’s closing remarks

    Speakers

    John Seman
    Chief Executive Officer, REVITALE Pharma

    END OF DAY 1 AND NETWORKING DRINKS

    8 AM

    Registration and refreshments

    8:50 AM

    Chairperson’s opening remarks

    9 AM

    Interactive Speaker-Hosted Roundtable Discussions

    Interactive roundtable sessions offer a unique opportunity to come together with your peers to share best practice and develop solutions to critical challenges facing the industry as a whole. Each discussion will be led by a table moderator and will focus on a different challenge within rare disease clinical trials.

    Roundtables are an exciting, interactive way to build your personal network and learn from the experience and expertise of others. After 45 minutes, delegates will have the opportunity to swap and choose a different table, and each roundtable will run twice.

     

    Roundtable 1: Sharing experiences and highlighting importance of decentralizing in rare disease trials

    Leigh Dallow, Patient Advocate & Caregiver

    Roundtable 2: Overcoming patient retention challenges in rare disease trials

    Jenifer Waldrop, Executive Director, Rare Disease Diversity Coalition

     

    Roundtable 3: Using new technologies in rare disease trials

    Bruce Bloom, Chief Collaboration Officer, Healx

    Speakers

    Leigh Dallow
    Patient Advocate & Caregiver
    Jenifer Waldrop
    Executive Director, Rare Disease Diversity Coalition
    Bruce Bloom
    Chief Collaboration Officer, Healx

    10:30 AM

    Morning refreshments and networking

    11 AM

    Participating and care giving in a rare disease trial: a family perspective

    • Understanding how living with a rare disease impacts decision on enrolling in a trial
    • Sharing experience of participating in trial from start to finish
    • Considering hurdles and barriers and what biopharma can do to relieve patient burden and improve access to studies

    Speakers

    Leigh Dallow
    Patient Advocate & Caregiver

    11:30 AM

    Reserved for event sponsor

    12 PM

    Medically underserved populations in rare disease research

    • Discussing population inclusion and access to care and clinical trials regardless of diversity and socioeconomics, with a focus on improving patient care
    • Mental health and well-being: the emotional toll rare diseases take on patients and caregivers
    • Insights on the differences between represented and underrepresented populations on their barriers to care
    • Identifying realistic ways to improve opportunities for all patients

    Speakers

    Jenifer Waldrop
    Executive Director, Rare Disease Diversity Coalition

    12:30 PM

    Lunch and networking

    1:30 PM

    Fireside Chat: Selecting a CRO for a rare disease trial: key considerations

    • Assessing experience in trials with small patient populations an recruitment challenges
    • Evaluating ability to adapt and innovate for unique needs of rare disease trials
    • Considering regulatory knowledge and patient access

    Speakers

    Bruce Bloom
    Chief Collaboration Officer, Healx
    Paul Lupo
    Senior Director Registry Operations, PTC Therapeutics

    2 PM

    Development strategies for orphan-designated programs

    • Understanding regulatory pathways and incentives
    • Identifying clinical development challenges and solutions
    • Considering pricing, reimbursement and patient advocacy engagement

    Speakers

    Venkat Nelabhotla
    Co-Founder, President & CEO, Vyome Therapeutics

    2:30 PM

    Fireside Chat: Contracting clinical sites for rare disease trials: accelerating timelines

    • Attracting an academic organization’s attention to prioritize your need
    • Considerations for contract negotiations and speeding up processing time scales
    • Building relationships and providing two-way support

    Speakers

    Kinjal Patel
    Director Medical Affairs – Operations, Mitsubishi Tanabe Pharma America
    Janine Sampong
    Senior Manager Medical Affairs – Operations, Mitsubishi Tanabe Pharma America

    3 PM

    Afternoon break, networking and prize draw

    3:30 PM

    Case study: Developing a company and setting up rare disease trials: challenges and successes

    • Creating a company and ensuring the brand becomes known
    • Leveraging patient advocacy as part of enrolment strategy
    • Learnings from first trial challenges to improve in next study

    Speakers

    Niha Kamat
    Senior Director Clinical Operations, Tenaya Therapeutics

    4 PM

    Overcoming challenges and optimizing a phase II rare disease trial using data and technology: a case study

    • Understanding successful protocol design trends and competitive activity in the rare disease space
    • Utilizing data analytics and benchmark models for assessing geographic feasibility, timelines, enrolment, and trials cost forecasting
    • Improving subject accrual and the site selection process
    • Execution phase and beyond – tracking investigators and competitive trials, mitigating potential complications

     

    Senior Representative, GlobalData

    4:30 PM

    END OF CONFERENCE

    Speakers

    Select a speaker to learn more

    Back
    Peter Covitz
    Executive Director, Head Digital Innovation, Data Generation & Managed Access, Alexion

    Session Details:

    Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?

    2025-09-17, 11:30 AM

    View In Agenda
    Next speaker
    Back
    Gurdyal Kalsi
    Chief Medical Officer, Asklepion Pharmaceuticals

    Session Details:

    Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?

    2025-09-17, 11:30 AM

    Session Details:

    Navigating clinical trials for rare diseases: hurdles and opportunities

    2025-09-17, 2:15 PM

    View In Agenda
    Next speaker
    Back
    Tiara Johnson
    Associate Director Global Patient Affairs Program Lead and Regulatory Patient Engagement, BioMarin

    Session Details:

    Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?

    2025-09-17, 11:30 AM

    View In Agenda
    Next speaker
    Back
    Bruce Bloom
    Chief Collaboration Officer, Healx

    Dr. Bruce Bloom is Chief Collaboration Officer of Healx, a Cambridge UK biotech using AI and drug redevelopment to create novel therapies for rare disease patients.  He is the founder and former CEO of the global charity Cures Within Reach that brought over a dozen redeveloped drug therapies to patients through proof of concept clinical trials, and developed CureAccelerator®, the online drug redevelopment collaboration marketplace.  Dr. Bloom is an Ashoka Social Entrepreneur Fellow, the Patient Advisory Board Chair for the Institute for Translational Medicine, board member of the Drug Discovery Center and Chair of the Cancer Center Advancement Advisory Boards at the University of Illinois Chicago, member of the Vanderbilt Institute for Clinical and Translational Research External Advisory Board, Executive Board member of Mission: Cure, and is on the Science Advisory Boards of Rediscovery Life Sciences, the Dr. Ralph and Marian Falk Medical Research Trust Awards Programs, the Findacure Fundamental Disease Charity, the Rare Disease Research Hub of the Westchester Biotech Project, OneThree Biotech, and ReBootRx, a member of the IRDiRC Working Group on Rare Disease Treatment Access and the editorial board of ASSAY and Drug Development Technologies.

    Session Details:

    Case Study: Cutting edge technology – using AI in rare diseases

    2025-09-17, 9:00 AM

    Session Details:

    Interactive Speaker-Hosted Roundtable Discussions

    2025-09-18, 9:00 AM

    Session Details:

    Panel Discussion: Looking at the future of clinical trials in rare diseases: where are we now and where are we headed

    2025-09-17, 4:45 PM

    Session Details:

    Fireside Chat: Selecting a CRO for a rare disease trial: key considerations

    2025-09-18, 1:30 PM

    View In Agenda
    Next speaker
    Back
    Jamie Switzer
    Associate Director Clinical Operations, Insmed

    Session Details:

    Case study: Tackling patient recruitment to ensure your trial is delivered on time

    2025-09-17, 10:00 AM

    Session Details:

    Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?

    2025-09-17, 11:30 AM

    View In Agenda
    Next speaker
    Back
    Maria Dunaway-Bryant
    Director Clinical Operations - Neuroscience & Oncology, Jazz Pharmaceuticals

    Session Details:

    Running decentralized rare disease trials: challenges and benefits

    2025-09-17, 12:45 PM

    View In Agenda
    Next speaker
    Back
    Jennifer Oertli
    Senior Clinical Trials Manager, Jazz Pharmaceuticals

    Session Details:

    Running decentralized rare disease trials: challenges and benefits

    2025-09-17, 12:45 PM

    View In Agenda
    Next speaker
    Back
    Kasia Wajer
    Senior Clinical Trial Manager, Jazz Pharmaceuticals

    Session Details:

    Running decentralized rare disease trials: challenges and benefits

    2025-09-17, 12:45 PM

    View In Agenda
    Next speaker
    Back
    Anka Ehrhardt
    Director Cell-Based Assays / Analytical Research & Development, Merck

    Dr. Ehrhardt is a biophysicist with a Ph.D. in human physiology. Throughout her career, she focused on driving biomedical research through efficient application of innovative technologies and strategies. She built and led successful teams in industry, including at Merck, where she headed a laboratory providing leading technologies for cell-based research from early discovery through manufacturing; and at BMS where Dr. Ehrhardt led and grew an international clinical assay team to robustly cover large scale registrational and translational clinical analysis generating clinical decision-driving data and mechanistic insights for immuno-oncology, cardiovascular, immunology, metabolic and rare disease studies. Before returning to Merck, Dr. Ehrhardt fulfilled one of her scientific aspirations at CHDI Foundation, designing and implementing successful clinical biomarker discovery and development strategies for Huntington’s disease. Currently, at Merck, Dr. Ehrhardt is providing strategic and technical leadership for the development and implementation of industry-leading and QC-friendly cell-based assays and models for potency determination that reflect the unique mechanisms of biologics products in clinical studies and beyond.

    Session Details:

    Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?

    2025-09-17, 11:30 AM

    Session Details:

    Panel Discussion: Looking at the future of clinical trials in rare diseases: where are we now and where are we headed

    2025-09-17, 4:45 PM

    View In Agenda
    Next speaker
    Back
    Kinjal Patel
    Director Medical Affairs – Operations, Mitsubishi Tanabe Pharma America

    Session Details:

    Fireside Chat: Contracting clinical sites for rare disease trials: accelerating timelines

    2025-09-18, 2:30 PM

    View In Agenda
    Next speaker
    Back
    Janine Sampong
    Senior Manager Medical Affairs – Operations, Mitsubishi Tanabe Pharma America

    Session Details:

    Fireside Chat: Contracting clinical sites for rare disease trials: accelerating timelines

    2025-09-18, 2:30 PM

    View In Agenda
    Next speaker
    Back
    Eve Dryer
    Board Member, NephCure

    Session Details:

    Engaging with advocacy groups to aid with trial design, patient enrolment and patient engagement

    2025-09-17, 2:45 PM

    View In Agenda
    Next speaker
    Back
    Leigh Dallow
    Patient Advocate & Caregiver

    Session Details:

    Interactive Speaker-Hosted Roundtable Discussions

    2025-09-18, 9:00 AM

    Session Details:

    Participating and care giving in a rare disease trial: a family perspective

    2025-09-18, 11:00 AM

    View In Agenda
    Next speaker
    Back
    Amy DeWerth
    Senior Director Regional Clinical Lead, Pharvaris

    Session Details:

    Case Study: Discussing an approach to quality and clinical operations oversight in a rare disease trial at a small biotech

    2025-09-17, 3:45 PM

    View In Agenda
    Next speaker
    Back
    Vinay Edwin
    Director Clinical Quality, Pharvaris

    Session Details:

    Case Study: Discussing an approach to quality and clinical operations oversight in a rare disease trial at a small biotech

    2025-09-17, 3:45 PM

    View In Agenda
    Next speaker
    Back
    Paul Lupo
    Senior Director Registry Operations, PTC Therapeutics

    Session Details:

    Fireside Chat: Selecting a CRO for a rare disease trial: key considerations

    2025-09-18, 1:30 PM

    View In Agenda
    Next speaker
    Back
    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:

    Interactive Speaker-Hosted Roundtable Discussions

    2025-09-18, 9:00 AM

    Session Details:

    Medically underserved populations in rare disease research

    2025-09-18, 12:00 PM

    View In Agenda
    Next speaker
    Back
    John Seman
    Chief Executive Officer, REVITALE Pharma

    John Seman is the CEO and Founder of REVITALE Pharma.  He began his career working in Big Pharma - Johnson & Johnson and Bristol Myers Squibb - in sales, marketing and business development.  The second half of his career was spent as a serial entrepreneur having Founded a half dozen companies that provided either products or services for the pharmaceutical industry.  John earned his bachelor's and master's degrees from the Massachusetts College of Pharmacy.

    Session Details:

    Chairperson’s opening remarks

    2025-09-17, 8:50 AM

    Session Details:

    Chairperson’s closing remarks

    2025-09-17, 5:30 PM

    View In Agenda
    Next speaker
    Back
    Ashok Srivastava
    Co-Founder & Chief Medical Officer, rTR Technovation Corporation

    Session Details:

    Case Study: Sharing success story of rare brain tumor clinical trial

    2025-09-17, 4:15 PM

    View In Agenda
    Next speaker
    Back
    Niha Kamat
    Senior Director Clinical Operations, Tenaya Therapeutics

    Session Details:

    Case study: Developing a company and setting up rare disease trials: challenges and successes

    2025-09-18, 3:30 PM

    View In Agenda
    Next speaker
    Back
    Venkat Nelabhotla
    Co-Founder, President & CEO, Vyome Therapeutics

    Session Details:

    Development strategies for orphan-designated programs

    2025-09-18, 2:00 PM

    View In Agenda
    Next speaker

    Plan Your Visit

    Venue

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

    Book Your Stay

    Accommodation

    Arena International Events Group “(Arena”) is aware of numerous third party agencies which are contacting Arena’s customers and purporting to have access to hotel room bookings and events attendee lists. For the avoidance of doubt, Arena will only share attendee lists with carefully selected third party partners for event registration purposes. Whilst Arena International Events Group pursues third party agencies for their potentially illicit activity, Arena encourages you to be cautious in engaging with such third party agencies and Arena shall not accept any responsibility for any losses incurred by you should you choose to engage with third parties that have no affiliation to Arena International Events Group.

    Sponsors

    Select a sponsor to learn more

    Featured Sponsor

    Session Sponsors

    Technology spotlight Sponsor

    coffee break sponsor

    Exhibitors

    Co-Sponsor

    Why partner?

     

    Contact us to become a partner

    Partnership Opportunities

    We work with you to design a bespoke package to meet your business needs, where you could:

    • Demonstrate your expertise on the agenda – have the attention of the entire audience. For greatest impact, why not position your talk before a networking break and immediately conduct follow up conversations with highly engaged attendees
    • Showcase your business and its services at an exhibition booth, which provides a perfect meeting place for prospective new clients
      Present the value proposition of your offering over your competitors, in front of an audience of active, engaged potential customers
    • Speak with key decision makers as part of our intimate, interactive roundtable style workshop sessions with over 8 hours allowed for networking

    Event reach

    • 100+ attendees each year
    • 80% attendees C-suite level

     

    Take a look at our current sponsors

    Media Centre

    Become A Media Partner

    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

    Conor Taylor

    Sponsorship Sales Manager

    +44 (0) 20 7936 6870

    SPEAKING OPPORTUNITIES

    Ruth Atterbury

    Clinical Trial Supply Portfolio Manager

    MARKETING ENQUIRIES

    Moona Popal

    Marketing Manager

    DELEGATE ENQUIRIES

    Sunny Saikia

    VIP Delegate Manager

    SPONSORSHIP ENQUIRIES

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

    Nick McCudden

    Commercial Director


    +61 280 978 126

    SPEAKER ENQUIRIES

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

    Ruth Atterbury

    Clinical Trial Supply Portfolio Manager