2nd Annual Clinical Trials in Rare Diseases 2024

We are pleased to announce the launch of a brand new event: Clinical Trials in Rare Diseases will take place on 10th-11th September 2024!

10 - 11

September

2024
  • Hyatt Regency San Francisco Airport, San Francisco, USA
  • Complimentary
  • Why attend?
  • Agenda
  • Speakers
  • Plan Your Visit
  • Sponsors
  • Media Centre
  • Event Gallery
  • Why partner?
  • Contact Us
close

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.

100+

Attendees

30+

Exhibitors

100+

Attendees

30+

Exhibitors

See What It's All About

Agenda

  • 10 Sep 2024
  • 11 Sep 2024
Expand All

8 AM

Registration and refreshments

8:50 AM

Chairperson’s opening remarks

Speakers

Revati Tatake
Global Head of Pharma Research, Analysis and Competitive Intelligence, GlobalData Plc

9 AM

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

  • Overview of current and past mRNA therapeutics technologies
  • Risks and issues to overcome for mRNA therapeutics in rare diseases
  • mRNA therapeutics’ applications beyond vaccines for infectious disease and cancer

Speakers

Aki Ko
Co-Founder & Chief Executive Officer, Elixirgen Therapeutics

9:30 AM

Reserved for event sponsor

10 AM

PANEL DISCUSSION Navigating a changing landscape: the future of rare disease treatments

  • Exploring shifts in rare disease treatments, focusing on gene therapy approaches
  • Discussing long-term thinking and forward vision in therapy development
  • Addressing challenges in clinical trial engagement and evolving trial structures

Moderator
Revati Tatake, Global Head of Pharma Research, GlobalData

Speakers

Revati Tatake
Global Head of Pharma Research, Analysis and Competitive Intelligence, GlobalData Plc
Aki Ko
Co-Founder & Chief Executive Officer, Elixirgen Therapeutics
Vij Senthilnathan
Associate Director, Clinical Operations, PTC Therapeutics
Hayley Mason
Policy Analyst, NORD

10:30 AM

Morning refreshments and networking

11:15 AM

Insights into barriers to care in the rare disease community

  • Challenges and nuances of rare disease patients’ journey to receive a diagnosis
  • Recognizing the pivotal role insurance plays in accessing healthcare services
  • Assessing adequacy of healthcare coverage, affordability of services, and the extent to which patients utilize available healthcare resources
  • Exploring overall experiences individuals have had within the healthcare system: strengths and areas for improvement
  • Mental health and well-being: the emotional toll rare diseases take on patients and caregivers

Speakers

Jenifer Waldrop
Executive Director, Rare Disease Diversity Coalition

11:45 AM

Reserved for event sponsor

12:15 PM

De-risking the clinical trial process in rare diseases

  • Innovative trial designs tailored for rare diseases: adaptive designs, basket trials.
  • Utilizing real-world evidence and novel endpoints in rare disease research.
  • Collaborating with advocacy groups, regulators, and industry for streamlined processes

Speakers

Hayley Lane
Vice President Clinical Operations, Star Therapeutics
Michelle Carpenter
Executive Vice President, Regulatory Affairs and Project Management, Star Therapeutics

12:45 PM

Reserved for technology spotlight sponsor

1 PM

Lunch and networking

2 PM

Selecting a CRO for rare disease trials: aspects to contemplate

  • Does possessing therapeutic expertise remain imperative in choosing a CRO for a rare disease inquiry?
  • Evaluating the advantages of niche specialized CROs versus expansive multinational CROs
  • Extra hurdles in the CRO selection procedure arise when addressing ultra-rare diseases, where CROs may lack familiarity with the condition
  • Discussing the merits and drawbacks of engaging a comprehensive service CRO for your rare disease examination versus collaborating with various vendors across the study

Speakers

Stephen Smith
Senior Program Director, Oncology, Corcept Therapeutics

2:30 PM

Reserved for event sponsor

3 PM

Designing and running a clinical trial for rare pediatric cancers: overcoming challenges when working with younger patients

  • Tailoring trial protocols to accommodate the unique needs and vulnerabilities of pediatric cancer patients
  • Implementing effective communication strategies to engage both parents and children in the trial process
  • Addressing regulatory and ethical considerations specific to pediatric populations to ensure patient safety and compliance

Speakers

Anthony Maida
Chief Clinical Officer, Oncotelic

3:30 PM

Afternoon refreshments and networking

4 PM

Using AI methods to enable clinical trial success

  • Increasing Clinical Trial success rate by better patient pre qualification
  • Tumor boards provide personalized advice and pre qualify patients.
  • Hackathons formalize, scale and tune the tumor board process
  • Working to fully automate Hackathons

Speakers

Bill Paseman
Co-Founder and Patient Advocate, Rare Kidney Cancer

4:30 PM

Reserved for event sponsor

5 PM

PANEL DISCUSSION Discovering ways to overcome enrollment challenges in rare disease clinical trials

  • Discussing the impact of eligibility criteria and small patient population
  • Exploring innovative approaches to recruit eligible participants, including leveraging digital platforms and social media outreach
  • Highlighting the significance of collaboration between researchers, clinicians, patient advocacy groups, and regulatory bodies in overcoming enrollment challenges
  • Strategies for navigating the complexities of rare diseases to ensure accurate diagnosis, patient identification, and enrollment

Moderator
Revati Tatake, Global Head of Pharma Research, GlobalData

Speakers

Revati Tatake
Global Head of Pharma Research, Analysis and Competitive Intelligence, GlobalData Plc
Anthony Maida
Chief Clinical Officer, Oncotelic
Stephen Smith
Senior Program Director, Oncology, Corcept Therapeutics
Kenneth Harvey
Director Clinical Operations, Epic Bio
Nadine Knowles
Vice President, Clinical Operations, Longboard Pharmaceuticals

5:30 PM

Chairperson’s closing remarks

Speakers

Amanda Murphy
Senior Director, Data Intelligence & Solutions, GlobalData

END OF DAY 1 AND NETWORKING DRINKS

8 AM

Registration and refreshments

8:50 AM

Chairperson’s opening remarks

Speakers

Revati Tatake
Global Head of Pharma Research, Analysis and Competitive Intelligence, GlobalData Plc

9 AM

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

Hayley Mason
Policy Analyst, NORD

9:30 AM

FIRESIDE CHAT Navigating the IVDR landscape: impact on rare disease studies and diagnostics use in clinical trials

  • Analyzing the specific regulatory challenges posed by the IVDR for rare disease studies and the implications for trial design and execution
  • Exploring the crucial role of diagnostics in rare disease clinical trials, including their use for patient stratification, biomarker identification, and treatment assessment
  • Addressing the need for streamlined processes and collaborative efforts to reduce the burden on test development for diagnostics used in rare disease studies, ensuring efficiency and compliance

Speakers

Revati Tatake
Global Head of Pharma Research, Analysis and Competitive Intelligence, GlobalData Plc
Nicole Miller
PhD, Vice President of Molecular Diagnostics at Ultragenyx

10 AM

CASE STUDY: Patient-centric paradigm: enhancing rare disease research through collaboration and engagement

  • Involving patients in trial design to ensure that trial outcomes are relevant and meaningful to patients
  • Patient-centric approach: integrating patient feedback and needs into research
  • Building a collaborative network

Speakers

Amanda Moore
CEO, Angelman Syndrome Foundation

10:30 AM

Morning refreshments and networking

11:15 AM

FIRESIDE CHAT Innovative funding models for treatment of rare diseases

  • Strategies for increasing government funding, collaboration among stakeholders, and international cooperation
  • Exploring different sources of funding for the study of specific rare diseases
  • How closing the funding gap in rare diseases requires a multi-faceted approach

Speakers

Revati Tatake
Global Head of Pharma Research, Analysis and Competitive Intelligence, GlobalData Plc
Aditi Kantipuly
MD, MS, Physician, Visiting Scholar University of New Mexico

11:45 AM

Possible integration of machine learning for data cleaning in rare disease clinical trials

  • Comparison of history of data cleaning in other industries (i.e., marketing) vs Pharma and Biotech, including rare disease clinical trials
  • Use of machine learning techniques for data cleaning in other industries
  • Discuss the unique challenges of data cleaning in rare disease research
  • Explain learnings for data cleaning between other industries and Pharma/Biotech, including rare disease clinical trials

Speakers

Katrina Paz
Director, Clinical Programming, Denali Therapeutics
Nasser Al-Ali
Senior Clinical Programmer, Denali Therapeutics

12:15 PM

An overview of rare disease clinical trials

  • Understanding the global clinical trial landscape of rare diseases
  • What therapy area and indications have the most rare disease clinical trials
  • Determine who the top sponsors are for rare disease clinical trials

Speakers

Scotty Chung-Siu
Managing Analyst, Clinical Trials Intelligence, GlobalData

12:45 PM

Engaging patients beyond just participating in a trial: the role of biopharma companies in supporting patients

  • Where rare disease trial sponsors can step in to fill scientific advice gap and teach patients about their condition
  • The importance of making patients feel cared for throughout and beyond trial participation
  • How rare disease trials differ from other clinical trials when it comes to supporting patients in understanding and managing their disease long term

Speakers

Katia Stakhiv
Associate Director, Research and Development, Rare Diseases, Amgen

1:15 PM

Lunch, networking and prize draw!

1:50 PM

EXHIBITION APPLE PRIZE DRAW

Visit our exhibitors’ booths throughout the day and collect stamps in order to enter our Prize Draw and be in for a chance of winning Apple devices or Amazon vouchers. The Prize Draw will take place in the Exhibition Hall and you will need to be physically present to be eligible for a prize. Make sure you don’t miss out!

2:15 PM

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 clinical data management. After 45 minutes, delegates will have the opportunity to swap and choose a different table, and each roundtable will run twice.

ROUNDTABLE 1
Discovering how to operationalize clinical supply chain for an autologous cell therapy program for a rare disease
Matthew Birmingham, Head Clinical Supply, Prime Medicine

ROUNDTABLE 2
Overcoming challenges in patient identification for ultra rare diseases
Kenneth Harvey, Director Clinical Operations, Epic Bio

ROUNDTABLE 3
Exploring strategies for building greater DE&I in rare disease trials
Aditi Kantipuly, MD, MS, Physician, Visiting Scholar University of New Mexico

ROUNDTABLE 4
Empowering patient access in rare diseases through real-world evidence integration: strategies and insights
Katia Stakhiv, Associate Director, Research and Development, Rare Diseases, Amgen

Speakers

Matthew Birmingham
Head Clinical Supply, Prime Medicine
Kenneth Harvey
Director Clinical Operations, Epic Bio
Aditi Kantipuly
MD, MS, Physician, Visiting Scholar University of New Mexico
Katia Stakhiv
Associate Director, Research and Development, Rare Diseases, Amgen

3:15 PM

Close of conference

END OF CONFERENCE

Speakers

Select a speaker to learn more

Back
Aki Ko
Co-Founder & Chief Executive Officer, Elixirgen Therapeutics

Aki 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 the University of Virginia, where he cultivated his interest in biology.

Session Details:

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

2024-09-10, 9:00 AM

Session Details:

PANEL DISCUSSION Navigating a changing landscape: the future of rare disease treatments

2024-09-10, 10:00 AM

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.

Next speaker
Back
Bill Paseman
Co-Founder and Patient Advocate, Rare Kidney Cancer

After being diagnosed with a meningioma and p1RCC (a rare kidney cancer) in 2014, Bill co-founded rarekidneycancer.org with Dr. James Hsieh in 2016.  In 2018 and 2020, with Pete Kane, he brought together 17 teams of more than 100 researchers who used his medical data to determine “genes of interest” related to his disease.  He applied ensemble reasoning to evaluate the results of these hackathons (which Bill calls “gamified tumor boards” ) to discover better ways to do cancer research.  Bill is currently automating these “gamified tumor boards” using Large Language Models (LLMs).  Bill is also involved in several patient organizations and is a CDMRP reviewer.

Prior to rarekidneycancer.orgBill worked with Sabrina Paseman to use non-invasive Blood fluorescence to detect iron deficiency anemia (Patent 8,306,594) and with Katherine Paseman to expand Pulse oximetry technology to do non-invasive measurement of Hemoglobin and Hematocrit 

Bill’s 50 year career in AI started at age 18, when he worked with Howie Johnson at Rice University using n-grams (like chatGPT) to do classical music composition.  He continued his music work at MIT in Steve Ward’s lab exploring generative grammars and constraints.  He then applied his Symbolic AI knowledge to Electrical Design Automation as the 16th employee at Daisy Systems. Bill next founded Atherton Technology (which failed) and then Calico Commerce (which IPOed), where he repurposed his music composition system to do sales configuration over the internet (Patent 5,745,765).  After Bill took Calico public, he retired and, as discussed above, has dedicated his time to rarekidneycancer.org, patient advocacy and working with his daughters on non-invasive blood analysis.

Session Details:

Using AI methods to enable clinical trial success

2024-09-10, 4:00 PM

View In Agenda
Next speaker
Back
Amanda Moore
CEO, Angelman Syndrome Foundation

Amanda Moore has served as the CEO of the Angelman Syndrome Foundation since 2019. With a background in nonprofit leadership and development, Amanda brings a wealth of experience to her role, having previously worked extensively with the YMCA.

In 2015, Amanda and her husband Adam were blessed with the joyous addition of twin boys, Jackson and Baden. Their world changed in 2016 when Jackson was diagnosed with Angelman Syndrome, sparking Amanda's unwavering commitment to advocacy and support for individuals like her son.

Driven by her personal experiences and a deep desire to make a difference, Amanda immersed herself in patient advocacy. She served on the Angelman Syndrome Board for one year before assuming her current position as CEO, where she continues to lead the foundation with passion, dedication, and a relentless pursuit of advancing the lives of those affected by Angelman Syndrome. Her advocacy efforts extend beyond her professional role, as she actively engages in initiatives aimed at raising awareness, promoting research, and advocating for improved care and support for individuals with Angelman Syndrome and their families.

Session Details:

CASE STUDY: Patient-centric paradigm: enhancing rare disease research through collaboration and engagement

2024-09-11, 10:00 AM

View In Agenda
Next speaker
Back
Hayley Lane
Vice President Clinical Operations, Star Therapeutics

Session Details:

De-risking the clinical trial process in rare diseases

2024-09-10, 12:15 PM

View In Agenda
Next speaker
Back
Michelle Carpenter
Executive Vice President, Regulatory Affairs and Project Management, Star Therapeutics

Session Details:

De-risking the clinical trial process in rare diseases

2024-09-10, 12:15 PM

View In Agenda
Next speaker
Back
Anthony Maida
Chief Clinical Officer, Oncotelic

Dr. Maida is the Chief Clinical Officer for Oncotelic, Inc. and its joint venture, SapuBioscience, LLC, focused on immunotherapy of cancer and infectious disease. He has over 30 years of experience in clinical research in oncology and has worked with many Center of Excellence and Key Opinion Leaders. He is responsible for overseeing the operations of the company and has experience in finance, operations, research, and commercial clinical and scientific development, regulatory, and manufacturing for various therapeutic modalities. Previously, he served as Vice President of Clinical Research and General Manager, Oncology, World-wide for PharmaNet, Inc., and President and CEO of Replicon NeuroTherapeutics. He raised or assisted in financings in excess of $200 million for start-up and emerging biotechnology companies and negotiated licensing agreements with various centers of excellence and premier pharmaceutical firms. Over the past 18 years, he has served in various executive roles, including Chairman, CEO, COO, CSO, CFO, and business development. He also served as Senior Financial Controller of Lockheed Missiles, a $1.7 billion division.

Session Details:

Designing and running a clinical trial for rare pediatric cancers: overcoming challenges when working with younger patients

2024-09-10, 3:00 PM

Session Details:

PANEL DISCUSSION Discovering ways to overcome enrollment challenges in rare disease clinical trials

2024-09-10, 5:00 PM

View In Agenda
Next speaker
Back
Nicole Miller
PhD, Vice President of Molecular Diagnostics at Ultragenyx

Nicole Miller, PhD is the Vice President of Molecular Diagnostics at Ultragenyx, a patient-focused biotech company led by Emil Kakkis that develops therapies for rare genetic disease. She focuses on advancing biochemical and genetic testing in support of reducing the diagnostic odyssey for rare disease patients. She has partnered to develop and launch ten gene locus specific databases and published several variant reviews for rare genetic disorders.  Prior to Ultragenyx, Nicole developed and led a similar function at BioMarin for about 9 years, focusing on rare disease patient diagnosis. Since 2010, Nicole has worked closely with the newborn screening and sequencing community in support of improving newborn screening for rare disease.  Prior to working in the rare disease field, she led a molecular pharmacology research team at Exelixis and led a biomarker program there for about a decade supporting research through clinical development. Nicole has a bachelor’s degree in genetics from U.C. Davis, a PhD in oncology from U.W. Madison and her postdoc was at UCSF. She sits on the board of the Professional Science Master’s program at the University of San Francisco since the program’s inception.

Session Details:

FIRESIDE CHAT Navigating the IVDR landscape: impact on rare disease studies and diagnostics use in clinical trials

2024-09-11, 9:30 AM

View In Agenda
Next speaker
Back
Stephen Smith
Senior Program Director, Oncology, Corcept Therapeutics

Stephen has been in biopharma for over 25 years and has worked in neurology with Elan Pharmaceuticals, gene therapy for rare diseases with Audentes Therapeutics and Encoded Therapeutics, virology with Gilead Sciences, and oncology with Genentech, Medivation, Clovis Oncology, and 23andme, and, currently, Corcept Therapeutics.  I started my career in clinical operations where I led company-sponsored studies and oversaw investigator-initiated studies.  I made the move to project/program management in 2014 to work on all phases of drug development from discovery through post-marketing activities and have assumed business development and alliance management responsibilities as needed for my programs.  I earned an MBA through the Gilead Sciences MBA cohort program, an MA in East Asian Art from the Sotheby’s Institute of Art in London, and an MS in Project Management.  I am a team leader dedicated to bringing new drugs to patients. I believe in life long learning, appreciate the here and now, strive to treat everyone with compassion and dignity, and lead with integrity and drive to meet corporate goals.

Session Details:

Selecting a CRO for rare disease trials: aspects to contemplate

2024-09-10, 2:00 PM

Session Details:

PANEL DISCUSSION Discovering ways to overcome enrollment challenges in rare disease clinical trials

2024-09-10, 5:00 PM

View In Agenda
Next speaker
Back
Katrina Paz
Director, Clinical Programming, Denali Therapeutics

Katrina "Kat" Paz is a Director of Clinical Programming from Denali Therapeutics.  Serving as a programmer for over two decades, Katrina has worked at Genentech, Gilead Sciences and Nektar Therapeutics.  She has worked as a Data Manager and Statistical Programmer but found her niche in Clinical Programming, creating outputs for data cleaning and monitoring.  Kat's joys are leading technical teams with a focus in mentoring and finding efficiencies via tools and processes.

Session Details:

Possible integration of machine learning for data cleaning in rare disease clinical trials

2024-09-11, 11:45 AM

View In Agenda
Next speaker
Back
Nasser Al-Ali
Senior Clinical Programmer, Denali Therapeutics

Nasser Al-Ali is currently a senior Clinical Programmer at Denali Therapeutics and a PhD student in Statistics and Data Science at the University of Nevada, Reno. He is deeply interested in programming, particularly in automating processes to enhance efficiency and effectiveness. His focus extends to the utilization of machine learning in data cleaning, aiming to elevate data quality through innovative, real-time cleaning strategies.

Session Details:

Possible integration of machine learning for data cleaning in rare disease clinical trials

2024-09-11, 11:45 AM

View In Agenda
Next speaker
Back
Kenneth Harvey
Director Clinical Operations, Epic Bio

Head of Clinical Operations for Epic Bio, where he oversees all Clinical Operations activities in support of Epic's clinical trials. He has a background in patient care setting, as well as research-focused clinics on the ground level of clinical trials before moving into research management. For the last 20+ years he has led cross-functional teams carrying out global multicenter trials - in all phases of drug development - and multiple therapeutics areas including Neuro, Oncology and Immunology. He has experience with both large and small molecules therapeutics as well as gene therapies and now epigenetic therapies with Epic Bio.

For the last decade his focus has been on building capabilities for startup Biotechs to tackle rare and ultra-rare diseases and has launched first-in-human therapeutics for companies like Genentech, Audentes, Cytokinetics, Jasper Therapeutics and Kezar Life Sciences.

Ken has an International Executive MBA in Business and Finance from the International School of Management (ISM) in Paris, France.

Session Details:

PANEL DISCUSSION Discovering ways to overcome enrollment challenges in rare disease clinical trials

2024-09-10, 5:00 PM

Session Details:

PROBLEM-SOLVING ROUNDTABLE DISCUSSIONS

2024-09-11, 2:15 PM

View In Agenda
Next speaker
Back
Nadine Knowles
Vice President, Clinical Operations, Longboard Pharmaceuticals

Session Details:

PANEL DISCUSSION Discovering ways to overcome enrollment challenges in rare disease clinical trials

2024-09-10, 5:00 PM

View In Agenda
Next speaker
Back
Hayley Mason
Policy Analyst, NORD

Hayley Mason MPA, is a dedicated professional with a fervent commitment to promoting effective governance and public policy. Holding a Master of Public Administration (MPA) degree from American University in Washington, DC, and a Bachelor's degree in Global Affairs from George Mason University, Hayley brings a comprehensive understanding of governmental structures, policy analysis, and program evaluation to her advocacy work for individuals with rare diseases. Throughout her career, Hayley has focused on enhancing public service delivery and driving positive societal change.

Hayley's journey began as an intern for the United States Senate and then at the Ability Center of Virginia, a nonprofit organization dedicated to supporting children and adults with disabilities lead productive lives in Virginia Beach, Virginia.

Drawing from these diverse experiences, Hayley currently serves as a Policy Analyst with the National Organization for Rare Disorders. In this role, she collaborates with advocates, Congress, and government officials to champion increased treatments, access, and inclusion for individuals living with rare diseases. Driven by a profound desire to effect meaningful societal change, Hayley is committed to inspiring others to pursue excellence in public service and harnessing the power of effective governance for the betterment of all

 

Session Details:

PANEL DISCUSSION Navigating a changing landscape: the future of rare disease treatments

2024-09-10, 10:00 AM

Session Details:

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

2024-09-11, 9:00 AM

View In Agenda
Next speaker
Back
Vij Senthilnathan
Associate Director, Clinical Operations, PTC Therapeutics

Session Details:

PANEL DISCUSSION Navigating a changing landscape: the future of rare disease treatments

2024-09-10, 10:00 AM

View In Agenda
Next speaker
Back
Aditi Kantipuly
MD, MS, Physician, Visiting Scholar University of New Mexico

Session Details:

FIRESIDE CHAT Innovative funding models for treatment of rare diseases

2024-09-11, 11:15 AM

Session Details:

PROBLEM-SOLVING ROUNDTABLE DISCUSSIONS

2024-09-11, 2:15 PM

View In Agenda
Next speaker
Back
Revati Tatake
Global Head of Pharma Research, Analysis and Competitive Intelligence, GlobalData Plc

Revati Tatake, PhD, is the Global Head of Pharma Research, Analysis and Competitive Intelligence at GlobalData Healthcare.  Revati has over 25 years of multidisciplinary experience in academic research and the healthcare industry, both on the client and agency sides of the business. Previously she worked as a VP of Databases and Analytics at GlobalData, where she made significant contributions to Pharma Intelligence Center. Before joining GlobalData, Revati worked at Citeline/Informa, where she was involved in competitive intelligence and analytics of clinical trials and drugs in the areas of Autoimmune/Inflammation, Central Nervous System, and Ophthalmology.  Earlier, Revati worked at Boehringer Ingelheim Pharmaceuticals for over 12 years as Senior Scientist to Senior Principal Scientist, where she led drug discovery projects involving traditional high-throughput screening, as well as innovative approaches for gene and cell therapies. She also worked at Boehringer Ingelheim as a Director of Competitive Intelligence for projects in cardiometabolic space and research beyond borders. She is a co-inventor on many issued US patents related cell and gene therapies and has several publications in peer reviewed scientific journals. Revati holds a PhD in Tumor Immunology from the Tata Cancer Research Institute in Mumbai, India. She did her postdoctoral fellowship at the University of Connecticut Health Center and Boehringer Ingelheim.

Session Details:

Chairperson’s opening remarks

2024-09-10, 8:50 AM

Session Details:

PANEL DISCUSSION Navigating a changing landscape: the future of rare disease treatments

2024-09-10, 10:00 AM

Session Details:

Chairperson’s opening remarks

2024-09-11, 8:50 AM

Session Details:

FIRESIDE CHAT Innovative funding models for treatment of rare diseases

2024-09-11, 11:15 AM

Session Details:

PANEL DISCUSSION Discovering ways to overcome enrollment challenges in rare disease clinical trials

2024-09-10, 5:00 PM

Session Details:

FIRESIDE CHAT Navigating the IVDR landscape: impact on rare disease studies and diagnostics use in clinical trials

2024-09-11, 9:30 AM

View In Agenda
Next speaker
Back
Matthew Birmingham
Head Clinical Supply, Prime Medicine

Session Details:

PROBLEM-SOLVING ROUNDTABLE DISCUSSIONS

2024-09-11, 2:15 PM

View In Agenda
Next speaker
Back
Katia Stakhiv
Associate Director, Research and Development, Rare Diseases, Amgen

Session Details:

Engaging patients beyond just participating in a trial: the role of biopharma companies in supporting patients

2024-09-11, 12:45 PM

Session Details:

PROBLEM-SOLVING ROUNDTABLE DISCUSSIONS

2024-09-11, 2:15 PM

View In Agenda
Next speaker

Plan Your Visit

Venue

Hyatt Regency San Francisco Airport

1333 Old Bayshore Hwy Burlingame CA 94010 United States

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

Exhibitors

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

MEDIA PARTNERS

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

Ralitsa Pashkuleva

Conference Producer

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:

Conor Taylor

Sponsorship Sales Manager


+44 (0) 20 7936 6870

SPEAKER ENQUIRIES

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

Ralitsa Pashkuleva

Conference Producer