EAST COAST RARE DISEASES CLINICAL STRATEGIES XCHANGE
Boston
December 6, 2023

Welcome to hubXchange’s Rare Diseases Clinical Strategies East Coast 2023, bringing together executives from pharma and biotech to address and find solutions to the key issues faced in the cardiovascular drug discovery space, through a series of roundtable discussions.

Discussion topics will cover Clinical Biomarkers, Preclinical Studies, Clinical Development, Clinical Operations

Take advantage of this unique highly interactive meeting format designed for maximum engagement and collaboration with your peers.

Please note this is an In-Person meeting.

VENUE DETAILS: Hilton Boston Woburn Hotel, 2 Forbes Road, Woburn MA 01801

SNAPSHOTS OF DISCUSSION TOPICS

Track 1: Clinical Biomarkers

Identifying and validating novel biomarkers and end points for rare diseases
Developing biomarkers that accurately reflect disease status or treatment response while minimizing false positives and false negatives

Track 2: Preclinical Studies

Innovation and development of new gene transfer technologies:
Managing potential immunogenicity risks and leveraging both preclinical and clinical data for best solutions
Overcoming the absence of animal models

Track 3: Clinical Development

Challenges in designing and conducting clinical trials in rare diseases
What is the best practice to ensure your rare disease trial is a success?

Track 4: Clinical Operations

Understand the patient barriers within rare disease
Innovative data collection methods in rare disease trials: remote monitoring, wearables, and digital health

Full Xchange Agenda

Click on each track for detailed agenda

Clinical Biomarkers

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation

09:05 – 10:05

Identifying and validating novel biomarkers and end points for rare diseases

Sarah Bowden, Vice President, Clinical, QurAlis

10:10 – 10:40

1-2-1 Meetings/Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

11:20 – 12:20

Sponsor-led Roundtable

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:00 – 15:30

Poster Session

15:35 – 16:35

Defining biomarkers early in development to accelerate the path to clinical treatments for rare genetic disorders

Importance of clinical biomarkers in rare disease drug development
Innovative methods and techniques to define biomarkers
Power of patient organizations and advocacy groups in biomarker development

Smitha Jagadish

Vice President, Head of Rare Diseases and New Modalities, BIAL

Smitha is the Vice-President, Head of New Modalities and Rare Diseases at Bial. Innovative treatments are essential to address the high unmet medical needs in Rare Diseases. Prior to joining Bial, Smitha created the strategy and led the new modalities group for developing novel medicines for pain at Grunenthal. Prior to that, she was in the Genomic Medicine Unit at Sanofi Genzyme. Smitha received her Ph.D. in Neuroscience from Columbia University, NY, did her postdoctoral fellowship in University of California in San Francisco, CA, and was a Research Scientist at the Whitehead and Picower Institutes, MIT.

16:35 – 17:35

Evening Drinks Reception

Preclinical Studies

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation

09:05 – 10:05

Innovation and development of new gene transfer technologies: Managing potential immunogenicity risks and leveraging both preclinical and clinical data for best solutions

Translatability of preclinical models to the clinic
Need to ID novel biomarkers as potential surrogates for clinical efficacy
Need to ID more effective therapeutic strategies to better address developmental pathophysiology
Need to optimize gene therapy strategy approaches and ability to re-dose and/or enhance durability as needed

Joe Sypek, Strategy & External Alliances Lead, Rare Diseases DDU, Research & Development, Takeda

Joe has over 30 years of experience in discovery R&D on small molecules, biologics and gene therapy while at Tufts University, Genetics Institute, Wyeth, Pfizer, Shire and Takeda. He has extensive expertise in in vivo pharmacology, cytokine (IL-12/IL-13) and protease biology, immunology, and drug development in inflammation, autoimmunity, respiratory, and rare diseases.
He completed his doctorate at William & Mary and post-doctorate in immunology at Tufts University. He has co-authored many peer-reviewed papers, IND supporting documents, and several patents. He is a member of the American Association of Immunology, American Thoracic Society, and American Society of Gene & Cell Therapy.

10:10 – 10:40

1-2-1 Meetings/Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

11:20 – 12:20

Sponsor-led Roundtable

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:00 – 15:30

Poster Session

15:35 – 16:35

Overcoming the absence of animal models

Why is establishing robust disease models crucial for orphan drug research?
How can academia, industry, and patient groups collaborate more effectively to develop and validate new disease models?
Where can we identify and secure funding and resources specifically for developing disease models for rare diseases?
Are there any case studies demonstrating the significant impact of a robust disease model on rare disease drug research?
How can emerging technologies like organ-on-a-chip or computational modelling play a role in developing or supplementing disease models for rare diseases?

Scott Freeman, Chief Executive Officer, FF Biotherapeutics

Scott is the CEO of FF Biotherapeutics, with over 15 years in regulatory affairs and drug development. He is dedicated to revolutionizing orphan drug therapies. Holding a PhD in Cancer Biology, he has published extensively and made significant contributions to FDA regulatory practices, even reviewing over 100 orphan drug designations. His leadership roles, like overseeing FDA OOPD neuro-oncology grant reviews, emphasize his profound influence on orphan drug development. His unique blend of regulatory insight and scientific acumen ensures comprehensive drug development, prioritizing patient outcomes. Outside work, he’s a seasoned musician and cherishes moments with his two sons and also enjoys swimming and chess.

16:35 – 17:35

Evening Drinks Reception

Clinical Development

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation

09:05 – 10:05

Challenges in designing and conducting clinical trials in rare diseases

How industries could collaborate to generate and validate approvable intermediated endpoints to accelerate patients’ access to drugs
How to reach under-represented populations – where are we with decentralized clinical trials?
How to take into account the rapid changes in standard of care when designing clinical trials
How take into account heterogeneity (eg genetic, underlying SoC, disease severity): the broader the better?

Cristina Quarta

Senior Medical Director, MeTA+ Clinical Development & Translational Sciences, Alexion

Cristina Quarta, Senior Medical Director at Alexion, AstraZeneca Rare Disease, Boston, holds a Ph.D. in medical sciences from the University of Bologna, specializing in Cardiology.

At Alexion, she has been leading the global amyloidosis programs, including CAEL-101 and ALXN2220 monoclonal antibody therapies, as well as the ALXN2060 TTR stabilizer for Japanese patients with ATTR cardiomyopathy.

Before joining industry Cristina was a Senior Researcher and consultant cardiologist at National Amyloidosis Centre & Royal Free Hospital in London. Formerly, as a Research Fellow at Harvard Medical School, she managed echocardiographic projects and collaborated with the Brigham and Boston University’s Amyloid Centers. Cristina authored 80+ papers and is active in cardiology and amyloidosis professional societies.

10:10 – 10:40

1-2-1 Meetings/Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

11:20 – 12:20

Sponsor-led Roundtable

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:00 – 15:30

Poster Session

15:35 – 16:35

What is the best practice to ensure your rare disease trial is a success?

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

Daniel Klamer, VP of BD and Scientific Strategy, Anavex

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, Daniel led and evaluated multiple discovery-phase neuropharmacological research products with an emphasis on strategic evaluation of preclinical and clinical development.

Daniel 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, he holds a position as an Associate Professor at the Department of Pharmacology at The Sahlgrenska Academy at the University of Gothenburg.

16:35 – 17:35

Evening Drinks Reception

Clinical Operations

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation

09:05 – 10:05

Maximizing patient identification for rare disease and translational strategies

Christopher Winrow, Chief Scientific Officer, Lucy Therapeutics

10:10 – 10:40

1-2-1 Meetings/Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

11:20 – 12:20

Sponsor-led Roundtable

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:00 – 15:30

Poster Session

15:35 – 16:35

Understand the patient barriers within rare disease

Not enough information for patients on rare diseases.
Patients go undiagnosed for a long time.
Low interest from most pharmas for investing in rare diseases given the low population

Ricardo Mondragon Gonzalez, Senior Scientist Muscle Biology and Rare diseases, Sanofi

PhD in Genetics and Molecular Biology specialized in the study of the molecular and cellular mechanisms driving muscle function and disease. Experience working in muscle and neuromuscular-focused teams in companies such as: Novartis, Biogen and Sanofi. Currently member of the Sanofi Rare Disease ambassador program. He has also served to rare diseases patients in latin america by providing information and guidance on muscle diseases.

16:35 – 17:35

Evening Drinks Reception

EAST COAST RARE DISEASES CLINICAL STRATEGIES XCHANGE
Boston
December 6, 2023