Highlights of the Program

2 days business program:

Learn from real-world case studies by industry leaders.

SHOWCASING INNOVATION:

Discover the latest technology and techniques from across the industry.

leaders talk:

Hear from top-level experts about how to stay ahead in a fast-changing industry.

MULTIPLE STREAMS:

A business program that is multi-disciplinary, giving you a broad view of the industry.

SMART TECHNOLOGIES:

Explore the latest smart and AI-driven solutions, and see how they can be used in your business.

roundtable discussion:

Join talks with industry peers. Share ideas, make connections, and find new partners.

Program

Day 1 :
MONDAY, JULY 13, 2026
08:30 - 09:30
REGISTRATION AND MORNING REFRESHMENTS
09:30 - 09:40
OPENING ADDRESS
09:40 - 10:05
A RADICALLY NEW ERA OF PATIENT-DRIVEN THERAPEUTICS
Ben Lenail
American Brain Foundation

Ben Lenail

American Brain Foundation

  • Mobilizing technology and patient networks to deepen disease understanding across the real-life burden
  • Characterizing natural history, biomarkers, and endpoints to reflect outcomes that matter to families
  • Guiding industry partnership on trial design, dosing, delivery modalities, and access considerations
  • Co-developing hybrid discovery models with academia and industry, drawing on X-ALD initiatives
10:05 - 10:10
Q&A SESSION ON PATIENT-DRIVEN RARE DISEASE THERAPEUTICS
10:10 - 10:40
SPEED NETWORKING SESSION
  • Exchange business cards and get connected in short one-to-one meetings
  • Start the conversation to arrange a more formal meeting later on in the conference
  • Share your professional background and discuss your biggest business issues – don't forget your business cards!
10:40 - 11:00
MORNING COFFEE BREAK IN THE EXHIBIT AREA
11:00 - 11:30
PANEL DISCUSSION ON ORPHAN DRUG
11:30 - 11:55
UMBRELLA STUDY DESIGNS IN CLINICAL TRIALS: A CASE STUDY IN FSGS
Arjun Channi
Sanofi

Arjun Channi

Sanofi

  • Designing umbrella trials in FSGS to evaluate multiple candidates within one shared protocol
  • Accelerating readouts by cutting costs and time while identifying winning development paths earlier
  • Negotiating study parameters with health authorities, clinicians, and patients to manage complexity
11:55 - 12:00
Q&A SESSION ON UMBRELLA DESIGNS IN FSGS CLINICAL TRIALS
12:00 - 12:25
PHAROS 2.0: SCALING TARGET DATA FOR RARE DISEASE DISCOVERY
Jessica Maine
National Institute of Health

Jessica Maine

National Institute of Health

  • Modernizing Pharos with curated targets, diseases, drugs, pathways, and protein isoforms
  • Harmonizing datasets via modular pipelines for entity resolution, provenance tracking, and quality
  • Enabling graph queries and ML workflows to recalculate Target Development Levels from evidence
12:25 - 12:30
Q&A SESSION ON PHAROS 2.0 FOR TARGETS AND REPURPOSING
12:30 - 13:30
NETWORKING LUNCH AND VISITING THE ORPHAN DRUG EXHIBITION
13:30 - 13:55
OPPORTUNITIES FOR RARE DISEASE PRODUCT COMMERCIALIZATION
Mohamed H. Ladha
Recordati Rare Diseases

Mohamed H. Ladha

Recordati Rare Diseases

  • Defining what success looks like for commercialization in rare disease product launches
  • Aligning resources across access, medical, and commercial teams to support launch readiness
  • Prioritizing where to invest time and budget given limited in-house resources and capabilities
13:55 - 14:00
Q&A SESSION ON RARE DISEASE PRODUCT COMMERCIALIZATION
14:00 - 14:25
CLINICAL DEVELOPMENT PATHWAYS FOR THE APPROVAL OF SUBCUTANEOUSLY DELIVERED BIOLOGICS, INCLUDING FOR ORPHAN DISEASES
Charles Theuer
Halozyme

Charles Theuer

Halozyme

  • Converting IV-approved antibodies to subcutaneous delivery while preserving safety evidence
  • Standardizing pathways with consistent trial designs, endpoints, and bridging requirements
  • Leveraging authority flexibility on endpoints and extrapolation to speed SC approvals
14:25 - 14:30
Q&A SESSION ON SUBCUTANEOUS BIOLOGICS FOR ORPHAN CARE
14:30 - 14:55
A PARTNERSHIP MODEL TO ACCELERATE RARE DISEASE INNOVATION
Samuel Chuang
Charles River Laboratories 

Samuel Chuang

Charles River Laboratories 

  • Structuring foundation–industry partnerships to guide grants through IND-enabling studies
  • Advising patient-led programs with translational SMEs to de-risk early scientific decisions
  • Optimizing preclinical strategies with creative study design and tailored cost-saving measures
14:55 - 15:00
Q&A SESSION ON PARTNERSHIP MODELS FOR RARE DISEASE INDS
15:00 - 15:30
AFTERNOON COFFEE BREAK IN THE EXHIBIT AREA
15:30 - 15:55
FROM LIVED EXPERIENCE TO SYSTEM IMPACT: THE PATIENT ADVOCATE PERSPECTIVE ON ORPHAN DRUG ACCESS
Felice Bombaci
Associazione Italiana contro le Leucemie - linfomi e mieloma

Felice Bombaci

Associazione Italiana contro le Leucemie - linfomi e mieloma

  • Amplifying advocate insights on daily barriers to starting, using, and sustaining orphan therapies
  • Capturing patient-generated evidence to complement clinical and regulatory data across pathways
  • Centering health equity by identifying access disparities and enabling more inclusive strategies
15:55 - 16:00
Q&A SESSION ON PATIENT ADVOCACY FOR ORPHAN ACCESS
16:00 - 16:25
PRECLINICAL TOXICOPATHOLOGY FINDINGS ASSOCIATED WITH SYSTEMICALLY ADMINISTERED ANTI-SENSE OLIGONUCLEOTIDES (ASOS) AND SMALL INTERFERING RNAS (SIRNAS)
Kuldeep Singh
Wave Life Sciences

Kuldeep Singh

Wave Life Sciences

  • Evaluating FDA’s 2024 draft guidance to align preclinical safety packages for oligos
  • Detailing in vitro screens and NOAEL studies in rodent and nonrodent species to set dosing
  • Interpreting systemic liabilities like immune activation, complement effects, and thrombocytopenia
  • Benchmarking histopathology across species to judge human relevance and improve predictive value
16:25 - 16:30
Q&A SESSION ON PRECLINICAL ASO AND SIRNA TOXICOPATHOLOGY
16:30 - 16:55
THE FUTURE OF INNOVATION FROM BREAKTHROUGHS TO PATIENTS
Carolina Alarco
Bio Strategy Advisors, LLC

Carolina Alarco

Bio Strategy Advisors, LLC

  • Streamlining biotech startup formation and strategy to move breakthroughs toward patients faster
  • Positioning pipelines for capital efficiency and investor readiness in next-gen orphan tech
  • Catalyzing partnerships and early investment to scale therapies for ultra-rare disorders
16:55 - 17:00
Q&A SESSION ON MOVING BREAKTHROUGHS INTO RARE THERAPIES
17:00 - 18:00
NETWORKING DRINKS RECEPTION
Day 2 :
TUESDAY, JULY 14, 2026
09:00 - 09:30
MORNING REFRESHMENTS
09:30 - 09:40
OPENING ADDRESS
09:40 - 10:05
BEYOND THE MOLECULE: PATENT AND REGULATORY STRATEGIES FOR RARE DISEASE BIOLOGICS AND GENE THERAPIES
Alicia Palladino
Goodwin Procter LLP

Alicia Palladino

Goodwin Procter LLP

  • Fortifying exclusivity for rare biologics and gene therapies through IP and regulatory levers
  • Drafting patent claims beyond molecule or vector to protect platforms, use, and manufacturing
  • Synchronizing orphan and biologic exclusivities with patent terms to manage cliffs in small markets
10:05 - 10:10
Q&A SESSION ON EMPLOYER PLAN COVERAGE IN RARE DISEASE
10:10 - 10:35
EMPLOYER TRENDS IN COVERAGE IN THE RARE DISEASE SPACE, CONSIDERATIONS, OPPORTUNITIES, AND PITFALLS
Ann Lewandowski
Healthcare Rebel Alliance

Ann Lewandowski

Healthcare Rebel Alliance

  • Tracking employer plan coverage shifts for orphan drugs and rare diseases since 2025
  • Weighing the ethical tension of funding one $1M therapy versus covering care for many members
  • Identifying market access levers that improve affordability and sustain equitable employer coverage
10:35 - 10:40
Q&A SESSION ON EMPLOYER PLAN COVERAGE IN RARE DISEASE
10:40 - 11:05
THE ECONOMICS OF ORPHAN DRUG DEVELOPMENT
Anna Kaltenboeck
Verdant Research

Anna Kaltenboeck

Verdant Research

  • Scrutinizing Medicare negotiation changes that exempt orphan drugs despite blockbuster revenues
  • Debating whether longer market time is needed for orphan drugs to recoup clinical investments
  • Estimating time to clinical trial cost recovery across different categories of orphan therapies
11:05 - 11:10
Q&A SESSION ON ORPHAN DRUG COST RECOVERY AND MEDICARE POLICY
11:10 - 11:30
MORNING COFFEE BREAK IN THE EXHIBIT AREA
11:30 - 11:55
THE FUTURE OF RARE DISEASE INCENTIVES: ARE PRIORITY REVIEW VOUCHERS STILL DRIVING INNOVATION?
Nana Mainoo
Cleracs Consulting

Nana Mainoo

Cleracs Consulting

  • Reassessing if priority review vouchers (PRV) still shift rare-disease capital as prices decline
  • Disentangling patient-need incentives from investor returns in voucher-driven orphan strategies
  • Proposing reforms such as PRV modernization and global alignment to sustain ultra-rare innovation
11:55 - 12:00
Q&A SESSION ON THE FUTURE OF PRIORITY REVIEW VOUCHERS
12:00 - 12:25
FROM LIVED EXPERIENCE TO LASTING CHANGE: CENTERING PATIENTS AND FAMILIES IN RARE DISEASE DRUG DEVELOPMENT
Amanda Moore
Angelman Syndrome Foundation

Amanda Moore

Angelman Syndrome Foundation

  • Embedding patient and caregiver insights across development from trials to endpoints and access
  • Leveraging real-world and community evidence to surface unmet needs and reduce access inequities
  • Translating Angelman advocacy lessons into trust-building partnerships and patient-centered systems
12:25 - 12:30
Q&A SESSION ON CENTERING PATIENTS IN ORPHAN DRUG DEVELOPMENT​
12:30 - 12:55
BREAKING RESISTANCE: ADVANCING ORPHAN THERAPIES FOR IMMUNE CHECKPOINT–REFRACTORY PANCREATIC CANCER
José Jiménez
Novita Pharmaceuticals

José Jiménez

Novita Pharmaceuticals

  • Overcoming immune resistance in checkpoint-refractory pancreatic cancer to enable new options
  • Utilizing FDA orphan designation to accelerate development and broaden access to innovation
  • Extending lessons from pancreatic cancer to guide orphan oncology combinations and paradigms
12:55 - 13:00
Q&A SESSION ON ORPHAN DRUG DESIGNATION IN ONCOLOGY
13:00 - 13:15
FEEDBACK AND RAFFLE DRAW
13:15 - 14:30
NETWORKING LUNCH

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