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
08:00 - 08:50
REGISTRATION AND MORNING REFRESHMENTS
08:50 - 09:00
OPENING ADDRESS
09:00 - 09:25
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
09:25 - 09:30
Q&A SESSION ON PATIENT-DRIVEN RARE DISEASE THERAPEUTICS
09:30 - 10:00
RESERVED PRESENTATION
10:00 - 10:30
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!
10:30 - 11:00
MORNING COFFEE BREAK IN THE EXHIBIT AREA
11:00 - 11:30
PANEL DISCUSSION ON PATIENT ADVOCACY AS INFRASTRUCTURE FOR RARE DISEASE DEVELOPMENT
  • Positioning advocacy as a strategic function shaping enrollment, access, trust, and performance across rare disease development
  • Demonstrating how close patient partnerships improve trial design, recruitment, communications, and postapproval planning
  • Embedding patient organizations in policy, awareness, education, and care priorities to build lasting rare disease support systems
  • Advancing practical support that strengthens standards of care, quality of life, and reimbursement navigation for rare disease families
  • Defining meaningful endpoints, improving informed consent, and preparing reimbursement pathways so approved therapies reach patients

| ARCHO

| Agios Pharmaceuticals

| Medasol

| Aclipse Therapeutics

| Independent

11:30 - 11:55
UMBRELLA STUDY DESIGNS IN CLINICAL TRIALS: A CASE STUDY IN FSGS
Arjun Channi
Takeda

Arjun Channi

Takeda

  • 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
SHOWING UP BEFORE ROI: TRUST IN THE RARE DISEASE ECOSYSTEM
Melody Joy Paine
N=1 Collaborative

Melody Joy Paine

N=1 Collaborative

  • Showing up early to build trust in ultra-rare communities before portfolio fit or ROI
  • Standing alongside patient ecosystems with transparent support, listening, and accountability
  • Partnering with N=1 Collaborative to advance individualized medicine before it is fully scalable
13:55 - 14:00
Q&A SESSION ON EARNING TRUST BEFORE RARE DISEASE ROI
14:00 - 14:30
RESERVED PRESENTATION
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 - 15:20
AFTERNOON COFFEE BREAK IN THE EXHIBIT AREA
15:20 - 15:50
PANEL DISCUSSION ON BUILDING APPROVABLE EVIDENCE WITH FEW PATIENTS: TRIAL INNOVATION, RWE, AND PATIENT-FOCUSED ENDPOINTS
15:50 - 16:15
THERAPEUTICS FOR RARE DISEASES AND CONDITIONS ASSOCIATED WITH MALABSORPTION
Andreas M Papas
Antares Health Products

Andreas M Papas

Antares Health Products

  • Clarifying how biliary, pancreatic, and gut dysfunction drives malabsorption in children
  • Mitigating fat-soluble vitamin loss, including vitamin E deficiency tied to neurologic decline
  • Adapting liquid, powder, chewable, and softgel formats for infants, children, and older adults
16:15 - 16:20
Q&A SESSION ON THERAPEUTICS FOR MALABSORPTION IN RARE DISEASES
16:20 - 16:45
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
16:45 - 16:50
Q&A SESSION ON PRECLINICAL ASO AND SIRNA TOXICOPATHOLOGY
16:50 - 17:15
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
17:15 - 17:20
Q&A SESSION ON MOVING BREAKTHROUGHS INTO RARE THERAPIES
17:20 - 18:20
NETWORKING DRINKS RECEPTION
Day 2 :
TUESDAY, JULY 14, 2026
08:00 - 08:30
MORNING REFRESHMENTS
08:30 - 08:40
OPENING ADDRESS
08:40 - 09:10
PANEL DISCUSSION ON FROM DESIGNATION TO APPROVAL: REGULATORY STRATEGY FOR ORPHAN DRUG
09:10 - 09:35
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
09:35 - 09:40
Q&A SESSION ON EMPLOYER PLAN COVERAGE IN RARE DISEASE
09:40 - 10:05
TAMING COMPLEXITY IN CELL AND GENE THERAPIES FOR RARE AND ORPHAN DISEASES
Vishal Singal
Beghou

Vishal Singal

Beghou

  • Mapping CGT market trends and 2026 FDA milestones shaping rare and orphan pipelines
  • Stabilizing the shift from lab builds to scalable operations with simpler, defined models
  • Sustaining access by aligning US regulatory pathways with patient success stories and outcomes
10:05 - 10:10
Q&A SESSION ON TAMING CGT COMPLEXITY FOR RARE DISEASES
10:10 - 10:30
MORNING COFFEE BREAK IN THE EXHIBIT AREA
10:30 - 11:00
PANEL DISCUSSION ON THE BURNING QUESTION: WHO PAYS FOR ONE-AND-DONE THERAPIES
11:00 - 11:25
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
11:25 - 11:30
Q&A SESSION ON ORPHAN DRUG COST RECOVERY AND MEDICARE POLICY
11:30 - 11:55
LYOPHILIZED ODT PLATFORM FOR WATER-FREE ORAL AND SUBLINGUAL DELIVERY
Keith Dodson
InstaPill

Keith Dodson

InstaPill

  • Introducing InstaPill lyophilized ODTs that disintegrate in saliva for water-free dosing
  • Supporting pediatric and geriatric patients when swallowing conventional tablets is difficult
  • Addressing orphan drug delivery needs by simplifying administration for dispersed populations
  • Stabilizing protein and peptide orphan drugs with a lyophilized ODT platform for oral dosing
11:55 - 12:00
Q&A SESSION ON ORPHAN DRUG DELIVERY WITH LYOPHILIZED ODTS
12:00 - 12:25
10 BEST PRACTICES IN RARE DISEASE PRODUCT LAUNCHES
Jack Davis
Rare Expertise

Jack Davis

Rare Expertise

  • Anticipating where pharma playbooks fail and resetting launch logic for rare diseases
  • Calibrating patient finding, KOL timing, and support programs to reach treated patients
  • Institutionalizing payer readiness, expectation setting, and post-launch persistence
12:25 - 12:30
Q&A SESSION ON BEST PRACTICES FOR RARE DISEASE LAUNCHES
12:30 - 13:30
NETWORKING LUNCH & VISIT THE ORPHAN DRUG EXHIBITION
13:30 - 14:00
RESERVED PRESENTATION
14:00 - 14:25
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
14:25 - 14:30
Q&A SESSION ON CENTERING PATIENTS IN ORPHAN DRUG DEVELOPMENT​
14:30 - 14:55
BREAKING RESISTANCE: ADVANCING ORPHAN THERAPIES FOR IMMUNE CHECKPOINT–REFRACTORY PANCREATIC CANCER
Jose Jimeno
Novita Pharmaceuticals

Jose Jimeno

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
14:55 - 15:00
Q&A SESSION ON ORPHAN DRUG DESIGNATION IN ONCOLOGY
15:00 - 15:15
FEEDBACK AND RAFFLE DRAW
15:15 - 15:30
CLOSING REMARKS

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