ADVANCED THERAPIES - CLINICAL

 

On the Advanced Therapies- Clinical track you will hear from companies, regulators and researchers currently developing call and gene therapies to treat rare diseases.

 

Key Speakers

 

Advanced Therapies - Clinical, Thursday 11 April 2019

07:00

7:00 Registration and Breakfast

John Maraganore
08:30

Keynote Address: Advancing RNAi therapeutics as a whole new class of medicines

Christopher Austin
08:55

Keynote Address: Re-thinking and reshaping “rare” – continuing the march towards curative therapies for the rare disease community

Arndt Rolfs
09:20

Keynote Address: Accelerating Orphan Drug development and improving outcomes, by unlocking genetic information

10:30

10:30 SPEED NETWORKING FOLLOWED BY MORNING NETWORKING BREAK

Charlie Albright
Advanced Therapies - Clinical
11:50

Further defining the path to the clinic for in vivo gene editing

Stephen Hyde
Advanced Therapies - Clinical
12:15

Getting to the clinic through strategic partnership – combining scientific, clinical development, and manufacturing skills to develop new treatments for Cystic Fibrosis patients in Europe

Maryna Kolochavina
Advanced Therapies - Clinical
12:40

Use of real world data to support the path to approval for advanced therapies in rare diseases

Michael Murphy
Advanced Therapies - Clinical
13:00

When predictable execution trumps brilliant planning: the interplay of strategic and transactional relationships in orphan disease drug development

13:05

1:05 NETWORKING LUNCH

2:30 ROUNDTABLES (1-hour roundtable sessions split between 2 rounds of 30min) – SPONSORED ROUNDTABLES AVAILABLE

Tiina Urv
14:30

ROUNDTABLE 10: Data ownership – addressing the need for increased data sharing while also considering company/patient data "ownership" through clinical trials and beyond

Tara Mathiesen
14:30

ROUNDTABLE 11: Educating HCPs – when considering the absence of newborn screening lessons learned from Metachromatic Leukodystrophy (MLD)

14:30

ROUNDTABLE 12: Chilling investment? – the potential implications of increased public scrutiny of rare disease drug prices in the US

Vipul Kashyap
14:30

ROUNDTABLE 13: Cross-Ecosystem collaborations – mutual value propositions and collaborations for pharma, payers and providers to collaborate in the rare diseases space

Harsha Rajasimha
14:30

ROUNDTABLE 14: Bridging US and India - strategies to accelerate rare diseases clinical research and patient recruitment

Paul Melmeyer
14:30

ROUNDTABLE 15: Federal Policy – update on rare disease federal policy and NORD initiatives to engage capital hill

Susan Robinson
14:30

ROUNDTABLE 18: Market Access & commercialization – how specialty services and options should vary for patient populations of 20, 2,000, and 20,000

Leone Atkinson
14:30

ROUNDTABLE 19: Update on patient centricity in research – preliminary survey results from patient advocacy group leaders

Anne Cropp
14:30

ROUNDTABLE 1: Global expanded access – applying artificial intelligence and access innovation for expanded access programs

Carol Houts
14:30

ROUNDTABLE 21: Inhouse versus outsource – determining the right orphan drug manufacturing fit for your organization

Scott Gray
David Lapidus
14:30

ROUNDTABLE 3: Epidemiology & forecasting – value, challenges, and methods for rare diseases

Joseph Musumeci
14:30

ROUNDTABLE 4: BD & forecasting – how to build forecasts based on available epidemiological information and considerations for pricing and access

Scott Schliebner
14:30

ROUNDTABLE 5: Patient-focused research – new approaches to bring clinical trials directly to patients and change the patient-centric trial paradigm in rare diseases

Jamie Arnott
14:30

ROUNDTABLE 6: Patient-centered Development: Early patient input, understanding patient goals in clinical trials, and furthering relationships between all stakeholders

Lisa Dilworth
14:30

ROUNDTABLE 7: Meaningful endpoints – navigating the differences in what it means to patients, caregivers, physicians and regulators during advanced therapies clinical development

Kate Grady
14:30

ROUNDTABLE 8: Driving diagnosis – steps industry can take to improve the diagnosis of rare disease

14:30

ROUNDTABLE 9: Developing and Commercializing Orphan Drugs: Outsourcing and CMC Strategies

ROUND 2 (select a second roundtable to attend from the options above)

Sal Rico
Advanced Therapies - Clinical
15:40

Advancing the clinical program development of AAV-Based Gene therapies for rare diseases: a case study

Sal Rico, Vice President, Clinical Development, Audentes Therapeutics
Barrett Katz
Advanced Therapies - Clinical
16:00

Advancing the clinical program development of gene therapy for ultra-orphan disease: finding the patients, getting them enrolled

Ilan Irony
Advanced Therapies - Clinical
16:20

FDA gene therapy draft guidance documents – disease specific guidances and long-term follow up

Advanced Therapies - Clinical
16:40

Unique challenges for expanded access in gene therapies – considerations for companies, patients

17:00

5:00 AFTERNOON NETWORKING BREAK

18:30

6:30 NETWORKING DRINKS RECEPTION – Sponsored by Orchard Therapeutics

last published: 20/Mar/19 21:45

Advanced Therapies - Clinical, Friday 12 April 2019

07:30

7:30 Registration and Breakfast

Jeff Ajer
08:50

Keynote Address: Preparing for a new treatment paradigm - gene therapy access and affordability

Stuart Bell
09:10

Keynote address: A staged approach to European patient access

10:05

Keynote address – PLATINUM SPONSOR

10:15

10:15 MORNING NETWORKING BREAK

Brian Kaspar
Advanced Therapies - Clinical
11:15

Translation of Gene Therapeutics in Neurological and Neuromuscular Diseases

Advanced Therapies - Clinical
11:15

Translation of Gene Therapeutics in Neurological and Neuromuscular Diseases

Marco Ruella
Advanced Therapies - Clinical
11:40

Combatting resistance in CAR-T clinical trials – developing new approaches to treat patients

Advanced Therapies - Clinical
12:05

Delivering cancer treatments faster through allogeneic CAR-T therapies – clinical considerations and the future of allogeneic therapies

Mark Pykett
Advanced Therapies - Clinical
12:05

Development of gene therapies for rare CNS disorders

Advanced Therapies - Clinical
12:30

Gold Sponsor Slot

12:50

12:50 NETWORKING LUNCH

14:30

2:30 ROUNDTABLES (1-hour roundtable sessions split between 2 rounds of 30min) – SPONSORED ROUNDTABLES AVAILABLE

Tim Boyd

ROUNDTABLE 10: State Policy – continued efforts to advance rare disease policy and initiatives

Theodore Roth

ROUNDTABLE 11: Alternative to viral vectors – reprogramming human T cell function and specificity with non-viral genome targeting

Melissa Hogan

ROUNDTABLE 12: Gene therapy consortium – elevating patient groups’ collective knowledge of gene therapy and working with companies though the first ever patient group gene therapy consortium

Karen Anderson

ROUNDTABLE 13: Disease burden – characterizing the burden of disease to more effectively educate regulators, physicians and payers

Roser Francisco Bordas

ROUNDTABLE 14: Rare in Catalonia – Regional policies for an integrated health care in rare diseases

Samir Shaikh
Jeremy Cress

ROUNDTABLE 16: Advanced manufacturing – building a cGMP in-house cell & gene therapy facility

Christina Poschen

ROUNDTABLE 17: Gene therapy commercial readiness - how to get yourself commercially ready for gene therapy. Practicalities of access agreements, concierge services and technology stewardship

Christina Poschen, Associate Consultant, Partners4Access
Jay Weaver
14:30

ROUNDTABLE 18: Sustainable pricing – will value-based payment models be sustainable for the healthcare system?

Scott Schliebner

ROUNDTABLE 1: Gene therapy development – challenges, Issues, and solutions from the new frontier of rare disease therapies

Carolyn Chapman
14:30

ROUNDTABLE 20: Compassionate Use--- Ethical considerations for pre-approval access to gene therapy products

Zizi Imatorbhebhe
14:30

ROUNDTABLE 21: Moving from n=1 to marketing application – clinical and regulatory considerations for accelerating rare disease product development

Doug Pfaff

ROUNDTABLE 2: Rare Disease Forecasting - Key drivers of uncertainty and how to deal with imperfect data

Han Phan

ROUNDTABLE 3: Site selection – how can dedicated rare disease trial sites enable studies to be conducted on a timely and effective manner

ROUNDTABLE 5: Excessive pricing - a deep dive into US and European approaches, recent cases and possible implications for industry and pricing strategies

Trista Morrison

ROUNDTABLE 6: Patient education - The importance of educating the patient community about rare diseases and newly approved and advanced therapies

Christopher Chute

ROUNDTABLE 8: Analyzing ICD-11 – challenges and complications around coding for rare diseases in the healthcare setting and understanding if the new ICD-11 will aid rare diseases coding

16:15

End of Conference

last published: 20/Mar/19 21:45