ADVANCED THERAPIES - MANUFACTURING

 

Advanced Therapies - Manufacturing will feature the FDA and leading advanced therapies manufacturers will be discussing successful approaches to key manufacturing challenges and how industry and regulators can work together to advance cell and gene therapy development smoothly.

 

Key Speakers

 

Advanced Therapies - Manufacturing, Wednesday 10 April 2019

last published: 13/Feb/19 21:55

Advanced Therapies - Manufacturing, Thursday 11 April 2019

Ellen Coleman
08:25

Chairperson's opening remarks

Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
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

Derek Adams
Advanced Therapies - Manufacturing
11:25

One batch, one patient – can innovation in autologous therapies process development be radically accelerated to serve larger populations?

Stewart Craig
Advanced Therapies - Manufacturing
11:50

In-housing vs outsourcing manufacturing and process development – assessing benefits and risk

David Sourdive
Advanced Therapies - Manufacturing
12:15

How Cellectis evolves its gene-editing enabled off-the-shelf CAR-T manufacturing to meet cell therapy commercial standards

Advanced Therapies - Manufacturing
12:40

Gold Sponsor Slot

13:05

1:05 NETWORKING LUNCH

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

Tiina Urv

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

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

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

Vipul Kashyap

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

Harsha Rajasimha

ROUNDTABLE 14: Rare in India – engaging patients with rare diseases in India with global clinical trials and resources

Paul Melmeyer

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

Clive Whitcher

ROUNDTABLE 17: Patient Access in Europe & Beyond - Enabling access for rare disease therapies exploring alternative pathways for patients in need

Clive Whitcher, Vice President Head of Global Patient Access, Inceptua Commercial Products
Kelly Fearn, Principal Consultant, Inceptua Medicines Access
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

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

David Lapidus

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

Joseph Musumeci

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

Scott Schliebner

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

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

Lisa Dilworth

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

Kate Grady

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

14:30

ROUNDTABLE 9: Sponsored by Catalent Pharma Solutions

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

Alfred Boyle
Advanced Therapies - Manufacturing
15:40

Development of commercial scale manufacturing of GalNAc conjugated siRNAs. analytical and process perspective

Mark Galbraith
Advanced Therapies - Manufacturing
16:00

Challenges in analytical development for gene therapy for rare diseases – Luxturna as a case study

Advanced Therapies - Manufacturing
16:00

Using analytical development to mitigate impurity and determine potency of cell products

Herbert Runnels
Advanced Therapies - Manufacturing
16:20

Gene Therapy and AAV: Analytical Advancements Enabling Successful Product Development and Life-Cycle Management

17:00

5:00 AFTERNOON NETWORKING BREAK

18:30

6:30 NETWORKING DRINKS RECEPTION – Sponsored by Orchard Therapeutics

last published: 13/Feb/19 21:55