Next Gen and Personalized Medicine Seminar

NEXT GENERATION THERAPIES SEMINAR

8:00 Registration & Networking Breakfast

08:55

Chairperson Opening Remarks

John Leonard
09:20

Using CRISPR-Cas9 to develop drugs for rare liver disorders and other rare diseases

Sudhir Agrawal
09:40

Third-generation antisense targets to overcome the hurdles of current antisense technologies in rare disease research

Takashi K Kishimoto
10:00

Synthetic vaccine particles in the prevention of anti-Drug antibodies in rare disease treatments

10:20 Networking coffee break

Nikhil Nair
11:00

Synthetic biology approaches in the gut microbiome to treat inborn errors of metabolism (IEMs)

Bill Lundberg
11:20

Ex vivo programs using CRISPR/Cas9 gene editing to treat a wide variety of rare diseases

Bill Lundberg, Chief Scientific Officer, CRISPR Therapeutics
Ms Annemarie Moseley
11:40

A new paradigm in Hematopoietic stem cell transplantation (HSCT) for orphan inherited blood disorders

12:00 Networking Lunch

PERSONALIZED MEDICINE SEMINAR

13:00

Opening remarks

William Gahl
13:05

The NIH Undiagnosed Diseases Program and Network: a cross-disciplinary approach to disease diagnosis, new disease identification and the application of precision medicine to patient care

Adam Resnick
13:25

Building the rare disease ecosystem and enabling innovation and drug discovery through data collaboration

Murray Brilliant
13:45

Personalized to precision – Studying large cohorts of people under precision medicine to identify drug repurposing for rare diseases and gain insight into more common disorders

14:05

Data sharing and natural history studies to bridge the gap between heterogeneity of disease progression and trial design in Duchenne Muscular Dystrophy

2:25 Networking coffee break

Dale Bodian
15:00

Utility of whole-genome sequencing for detection of new-born screening disorders

Andrea Califano
15:20

The personalization of clinical trials – N of 1 trials in rare disease

Romina Ortiz
16:00

The iHope program: generating awareness of the needs of those with rare, undiagnosed genetic diseases by providing whole genome sequencing to patients and parents in financial need

David Litwack
16:20

The FDA’s role in moving towards a global precision medicine industry- Impact on Rare Diseases

16:40 Welcome drinks reception

17:40 End of pre-conference day

last published: 30/Mar/17 16:05 GMT