Speakers

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Barry Byrne
Professor & Associate Chair of Department of Paediatrics
University of Florida

Day One

Thursday 13th December 2018

3:20 pm | Live Presenter Q&A

2:40 pm | AAV Systemic Therapy: Immunological Considerations and Redosing

Charles Vite
Professor
University of Pennsylvania

Day One

Thursday 13th December 2018

3:20 pm | Live Presenter Q&A

2:20 pm | Maximizing regional and global CNS transduction in large brains

Eric Kmiec
Director
Gene Editing Institute

Day One

Thursday 13th December 2018

5:20 pm | Live Presenter Q&A

4:40 pm | Development of an equitable approach to CRISPR-directed gene editing for genetic diseases.

1:20 pm | Panel Discussion: Discuss the Safety of Different Viral & Non-Viral Vectors

Gabriel Brooks
Vice President of Translational & Clinical Research
4D Molecular Therapeutics

Day One

Thursday 13th December 2018

3:20 pm | Live Presenter Q&A

3:00 pm | Leveraging Industrialized Directed Evolution for Tissue Targeted AAV Capsids in Fabry Disease

1:20 pm | Panel Discussion: Discuss the Safety of Different Viral & Non-Viral Vectors

Gerard Vockley
Chief of Medical Genetics & Director of the Centre for Rare Disease Therapy
Children’s Hospital of Pittsburgh

Day One

Thursday 13th December 2018

5:20 pm | Live Presenter Q&A

5:00 pm | Gene therapy for inborn errors metabolism. Moving from preclinical studies to clinical trials.

Hung Do
Chief Scientific Officer
Amicus Therapeutics

Day One

Thursday 13th December 2018

10:20 am | Live Presenter Q&A

9:20 am | A Novel Approach for Producing More Potent Therapeutic Enzymes & Enhancing In Vivo Efficacy of Gene Therapies

Jonathan Schwartz
Chief Medical Officer
Rocket Pharmaceuticals

Day One

Thursday 13th December 2018

12:50 pm | Live Presenter Q&A

12:10 pm | Confidently Translate from Small Animals into Larger Animals & Humans

Kevin Strauss
Medical Director
Clinic for Special Children

Day One

Thursday 13th December 2018

5:20 pm | Live Presenter Q&A

4:20 pm | Natural History of Maple Syrup Urine Disease: A Case Study in Clinical Trial Readiness

Manolo Bellotto
President & General Manager
Gain Therapeutics

Day One

Thursday 13th December 2018

10:20 am | Live Presenter Q&A

9:40 am | Pathophysiological mechanisms and possibilities for combinatorial therapeutic intervention in rare disease: the example of Lysosomal storage diseases (LSDs)

Paolo Martini
Chief Scientific Officer Rare Diseases
Moderna Therapeutics

Day One

Thursday 13th December 2018

10:20 am | Live Presenter Q&A

10:00 am | An mRNA Therapeutic Approach to Targeting Inborn Errors of Metabolism

Roberto Nitsch
Associate Director of Clinical Pharmacology & Safety Sciences for CRISPR Medicines
AstraZeneca

Day One

Thursday 13th December 2018

12:50 pm | Live Presenter Q&A

1:20 pm | Panel Discussion: Discuss the Safety of Different Viral & Non-Viral Vectors

12:30 pm | Safety considerations to deliver better CRISPR medicines

Stephen Strom
Senior Professor
Karolinska Institute

Day One

Thursday 13th December 2018

12:50 pm | Live Presenter Q&A

11:50 am | Assessing Animal Models vs Cell-Based Systems for Recapitulating the Human Disease State