The past few years have seen some remarkable successes for gene replacement therapy in diseases, including hereditary blindness, sickle-cell disease, and muscular dystrophy. With the promise of genome editing entering the clinic, there is palpable excitement
for the future of genetic therapy. The Inaugural Renaissance of Gene Therapy and Genome Editing conference program at the 27th International Molecular Medicine Tri-Conference will showcase topics spanning fundamental research, genome editing (CRISPR
and zinc-finger nucleases), preclinical and clinical investigation, as well as considering manufacturing, delivery, and ethical challenges. Hear from leaders in the field of gene therapy and genome editing about the latest advances, future hurdles,
and learn where the current-state-of-the art is for changing the course of disease in our time.
Scientific Advisory Board
Kevin Davies, PhD, Executive Editor, The CRISPR Journal, Mary Ann Liebert, Inc.
Ross Wilson, PhD, Project Scientist and Principal Investigator, UC Berkeley & the Innovative Genomics Institute
Monday, March 2
10:30 Conference Program Registration Open
11:45 Organizer’s Opening Remarks
Christina Lingham, Executive Director, Conferences and Fellow, Cambridge Healthtech Institute
11:50 Chairperson’s Remarks
Kevin Davies, PhD,
Executive Editor, The CRISPR Journal, Mary Ann Liebert, Inc.
11:55 Climbing Mt. OMIM: Genome Editing and "Rare" Disease
Fyodor Urnov, PhD, Professor, Department of Molecular and Cell Biology, University of California, Berkeley and Director, Innovative Genomics Institute
12:25 pm Genome Editing of Stem Cells to Create Human Medicines
Matthew Porteus, MD, Professor, Pediatrics, Stanford School of Medicine
Genome editing provides a method to precisely change the DNA sequence of a cell. It can be used to make single nucleotide changes or to precisely insert new genes into cells. We have developed a system combining the use of Cas9, synthetic guide RNAs,
and AAV6 that is highly efficient in homologous recombination-based genome editing in a wide variety of primary human cells. I will discuss the development of this system and our use of this system to develop genetically engineered, cell-based drugs
for a variety of diseases.
12:55 Session Break
1:05 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own
2:05 Session Break
2:20 Chairperson’s Remarks
Kevin Davies, PhD,
Executive Editor, The CRISPR Journal, Mary Ann Liebert, Inc.
2:25 Correction of Duchenne Muscular Dystrophy by Genome Editing
Leonela Amoasii, PhD, Director, Gene Editing Research, Vertex Genetic Therapies
The Dystrophin protein connects the muscle cytoskeleton and extracellular matrix to maintain muscle integrity and function. Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), the most common fatal genetic disorder diagnosed in childhood.
The majority of DMD mutations are deletions that prematurely terminate the dystrophin protein. The major genomic “hot spot” for DMD deletions encompasses exon 50, which places exon 51 out of frame with preceding exons. To investigate the
potential of CRISPR/Cas9 gene editing for correction of DMD, we used adeno-associated virus to deliver Cas9 and a single-guide RNA in mice and canine models of DMD. By both intramuscular and systemic delivery of gene editing components, our approach
led to efficient restoration of dystrophin protein expression in the affected. Our method bypassing DMD mutations using a single cut in genomic DNA represents a significant step toward clinical correction of DMD and other neuromuscular disorders.
2:55 AAV Gene Therapy: Translating from the Bench to the Clinic
Allison Keeler-Klunk, PhD, Assistant Professor, Pediatrics, University of Massachusetts Medical School
AAV gene therapy has made great strides in the last several years, with two AAV-based gene therapies recently receiving FDA approval. Pioneering work by Dr. Terence Flotte in translating AAV-based gene therapy for Alpha-1 antitrypsin deficiency and
ongoing clinical trials for both Alpha-1 antitrypsin deficiency and Tay-Sach’s disease will be discussed. Through this work, we have found T regulatory cells against AAV capsid that sustain long-term expression of transgene in the absence
of immune suppression.
3:25 Directed Evolution of New AAV Vectors for Gene Therapy and Genome Editing
David Schaffer, PhD, Hubbard Howe Jr. Distinguished Professor, Chemical and Biomolecular Engineering, Bioengineering, Molecular and Cell Biology, and the Helen Wills Neuroscience Institute, University of California, Berkeley; Director, Berkeley
Stem Cell Center
Gene therapy has succeeded in increasing numbers of human trials and approved products, establishing the strong therapeutic promise of viral vectors. However, highly efficient and targeted delivery remains a broad challenge. We developed and implemented
directed evolution, a process that emulates natural evolution by generating large libraries of biomolecules and selecting for enhanced function, to greatly enhance the properties of numerous viral vectors, particularly adeno-associated virus,
for clinical gene delivery.
3:55 FEATURED PRESENTATION: Facilitating the Use of Gene Therapy for Rare Disorders
Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration
It is becoming apparent that individualized therapeutics will become increasingly important to address the variety of molecular defects present in several thousand serious, rare diseases. Some of these therapies may be targeted to as many as a few
thousand individuals, but others only may be targeted to one or few individuals. Novel approaches in manufacturing, preclinical and clinical assessment, and regulatory evaluation that are necessary will be discussed.
4:25 Refreshment Break and Transition to Plenary Keynote
4:35 Welcome Remarks
Cindy Crowninshield, RDN, LDN, HHC, Executive Event Director, Cambridge Healthtech Institute
4:45 PLENARY KEYNOTE INTRODUCTION
Thomas Westerling-Bui, PhD, Senior Scientist, Regional Business Development, Aiforia
5:00 PLENARY KEYNOTE PRESENTATION: High-Performance Medicine
Eric Topol, MD, Founder and Director, Scripps Research Translational Institute (SRTI); Author, Deep Medicine: How Artificial
Intelligence Can Make Healthcare Human Again
6:00 Grand Opening Reception in the Exhibit Hall with Poster Viewing, Speed Networking, Book Signing, and Meetup Group
7:30 End of Day
Tuesday, March 3
7:30 am Registration Open and Morning Coffee
8:00 Organizer’s Remarks
Christina Lingham, Executive Director, Conferences and Fellow, Cambridge Healthtech Institute
8:05 Chairperson’s Remarks
Ross Wilson, PhD, Project Scientist and Principal Investigator, UC Berkeley & the Innovative Genomics Institute
8:10 FEATURED PRESENTATION: CRISPR’d Babies: He Jiankui and Beyond
Henry T. Greely, JD, Professor by courtesy of Genetics, Stanford School of Medicine; Director, Center for Law and the Biosciences; Director, Stanford Program in Neuroscience and Society; Chair, Steering Committee of the Center for Biomedical
Ethics
He Jiankui shocked the world in late November 2018 by announcing the birth of two baby girls whose DNA he had edited while they were embryos. This talk will critique the He Jiankui experiment (spoiler – it was criminally reckless), suggest some
important steps “Science” should take, and discuss the broader question of possible non-reckless human germline genome editing.
8:40 Genome Editing’s Last Mile: The Delivery Technologies Enabling Translation
Ross Wilson, PhD, Project Scientist and Principal Investigator, UC Berkeley & the Innovative Genomics Institute
Genome-editing enzymes, such as CRISPR-Cas9, have been successfully employed to target different genetic disorders in model systems, and ex vivo approaches have demonstrated the clinical potential of this technology.
However, the challenge of delivering such corrective enzymes in vivo represents a substantial barrier to therapeutic translation. Viral vectors – refined during their use in traditional gene therapy –
represent the most widely-used delivery platform, and nanoparticles are quickly maturing into a comparably powerful vehicle. Along with non-traditional and emerging approaches to delivery, there is a lush diversity of technologies that are enabling
the clinical translation of genome editing. I will review the recent history and likely future of these crucial delivery platforms.
9:10 Genome Editing and Transcriptional Regulation Using Synthetic Zinc Finger Nucleases
Adrian Woolfson, BM, BCh, PhD, Executive Vice President, R&D, Sangamo Therapeutics
Synthetic Zinc Finger Proteins (ZFPs) comprise a versatile platform for editing and regulating genes of therapeutic relevance and can target virtually any position within the human genome. Clinical-grade ZFPs can be rapidly developed and optimized
using mix-and-match assembly of pre-defined components, including finger modules and base-skipping linkers, and tuned to provide high specificity. This presentation will detail the optionality and exquisite electivity of the platform across several
disease indications.
9:40 Refreshment Break in the Exhibit Hall with Poster Viewing, Speed Networking, Book Signing, and Meetup Group
Chairperson’s Remarks
Ross Wilson, PhD, Project Scientist and Principal Investigator, UC Berkeley & the Innovative Genomics Institute
10:40 Regulatory Approaches for Development of CAR T Therapies
Elena Spanjaard, PhD, Global Head of Regulatory Affairs, Regulatory Affairs, Celyad
I will define IND requirements for genetically-modified CAR T therapies and discuss the tailored regulatory strategies to address unique program features.
11:10 Personalized Multi-Targeted Adoptive Cell Therapy
Steffen Walter, PhD, CSO, Immatics US
Despite its great potential, adoptive cellular therapy (ACT) has shown limited clinical success in solid tumors. Major challenges of ACT in solid tumors include heterogeneity of tumor antigen expression, tumor escape (e.g. after addressing only one
target) and toxicities (e.g. due to expression of targets on healthy tissue). In this presentation, we will show recent data from several complementary clinical-stage approaches to treat solid tumors using personalized combinations of multiple
novel targets.
11:40 Accelerating Biology in True Resolution - Single-Cell Genomics for Gene Editing and Cellular Therapies
Brian R. Fritz, PhD, Associate Director, AMR Regional Marketing, 10X Genomics
12:10 pm Session Break
12:20 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own
1:20 Refreshment Break in the Exhibit Hall with Poster Viewing, Speed Networking, Book Signing, and Meetup Group
2:00 Breakout Discussions in the Exhibit Hall (please click here for details)
3:00 Transition to Keynote Session
3:15 Organizer’s Remarks
Christina Lingham, Executive Director, Conferences and Fellow, Cambridge Healthtech Institute
3:20 Keynote Introduction
Allison Mallory, PhD, Director, R&D Molecular Biology, Stilla Technologies
3:35 What Does the New Era of Genomic Medicine Look Like? Effects on Patient Care, Therapeutics, and Diagnostics
20 years after the completion of the first draft of the Human Genome Project, there is compelling evidence of genomics delivering the rich promise of precision medicine. There have been major advances in the throughput and affordability of genome
sequencing, enhanced tools for genome analysis and interpretation, new paradigms for therapeutics and strong signs of clinical benefit using genome editing. But major challenges remain. In this special plenary roundtable, three established pioneers
of genomic medicine – David Haussler, Stephen Kingsmore, and Liz Worthey – offer their insights on the extraordinary advances in genomic medicine over the past 1-2 decades and share their hopes and concerns for the future of our field.
Moderator: Kevin
Davies, PhD, Executive Editor, The CRISPR Journal, Mary Ann Liebert, Inc.
Panelists: Stephen Kingsmore, MD, DSc, President/CEO, Rady Children’s Institute for Genomic Medicine
David Haussler, PhD, Investigator, Howard Hughes Medical Institute; Distinguished Professor, Biomolecular Engineering, University of California, Santa Cruz; Scientific Director, UC Santa Cruz Genomics Institute; Scientific Co-Director, California
Institute for Quantitative Biosciences (QB3)
Elizabeth Worthey, PhD, Director, Genomic Medicine, University of Alabama, Birmingham School of Medicine
4:50 Spring Fling Celebration in the Exhibit Hall with Poster Viewing, Speed Networking, Book Signing, and Meetup Group
6:00 Close of Conference