Renaissance of Gene Therapy and Genome Editing

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.

Final Agenda

Day 1 | Day 2 | Download Brochure

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

Sunday, March 1

2:00 - 5:00 pm Afternoon Short Courses*

*Separate registration required

5:30 - 8:30 Dinner Short Courses*

*Separate registration required

Monday, March 2

8:00 - 11:00 am Morning Short Courses*

*Separate registration required

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

Davies_Kevin Kevin Davies, PhD, Executive Editor, The CRISPR Journal, Mary Ann Liebert, Inc.

11:55 Climbing Mt. OMIM: Genome Editing and "Rare" Disease

Urnov_Fyodor 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

Porteus_Matthew 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

Davies_Kevin Kevin Davies, PhD, Executive Editor, The CRISPR Journal, Mary Ann Liebert, Inc.

2:25 Correction of Duchenne Muscular Dystrophy by Genome Editing

Amoasii_Leonela 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

Keeler-Klunk_Allison 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

Schaffer_David 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

Marks_Peter 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

(please see Keynote pages for details)

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

Day 1 | Day 2 | Download Brochure

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

Wilson_Ross 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

Greely_Hank 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

Wilson_Ross 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

Woolfson_Adrian 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

Wilson_Ross Ross Wilson, PhD, Project Scientist and Principal Investigator, UC Berkeley & the Innovative Genomics Institute

10:40 Regulatory Approaches for Development of CAR T Therapies

Spanjaard_Elena 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

Walter_Steffen 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

Mallory_Allison 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.

Davies_Kevin Moderator: Kevin Davies, PhD, Executive Editor, The CRISPR Journal, Mary Ann Liebert, Inc.

Kingsmore_Stephen Panelists: Stephen Kingsmore, MD, DSc, President/CEO, Rady Children’s Institute for Genomic Medicine

Haussler_David 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)