Program | ASGCT 25th Annual Meeting

The most up-to-date ASGCT Annual Meeting agenda

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Pre-Meeting Workshops

Career Fair & Mentor Meet-Up

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Last update: 5/13/22

Sunday, May 15 (Pre-meeting Workshops)
8 a.m. - 5 p.m.
Monday, May 16
8 a.m. - 7 p.m.
Tuesday, May 17
8 a.m. - 7 p.m.
Wednesday, May 18
8 a.m. - 7 p.m.
Thursday, May 19
8 a.m. - 12 p.m.

SESSION DETAILS

Viral Vector Development Workshop

This workshop will provide guidance on how to choose, design, and produce the appropriate viral vect...

08:00 AM – 08:20 AM - Room 202

Overview of Viral Vector Platforms

Negin P. Martin, PhD. NIEHS, Research Triangle Park, NC

08:00 AM – 12:00 PM - Room 202

Co-Chair

Anna Maurer. University of California Berkeley, CA

08:00 AM – 12:00 PM - Room 202

Co-Chair

Carol Miao. Seattle Children's Hospital, WA

08:20 AM – 08:40 AM - Room 202

Lentiviral Vector Design and Applications

Axel Schambach. Hannover Medical School, Hannover, Germany

08:40 AM – 09:00 AM - Room 202

Adenoviral Vector Design and Applications for Immune Mediated Gene Therapy: From the Bench to Clinical Implementation

Maria G. Castro, PhD. University of Michigan Medical School, Ann Arbor, MI

09:00 AM – 09:20 AM - Room 202

From the Lab to the Clinic: Designing Preclinical Programs to Assess Efficacy and Safety for Novel Viral Vectors

Eva Andres-Mateos, PhD. Atsena Therapeutics, NC

10:00 AM – 10:20 AM - Room 202

AAV Vector Development

Mark Kay, PhD. Stanford, CA

10:20 AM – 10:40 AM - Room 202

AAV Capsid Engineering and Library Approaches

Ben Deverman, PhD. Broad Institute, MA

10:40 AM – 11:00 AM - Room 202

AAV Vector Production from Small to Large Scales

Nathalie Clement, PhD. Unicorn Consultations, LLC, San Francisco, CA

11:00 AM – 11:20 AM - Room 202

AAV Vectors: Transgene Design

Charles Venditti, MD, PhD. National Human Genome Research Institute, Bethesda, MD

11:20 AM – 11:40 AM - Room 202

AAV Genomics and Its Implication in Gene Therapy

Weidong Xiao, PhD. Indiana University, IN

08:00 AM – 12:00 PM - Salon G - Pre-Meeting Program

A Focus on Gene and Cell Therapy for Clinicians

This workshop is designed for clinicians with experience caring for patients for which a gene or cel...

08:00 AM – 12:00 PM - Salon G

Co-Chair

Sunil S.. Raikar, MD. Emory University, Atlanta, GA

08:00 AM – 12:00 PM - Salon G

Co-Chair

Terence R.. Flotte, MD. , MA

08:00 AM – 08:20 AM - Salon G

A Focus on Gene and Cell Therapy for Clinicians - Introduction: What Therapies are Available Today

Terence R.. Flotte, M.D.. UMass Chan Medical School, Worcester, MA

08:20 AM – 08:45 AM - Salon G

Types of Therapies - Lentiviral/In-vivo HSC

Matthew Porteus, MD, PhD. , CA

08:45 AM – 09:10 AM - Salon G

Types of Therapies - AAV

Jerry R. Mendell, MD, PhD. The Research Institute at Nationwide Children's Hospital, Columbus, OH

09:10 AM – 09:35 AM - Salon G

Types of Therapies - CAR-T Therapies

David L. Porter, MD. , PA

09:35 AM – 10:00 AM - Salon G

Clinical Trials and My Role - How do I Find a Clinical Trial?

Florian Eichler, MD. Massachusetts General Hospital, Boston, MA

10:00 AM – 10:25 AM - Salon G

Clinical Trials and the Role of FDA - Identifying Definitive Clinical Outcomes

Peter Marks. Food & Drug Administration, Silver Spring, MD

10:25 AM – 10:50 AM - Salon G

Running a Clinical Trial

Kathryn R.. Wagner, MD, PhD. F. Hoffmann-La Roche, Basel, Switzerland

11:05 AM – 11:30 AM - Salon G

Ethics of Recommending a Clinical Trial

Alison Bateman-House, MPH, MA. New York University, New York, NY

11:30 AM – 11:55 AM - Salon G

Hospital Pharmacy Role in Managing Insurance Issues

Michael Storey, PharmD, MS. Nationwide Children's Hospital, Columbus, OH

08:00 AM – 12:00 PM - Room 206 - Pre-Meeting Program

Newborn Screening: Toward a System That Keeps Pace with Gene Therapy Advances Workshop (Organized by the Government Relations Committee)

This workshop will dive into the key policy issues affecting the U.S. newborn screening system, give...

08:00 AM – 08:15 AM - Room 206

Potential Solutions to Prepare Newborn Screening for Transformative Therapies

Don Bailey, PhD. RTI International, Research Triangle Park, NC

08:00 AM – 12:00 PM - Room 206

Co-Chair

Diane Berry, PhD. Sarepta Therapeutics, MA

08:00 AM – 12:00 PM - Room 206

Co-Chair

Meagan Perry. Orchard Therapeutics, DC

08:15 AM – 08:25 AM - Room 206

Streamlining Future RUSP Applications

Terri Klein, MPA. National MPS Society, NC

08:25 AM – 08:55 AM - Room 206

Roundtable Panel: The Recommended Uniform Screening Panel (RUSP) Current and Future Process

Kent Christopherson{} Orchard Therapeutics, MA

08:25 AM – 08:55 AM - Room 206

Roundtable Panel: The Recommended Uniform Screening Panel (RUSP) Current and Future Process

Stacey Frisk{} Sarepta Therapeutics, MA

08:25 AM – 08:55 AM - Room 206

Roundtable Panel: The Recommended Uniform Screening Panel (RUSP) Current and Future Process

Elisa Seeger. Aiden Jack Seeger Foundation, NY

08:55 AM – 09:05 AM - Room 206

Hastening the State Uptake of Newly Approved Conditions

Erin Frey. Ultragenyx, Novato, CA

09:05 AM – 09:45 AM - Room 206

Roundtable Panel: State RUSP Implementation and Innovation

Annie Kennedy. EveryLife Foundation, DC

09:05 AM – 09:45 AM - Room 206

Roundtable Panel: State RUSP Implementation and Innovation

Stacey Frisk{} Sarepta Therapeutics, MA

09:05 AM – 09:45 AM - Room 206

Roundtable Panel: State RUSP Implementation and Innovation

Scott Shone, PhD. HCLD – NC State Laboratory of Public Health, NC

09:05 AM – 09:45 AM - Room 206

Roundtable Panel: State RUSP Implementation and Innovation

Kent Christopherson{} Orchard Therapeutics, MA

10:15 AM – 10:25 AM - Room 206

Data Collection and Newborn Screening Programs: Opportunities of a Multi-Disease Panel Approach

Melissa Wasserstein, MD. Montefiore Medical Center, NY

10:25 AM – 10:35 AM - Room 206

Data Collection and Newborn Screening Programs: State Pilot Programs

Joeseph Orsini, PhD. New York State Department of Health, NY

10:35 AM – 11:05 AM - Room 206

Roundtable Panel: Data Collection and Newborn Screening Programs

Barbara Burton, MD. Lurie's Children's Hospital, IL

10:35 AM – 11:05 AM - Room 206

Roundtable Panel: Data Collection and Newborn Screening Programs

Amy Brower, PhD. ACMG, MD

10:35 AM – 11:05 AM - Room 206

Roundtable Panel: Data Collection and Newborn Screening Program

Kent Christopherson{} Orchard Therapeutics, MA

10:35 AM – 11:05 AM - Room 206

Roundtable Panel: Data Collection and Newborn Screening Programs

Stacey Frisk{} Sarepta Therapeutics, MA

11:05 AM – 11:15 AM - Room 206

Evaluating Emerging Screening Methods and Clinical Programs

Richard Parad, MD. , MA

11:15 AM – 11:25 AM - Room 206

Evaluating Emerging Screening Methods and Clinical Programs

Stephen Kingsmore, MD, DSc. Rady Children's Hospital, CA

11:25 AM – 11:55 AM - Room 206

Roundtable Panel: Evaluating Emerging Screening Methods and Clinical Programs

Kent Christopherson{} Orchard Therapeutics, MA

11:25 AM – 11:55 AM - Room 206

Roundtable Panel: Evaluating Emerging Screening Methods and Clinical Programs

Stacey Frisk{} Sarepta Therapeutics, MA

08:00 AM – 12:00 PM - Room 102 A/B - Pre-Meeting Program

Emerging Clinical and Pre-Clinical Approaches to Oncolytics

Oncolytic virus therapy has the potential to become a new arm of anti-cancer treatment, potentially ...

08:00 AM – 08:25 AM - Room 102 A/B

Progress and approval of G47Δ

Tomoki Todo, MD, PhD. Division of Innovative Cancer Therapy and Department of Surgical Neuro-Oncolog...

08:25 AM – 08:50 AM - Room 102 A/B

Development of Synthetic RNA Viral Immunotherapy

Jeff Bryant, Oncorus, Inc., MA

08:50 AM – 09:15 AM - Room 102 A/B

Drug Modulation of the Myeloid Tumor Microenvironment to Enable Oncolytic HSV Immunotherapy.

Tim Cripe, MD, PhD. Nationwide Children's Hospital, Columbus, OH

09:15 AM – 09:40 AM - Room 102 A/B

Combinations of Early Trials

Howard Kaufman. Harvard University, Woburn, MA

09:55 AM – 10:20 AM - Room 102 A/B

A Therapy Going Viral: Armed-oncolytic Adenoviruses for the Treatment of Solid Tumors

Candelaria Gomez-Manzano, MD. MD Anderson Cancer Center, TX

10:20 AM – 10:45 AM - Room 102 A/B

An Oncolytic Herpes Virus Armed with a Full-length Anti-CD47 Antibody to Target “Don’t Eat Me” Signal and Fc Receptor-Mediated Antitumor Immunity

Jianhan Yu. City of Hope National Medical Center, CA

10:45 AM – 11:10 AM - Room 102 A/B

Bacterial Antigens Harnessed for Virotherapy

Evanthia Galanis, MD, DSc. Mayo Clinic, Rochester, MN

11:10 AM – 11:35 AM - Room 102 A/B

Update in Clinical Trials of Oncolytic HSV for Brain Tumors

James M. Markert, MD, MPH. University of Alabama at Birmingham, AL

08:00 AM – 12:00 PM - Room 201 - Pre-Meeting Program

Coming of Age: Stem Cell Therapies - from Bench to Clinic

Curative stem cell therapies have been a dream for many researchers in the field. In the past 20-30 ...

08:00 AM – 12:00 PM - Room 201

Co-Chair

Tony Ho. Johns Hopkins University, MD

08:00 AM – 08:25 AM - Room 201

Development of Dual HDR Edited EngTreg for T1D Therapy

David Rawlings, MD. University of Washington, WA

08:00 AM – 12:00 PM - Room 201

Co-Chair

M Graca Almeida-Porada, Wake Forest Institute for Regenerative Medicine, Winston Salem, NC

08:25 AM – 08:50 AM - Room 201

Immune Evasive Cells

Ron Evans, PhD. The Salk Institute, CA

08:50 AM – 09:15 AM - Room 201

Using RBC to Deliver mRNAs

Ionita Giran, MD. Beth Israel Deaconess Medical Center, MA

09:15 AM – 09:40 AM - Room 201

Bioengineered Enucleated Stem Cells For Therapeutic Delivery

Richard Klemke. University of California - San Diego, -, CA

09:55 AM – 10:20 AM - Room 201

CRISPR Edited Islet Cells

Ali Rezania, PhD. CRISPR Therapeutics, MA

10:20 AM – 10:45 AM - Room 201

Liver Stem Cells

Lola Reid, UNC School of Medicine, NC

10:45 AM – 11:10 AM - Room 201

BOOSTB4: a European clinical trial investigating pre- and postnatal MSC transplantation as a treatment of Osteogenesis Imperfecta

Cecilia Cecilia Götherström, PhD. Karolinska Institutet, Sweden

11:10 AM – 11:35 AM - Room 201

NK cells

Yu-Waye Chu, MD. Fate Therapeutics, CA

08:00 AM – 12:00 PM - Room 207 - Pre-Meeting Program

Genome Editing Workshop - The Ever Expanding Editing Toolbox

Gene editing has become an essential technology in our day-to-day research and it is a promising app...

08:00 AM – 08:25 AM - Room 207

Enhancing HDR Efficiency in Human Pluripotent Stem Cells

Sridhar Selvaraj, PhD. Stanford University, Stanford, CA

08:25 AM – 08:50 AM - Room 207

Cas9-HRs: Manipulating DSB Repair Pathway Choice to Increase HDR

Chris Hackley, PhD. CRISP-HR Therapeutics, CA

08:50 AM – 09:15 AM - Room 207

Improved Methods for CRISPR HDR Research Using Alt-R™ Modified Donors and Alt-R HDR Enhancer V2

Mollie Schubert. Integrated DNA Technologies, IA

09:15 AM – 09:40 AM - Room 207

Multiplexed Genome Regulation in Vivo

Lucie Guo, MD, PhD. Stanford, CA

09:40 AM – 10:05 AM - Room 207

Growing the CRISPR Toolbox for Genetic Medicines

David Scott. Arbor Biotechnologies, MA

10:20 AM – 10:45 AM - Room 207

Identifying and Engineering Orthogonal and Synthetic RNA-Guided Nucleases (sRGN)

Andre Cohnen, PhD. Bayer AG, Germany

10:45 AM – 11:10 AM - Room 207

Treatment of Metabolic Liver diseases by in vivo Prime Editing

Gerald Schwank. University of Zurich, Switzerland

11:10 AM – 11:35 AM - Room 207

Targeting Double-strand Break Indel Byproducts with Secondary Guide RNAs Improves Cas9 HDR-mediated Genome Editing Efficiencies

Zsolt Bodai. University of California - San Diego, CA

11:35 AM – 12:00 PM - Room 207

Programmable Transposon-encoded RNA-guided Nucleases for Genome Editing

Soumya Kannan. Massachusetts Institute of Technology, MA

08:00 AM – 12:00 PM - Room 204 - Pre-Meeting Program

Issues and Trends in Clinical Development for Cell and Gene Therapies (Organized by the Regulatory Affairs Committee)

The workshop will discuss strategies for optimizing gene and cell therapies' clinical development. I...

08:00 AM – 08:20 AM - Room 204

Improving Clinical Pediatric R&D for Cell and Gene Therapies

Brian Tseng, MD. Unaffiliated, MA

08:00 AM – 12:00 PM - Room 204

Co-Chair

Daniela Drago, PhD. Aurion Biotech, MA

08:00 AM – 12:00 PM - Room 204

Co-Chair

Kaye M. Spratt, PhD. Senior Vice President, Regulatory Affairs, BridgeBio Gene Therapy, San Francisc...

08:20 AM – 08:40 AM - Room 204

Regulatory Perspective on Pediatric Studies

Elizabeth Hart, MD. FDA, MD

09:00 AM – 09:20 AM - Room 204

Managing Unexpected Adverse Events in Gene Therapy Clinical Trials

Bernhardt Zeiher, MD. Astellas Pharma, Northbrook, IL

09:20 AM – 09:40 AM - Room 204

Regulatory Considerations and Developmental Impact of Unexpected Adverse Events: DMD Experience

Natalie A. Schmidt. Pfizer, United Kingdom

09:40 AM – 10:00 AM - Room 204

Panel Discussion: When Things Go Wrong - Effectively Managing Adverse Events During Product Development

Larissa Lapteva, MD. U.S. Food and Drug Administration, Silver Spring, MD

10:30 AM – 11:00 AM - Room 204

Developing Innovative Clinical Trials for Rare Diseases: Danon Disease Experience

Jonathan D. Schwartz, MD. Rocket Pharmaceuticals, Cranbury, NJ

11:00 AM – 11:30 AM - Room 204

Regulatory Considerations for Clinical Trials with Advanced Therapies

Larissa Lapteva, MD. U.S. Food and Drug Administration, Silver Spring, MD

11:30 AM – 12:00 PM - Room 204

Panel Discussion: Innovation in Clinical Trial Design for Cell and Gene Therapies

Snehal Naik{} PA

01:00 PM – 05:00 PM - Room 206 - Pre-Meeting Program

Kick-Starting My Career in BioIndustry

The BioIndustry holds many opportunities for personal and professional growth, but how is it that I ...

01:00 PM – 01:55 PM - Room 206

Considerations for MDs & PhDs on transitioning to industry: How do I even know what jobs I should apply for, where they are, and how to access them?

Eric David, MD, JD. BridgeBio, Palo Alto, CA

01:55 PM – 02:50 PM - Room 206

I’m Now in BioIndustry: Where Do I Start and What Do I Do?

Ashton Lavoie, PhD. BridgeBio Gene Therapy, NC

01:55 PM – 02:50 PM - Room 206

I'm Now in BioIndustry: Where Do I Start and What Do I Do?

Amber Van Laar. AskBio, Columbus, OH

03:35 PM – 04:00 PM - Room 206

Planning My Career: Who will be My Board of Directors and Sponsors?

Bernard Ravina, MD, MS. Praxis Precision Medicines, Boston, MA

04:00 PM – 04:30 PM - Room 206

Life, Edited: Lessons For a Basic Scientist Building Gene-Editing Cures in Industry

Fyodor Urnov, PhD. Berkeley, CA, Innovative Genomics Institute, CA

04:30 PM – 05:00 PM - Room 206

Difference Between Structure and Culture (Academia vs. Industry): Value of Transparency

Chris Mason. , MA

01:00 PM – 05:00 PM - Room 202 - Pre-Meeting Program

AAV Vector Integration Workshop

While AAV vector genomes primarily persist as episomes, low rates of vector integration into the hos...

01:00 PM – 01:20 PM - Room 202

Overview: Knowns and Unknowns of AAV Integration Risk

Mark Kay, MD, PhD. , CA

01:00 PM – 05:00 PM - Room 202

Co-Chair

Guangping Gao, PhD. U Mass Med, Worcester, MA

01:00 PM – 05:00 PM - Room 202

Co-Chair

Mark Kay, MD, PhD. Stanford University, CA

01:20 PM – 01:40 PM - Room 202

The Impact of CRISPR Editing in Rodent CNS

Beverly L. Davidson, PhD. Childrens Hosp. of Philadelphia, Philadelphia, PA

01:40 PM – 02:00 PM - Room 202

Sequence based methods for quality control and assessing outcome in gene therapy

Frederic D. Bushman, PhD. University of Pennsylvania School of Medicine, Philadelphia, PA

02:00 PM – 02:25 PM - Room 202

Humanized Mouse Liver Systems

Markus Grompe, MD. Oregon Health & Science University, OR

02:25 PM – 02:55 PM - Room 202

AAV Vector Integration Behavior in Primary Human Hepatocytes in the FRG Mouse Liver

Ian E. Alexander, MBBS, PhD. The Children's Hospital at Westmead, Westmead, Australia

03:10 PM – 03:35 PM - Room 202

Pathogenesis of Hepatocellular Carcinoma and Implications for Clinical Monitoring

Dean Felsher, MD, PhD. Stanford University School of Medicine, CA

03:35 PM – 04:00 PM - Room 202

Genomics of Hepatocellular Carcinoma: Role of Viral Insertional Mutagenesis

Jessica Zucman-Rossi, MD, PhD. Sorbonne Université-Inserm-Université de Paris, France

04:00 PM – 04:20 PM - Room 202