Program

10:25 AM – 11:55 AM - Petree Hall C

Panelist

Emily Walsh. , MA

10:25 AM – 11:55 AM - Petree Hall C

Panelist

Hildegard Buning, Ph.D.. Hannover Medical School, Hannover, Germany

10:25 AM – 11:55 AM - Petree Hall C

Panelist

Avery D. Posey, PhD. University of Pennsylvania, Philadelphia, PA

10:25 AM – 11:55 AM - Petree Hall C

Panelist

Federico Mingozzi, PhD. Spark Therapeutics, Philadelphia, PA

8:00 AM – 12:00 PM - Petree Hall C

Co-Chair

Marina Pavlou, PhD. University of Washington, Seattle, WA

8:00 AM – 12:00 PM - Petree Hall C

Co-Chair

Nerea Zabaleta, PhD. Mass Eye and Ear/Harvard Medical School, Boston, MA

8:01 AM – 8:26 AM - Petree Hall C

How to Network at a Conference

Hildegard Buning, Ph.D.. Hannover Medical School, Hannover, Germany

8:25 AM – 8:50 AM - Petree Hall C

How to Maximize your Use of Social Media During the Conference

Emily Walsh, PhD. Tremont Therapeutics, MA

8:50 AM – 9:15 AM - Petree Hall C

Anticipating where the field is going

Federico Mingozzi, PhD. Spark Therapeutics, Philadelphia, PA

9:15 AM – 9:40 AM - Petree Hall C

Diversifying your Network to Improve Collaboration and Innovation

Avery D. Posey, Jr., PhD. University of Pennsylvania, Philadelphia, PA

10:30 AM – 11:15 AM - Concourse Hall 152 & 153

Follow up and Long-term Toxicity: What needs to be followed for follow up?

Denise E.. Sabatino, PhD. The Children's Hospital of Philadelphia, Philadelphia, PA

11:15 AM – 12:00 PM - Concourse Hall 152 & 153

Panelist

Denise E.. Sabatino, PhD. The Children's Hospital of Philadelphia, Philadelphia, PA

11:15 AM – 12:00 PM - Concourse Hall 152 & 153

Panelist

Kimberly Goodspeed, MD, MSCS. University of Texas Southwestern Medical Center, Dallas, TX

11:15 AM – 12:00 PM - Concourse Hall 152 & 153

Panelist

Mark A.. Kay, MD, PhD. Stanford University School of Medicine, Stanford, CA

11:15 AM – 12:00 PM - Concourse Hall 152 & 153

Panelist

Sandra Nino-Siddens. Ultragenyx Pharmaceutical Inc., Novato, CA

8:00 AM – 12:00 PM - Concourse Hall 152 & 153

Co-Chair

Nicolas J. Abreu, MD. NYU Langone, New York, NY

8:00 AM – 12:00 PM - Concourse Hall 152 & 153

Co-Chair

Charles P.. Venditti, PhD, MD. National Human Genome Research Institute, National Institutes of Heal...

8:01 AM – 8:45 AM - Concourse Hall 152 & 153

Preclinical/IND-enabling Basics of AAV Biology and GT Vectors Preclinical Enablement and the IB

Mark A.. Kay, MD, PhD. Stanford University School of Medicine, Stanford, CA

8:45 AM – 9:30 AM - Concourse Hall 152 & 153

Regulatory Considerations for Initiating Gene Therapy Clinical Studies

Sandra Nino-Siddens, PharmD. Ultragenyx Pharmaceutical Inc., CA

9:45 AM – 10:30 AM - Concourse Hall 152 & 153

Clinical Protocol Implementation: Who sees the Patient and What is Needed? Clinical Infrastructure Treating the Patient/Acute Monitoring Phase I/II vs III vs IV

Kimberly Goodspeed, MD. University of Texas Southwestern Medical Center, TX

10:05 AM – 10:30 AM - Room 408 AB

Market Approved Therapies- Focus on ex vivo Lenti Gene Therapy to Cover Other Technologies

Jasmine Greenamyer. Bristol Myers Squibb, DC

10:30 AM – 10:55 AM - Room 408 AB

Market Approved Therapies- focus on ex vivo lenti gene therapy to cover other technologies

Clark Paramore, MSPH. bluebird bio, MA

11:05 AM – 12:00 PM - Room 408 AB

Panelist

Clark Paramore, MSPH. , MA

11:05 AM – 12:00 PM - Room 408 AB

Panelist

Jasmine Greenamyer. , Lawrenceville, NJ

11:05 AM – 12:00 PM - Room 408 AB

Panelist

Barry J.. Byrne, MD, PhD. University of Florida, Gainesville, FL

11:05 AM – 12:00 PM - Room 408 AB

Panelist

Alison Bateman-House, PhD, MPH, MA. NYU Grossman School of Medicine, New York, NY

11:05 AM – 12:00 PM - Room 408 AB

Panelist

Rae Blaylark. Sickle Cell Foundation of Minnesota, MN

11:05 AM – 12:00 PM - Room 408 AB

Panelist

Joe Gleeson. , CA

8:00 AM – 12:00 PM - Room 408 AB

Co-Chair

Michelle Berg, BS. Aldevron, MN

8:00 AM – 12:00 PM - Room 408 AB

Co-Chair

Allison M.. Bradbury, PhD. Nationwide Children's Hospital; The Ohio State University Wexner Medical ...

8:01 AM – 8:35 AM - Room 408 AB

Enrolling a full clinical trial/Conventional Clinical Trial Design

Joe Gleeson, MD. Univ Calif, N-Lorem Foundation, CA

8:35 AM – 9:00 AM - Room 408 AB

Exploration Patient Specific Medicine: Ethical Perspective

Alison Bateman-House, PhD, MPH, MA. NYU Grossman School of Medicine, New York, NY

9:00 AM – 9:30 AM - Room 408 AB

Expanded Access Clinical Trials

Barry J.. Byrne, MD, PhD. University of Florida, Gainesville, FL

9:40 AM – 10:05 AM - Room 408 AB

Enrolling a Full Clinical Trial/Conventional Clinical Trial Design

Rae Blaylark. Sickle Cell Foundation of Minnesota, Minneapolis, MN

10:15 AM – 12:00 PM - Concourse Hall 150 & 151

Panelist

Sabrina D.. Poulos, J.D.. Goodwin Procter LLP, San Francisco, CA

10:15 AM – 12:00 PM - Concourse Hall 150 & 151

Panelist

Chris Garabedian. , MA

10:15 AM – 12:00 PM - Concourse Hall 150 & 151

Panelist

Melissa A. Kotterman, PhD. Iris Medicine, Berkeley, CA

10:15 AM – 12:00 PM - Concourse Hall 150 & 151

Panelist

Nicole K.. Paulk, PhD. University of California San Francisco, San Francisco, CA

10:15 AM – 12:00 PM - Concourse Hall 150 & 151

Panelist

Eric Kelsic. Dyno Therapeutics, Watertown, MA

8:00 AM – 12:00 PM - Concourse Hall 150 & 151

Co-Chair

Melissa A. Kotterman, PhD. Iris Medicine, Berkeley, CA

8:00 AM – 12:00 PM - Concourse Hall 150 & 151

Co-Chair

Nicole K.. Paulk, PhD. University of California San Francisco, San Francisco, CA

8:01 AM – 8:20 AM - Concourse Hall 150 & 151

IP/Contracts (getting legal representation, MTA, option/license)

Sabrina D.. Poulos, J.D.. Goodwin Procter LLP, San Francisco, CA

8:20 AM – 8:40 AM - Concourse Hall 150 & 151

Money (dilutive vs non-dilutive, angels, grants): Successful Operator-to-VC

Chris Garabedian. Xontogeny and Perceptive VC Firm, MA

8:40 AM – 9:00 AM - Concourse Hall 150 & 151

How to Vet if Your Idea is Worthy of Starting a Startup

Nicole K.. Paulk, PhD. University of California San Francisco, San Francisco, CA

9:00 AM – 9:20 AM - Concourse Hall 150 & 151

Defining Milestones for Program Development & Successful Fundraising

Melissa A.. Kotterman, PhD. Iris Medicine, Inc., Berkeley, CA

9:20 AM – 9:40 AM - Concourse Hall 150 & 151

Don’t worry, just do it: go ahead and create a successful biotech startup

Douglas Crawford, PhD. MBC BioLabs, CA

9:40 AM – 10:00 AM - Concourse Hall 150 & 151

Building the team that’s right for your startup

Eric Kelsic, PhD. Dyno Therapeutics, Watertown, MA

10:00 AM – 10:20 AM - Petree Hall D

Clinical Exploitation of SB Editing Platform

Zoltan Ivics, PhD. Paul Ehrlich Institut, Germany

10:20 AM – 10:40 AM - Petree Hall D

Base Editing for Therapeutic Applications

Holly Rees, PhD. , Cambridge, MA

10:40 AM – 11:00 AM - Petree Hall D

Base Editing CAR-T

Branden S. Moriarity, PhD. University of Minnesota, Minneapolis, MN

11:00 AM – 11:20 AM - Petree Hall D

Prime Editing

Julian Grünewald, MD. Technical University of Munich, Germany

11:20 AM – 11:45 AM - Petree Hall D

Panelist

Branden S. Moriarity. University of Minnesota, Minneapolis, MN

11:20 AM – 11:45 AM - Petree Hall D

Panelist

Zoltan Ivics. Paul-Ehrlich-Institut, Germany

11:20 AM – 11:45 AM - Petree Hall D

Panelist

Holly Rees, PhD. , Cambridge, MA

11:20 AM – 11:45 AM - Petree Hall D

Panelist

Julian Grünewald. , Germany

8:00 AM – 12:00 PM - Petree Hall D

Co-Chair

Alexis Komor, PhD. University of California, San Diego, La Jolla, CA

8:00 AM – 8:20 AM - Petree Hall D

Genome Editing Using TALENs

Alexandre Juillerat, PhD. Cellectis Inc, New York, NY

8:00 AM – 12:00 PM - Petree Hall D

Co-Chair

Claudio Mussolino, PhD. Institute for Transfusion Medicine and Gene Therapy at Center for Translatio...

8:20 AM – 8:40 AM - Petree Hall D

Harnessing the diversity of CRISPR-Cas proteins for genome editing

Giedrius Gasiunas, PhD. Caszyme, Lithuania

8:40 AM – 9:00 AM - Petree Hall D

Deploying ultracompact CRISPR systems for in vivo gene editing

Janice Chen, PhD. Mammoth Biosciences, CA

9:00 AM – 9:20 AM - Petree Hall D

Stem Cell Genome Editing to Treat Neurodegenerative Disorders

Natalia Gomez-Ospina, MD, PhD. Stanford University, Palo Alto, CA

9:20 AM – 9:45 AM - Petree Hall D

Panelist

Janice Chen. Mammoth Biosciences, Brisbane, CA

9:20 AM – 9:45 AM - Petree Hall D

Panelist

Natalia Gomez-Ospina. Stanford, Palo Alto, CA

9:20 AM – 9:45 AM - Petree Hall D

Panelist

Giedrius Gasiunas. Caszyme, Vilnius, Lithuania

9:20 AM – 9:45 AM - Petree Hall D

Panelist

Alexandre Juillerat, PhD. Cellectis Inc, New York, NY

10:15 AM – 10:30 AM - Room 515 AB

Panelist

Michael D.. Jain, MD, PhD. Moffitt Cancer Center, Tampa, FL

10:15 AM – 10:30 AM - Room 515 AB

Panelist

Yi Lin, M.D., Ph.D.. , MN

10:15 AM – 10:30 AM - Room 515 AB

Panelist

Sneha Ramakrishna, MD. Stanford University, Palo Alto, CA

10:45 AM – 11:05 AM - Room 515 AB

Beyond T Cells - Development of CAR-M

Michael Klichinsky, PhD. Carisma Therapeutics, PA

11:05 AM – 11:25 AM - Room 515 AB

CD 19 CAR T-cells for SLE

Samik Basu, MD. Cabaletta Bio - Philadelphia, PA, Philadelphia, PA

11:25 AM – 11:45 AM - Room 515 AB

Harnessing synthetic biology and gene editing technologies to engineer next generation CAR T cells

Justin Eyquem, PhD. UCSF, CA

11:45 AM – 12:00 PM - Room 515 AB

Panelist

Michael Klichinsky. University of Pennsylvania, Philadelphia, PA

11:45 AM – 12:00 PM - Room 515 AB

Panelist

Samik Basu, His/Him. Cabaletta Bio - Philadelphia, PA, Philadelphia, PA

11:45 AM – 12:00 PM - Room 515 AB

Panelist

Justin Eyquem, PhD. UCSF, CA

8:00 AM – 12:00 PM - Room 515 AB

Co-Chair

Daniel J.. Powell, Jr., PhD. University of Pennsylvania, Philadelphia, PA

8:00 AM – 12:00 PM - Room 515 AB

Co-Chair

Nirali Shah, MD, MHSc. National Cancer Institute, Bethesda, MD

8:01 AM – 8:20 AM - Room 515 AB

TILs in 2023: State of the Art and Future Directions

Stephanie Goff, MD. National Cancer Institute (NIH), MD

8:20 AM – 8:40 AM - Room 515 AB

TCR based strategies: State of the Art and Future Directions

Aude Chapuis, MD. Fred Hutchinson Cancer Research Center, Seattle, WA

8:40 AM – 9:00 AM - Room 515 AB

HSCT and Gene Editing to Optimize IEC Therapy

Saar Gill, MBBS, PhD, FRACP. University of Pennsylvania, Philadelphia, PA

9:00 AM – 9:15 AM - Room 515 AB

Panelist

Saar Gill, MBBS, PhD, FRACP. University of Pennsylvania, Philadelphia, PA

9:00 AM – 9:15 AM - Room 515 AB

Panelist

Aude Chapuis, MD. Fred Hutchinson Cancer Research Center, Seattle, WA

9:00 AM – 9:15 AM - Room 515 AB

Panelist

Stephanie L. Goff, MD. National Cancer Institute (NIH), Bethesda, MD

9:15 AM – 9:35 AM - Room 515 AB

GD2 Targeting and Midline Brainstem Tumors

Sneha Ramakrishna, MD. Stanford University, CA

9:35 AM – 9:55 AM - Room 515 AB

CD19-palooza: Summary of indications and factors impacting outcomes

Michael D.. Jain, MD, PhD. Moffitt Cancer Center, Tampa, FL

9:55 AM – 10:15 AM - Room 515 AB

BCMA CAR T-cells: Summary of Indications, Use and Outcomes in Multiple Myeloma

Yi Lin, M.D., Ph.D.. Mayo Clinic, MN

8:00 AM – 12:00 PM - Room 502 AB

Co-Chair

Angela S.. Whatley, PhD. PTC Therapeutics, Palo Alto, CA

8:00 AM – 12:00 PM - Room 502 AB

Co-Chair

Dan Leblanc. Ensoma, MA

8:01 AM – 8:25 AM - Room 502 AB

Introduction to the Issues: Common CMC Pitfalls and Best Practices

Jessica Chery, PhD. FDA CBER, MD

8:25 AM – 8:50 AM - Room 502 AB

CMC Case Study on Early Phase Development: Verve Therapeutics

Chris Cheng, PhD. Verve Therapeutics, MA

8:50 AM – 9:15 AM - Room 502 AB

CMC Case Study on Early Phase Development: Kite Pharmaceuticals

Alison Holzer-Speed. , CA

8:50 AM – 9:15 AM - Room 502 AB

CMC Case Study on Early Phase Development: Kite Pharmaceuticals

Jingying Xu, PhD. Kite Pharma, CA

9:15 AM – 9:40 AM - Room 502 AB

CMC Case Study on Later-Phase Development for Upstaza

Michael Chang. PTC Therapeutics, Inc., NJ

9:40 AM – 11:40 AM - Room 502 AB

Presenter Q&A + Attendee Breakout Discussions

Michael Chang. PTC Therapeutics, NJ

9:40 AM – 11:40 AM - Room 502 AB

Presenter Q&A + Attendee Breakout Discussions

Jessica Chery, PhD. FDA CBER, MD

9:40 AM – 11:40 AM - Room 502 AB

Presenter Q&A + Attendee Breakout Discussions

Chris Cheng, PhD. Verve Therapeutics, MA

01:30 PM – 01:45 PM - Concourse Hall 152 & 153

Identification of Host Factors Critical for rAAV2.5T Transduction of Polarized Human Airway Epithelia

Siyuan Hao{1}, Kang Ning{1}, Zehua Feng{2}, Cagla Aksu Kuz{1}, Ziying Yan{2}, Jianming Qiu{1} {1}Uni...

01:30 PM – 03:15 PM - Concourse Hall 152 & 153

Co-Chair

Elena E.. Balkanska-Sinclair. Ultragenyx Pharmaceutical Inc., Woburn, MA

01:45 PM – 02:00 PM - Concourse Hall 152 & 153

AAV2’s Defective Trafficking in the Absence of the AAV-Receptor is Due to a Block in Endosomal Escape

Marti Cabanes{1}, Sophia Liao{1}, Maddison Knight{1}, Renina Navarro{1}, Angus Johnston{2}, Leszek L...

02:00 PM – 02:15 PM - Concourse Hall 152 & 153

Modifying Immune Responses to Adeno-Associated Virus (AAV) Vectors by Capsid Engineering

Martin Bentler{1}, Romain Hardet{2}, Moritz Ertelt{3}, Dorota Kaniowska{4}, Daniela Rudolf{4}, Flori...

02:15 PM – 02:30 PM - Concourse Hall 152 & 153

Structural and Kinetic Characterization of Anti-AAV9 Monoclonal Antibodies Derived from Patients Post-Zolgensma Treatment

Jane Hsi{1}, Mario Mietzsch{1}, Austin R. Nelson{1}, Paul Chipman{1}, Jenny Jackson{2}, Peter Schofi...

02:30 PM – 02:45 PM - Concourse Hall 152 & 153

Co-Identification and Characterization of Host and Viral Protein Interactomes during AAV Transduction by Two Different Proximity Labeling Methods

Jisun Lee{1}, Guangping Gao{1,2}, Jun Xie{1,2} {1}Horae Gene Therapy Center, UMass Chan Medical Scho...

02:45 PM – 03:00 PM - Concourse Hall 152 & 153

Multiplexed Characterization of AAV Cellular Tropism and Promoter Expression in Large Animals Using Single-Cell RNA Sequencing

Alex Araki{1}, Martin Borch Jensen{1}, Chase Bowman{1}, Chris Carrico{1}, Linda Chio{1}, Atray Dixit...

03:00 PM – 03:15 PM - Concourse Hall 152 & 153

A New Class of AAVR Binders: Structure of a Goat Adeno-Associated Virus (AAVGo.1) Complexed with the Human Adeno-Associated Virus (AAVR) Receptor

Edward E. Large{1}, Mark Silveria{2}, Onellah Weerakoon{1}, Tommi White{3}, Michael S. Chapman{2} {1...

01:30 PM – 01:45 PM - Room 515 AB

Exploring the Robustness and Throughput of Size Exclusion Chromatography of Recombinant AAVs

Szabolcs Fekete{1}, Mateusz Imiolek{1}, Matthew Lauber{2} {1}Waters Corporation, Geneva, Switzerland...

01:30 PM – 03:15 PM - Room 515 AB

Co-Chair

Scott A.. Loiler, PhD. Apic Bio, Cambridge, MA

01:30 PM – 03:15 PM - Room 515 AB

Co-Chair

Nikitha Nimmagadda. St. Jude's Research Hospital, Memphis, TN

01:45 PM – 02:00 PM - Room 515 AB

Novel Repression System for Gene Expression Regulation during Recombinant Adeno-Assoiciated Virus and Lentiviral Vector Manufacturing

Jiali Yang{}, Liping Zhang{}, Jia Xu{}, Peimin Ma{}, Ruiju Yang{}, Guodong Jia{}, Weiran Shen{}, Xin...

02:00 PM – 02:15 PM - Room 515 AB

Ahead of the Game: A Scalable Purification AEX Platform Capable of Achieving <10% Empty Capsids

Ashish Sharma{}, Alex Meola{}, Kyla DeSanty{}, Alice Oliveira Aguiar{}, Thomas Thiers{}, Zaid Junaid...

02:15 PM – 02:30 PM - Room 515 AB

Impact of Downstream Buffer pH and Ionic Strength on rAAV Capsid Stability

Vijesh Kumar{}, Ohnmar Khanal{}, Mi JIn{} Technical Development, Downstream and Drug Product Develop...

02:30 PM – 02:45 PM - Room 515 AB

Challenging the Bind and Elute Convention: A Novel Approach to Partitioning Method for Full rAAV Capsid Enrichment Using AEX Chromatography

Tamara Zekovic{}, Paul Greback-Clarke{}, Jacob Smith{}, Jordan Hobbs{}, Robert Tikkanen{}, Eva Graha...

02:45 PM – 03:00 PM - Room 515 AB

Preserving High Transduction Efficiency - Serotype Agnostic Purification of Adeno-Associated Virus Using Adsorbents Functionalized with In Silco Discovered Peptide Ligand

Shriarjun Shastry{}, Stefano Menegatti{}, Wenning Chu{} Chemical and Biomolecular Engineering, North...

03:00 PM – 03:15 PM - Room 515 AB

IsoTag AAV - An Innovative, Scalable, Non-Chromatographic Method for Streamlined AAV Manufacturing

Michael Dzuricky{}, Kelli M. Luginbuhl{}, Jennifer Haley{}, Jarrett Dobbins{}, Christopher Ocker{}, ...

01:30 PM – 03:15 PM - Concourse Hall 150 & 151

Co-Chair

Saad Kenderian, M.D.. Mayo Clinic, Rochester, MN

01:30 PM – 03:15 PM - Concourse Hall 150 & 151

Co-Chair

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

01:31 PM – 01:55 PM - Concourse Hall 150 & 151

Review of Current Status of Tumor Immunologies and Obstacles to Tumor Microenvironment

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

01:55 PM – 02:20 PM - Concourse Hall 150 & 151

Armoring T-cells

Joseph A. Fraietta, PhD. University of Pennsylvania, PA

02:20 PM – 02:45 PM - Concourse Hall 150 & 151

In vivo macrophage engineering reshapes the tumor microenvironment leading to eradication of liver metastases

Mario Leonardo Squadrito, PhD. San Raffaele Telethon Institute for Gene Therapy, Italy

02:45 PM – 03:10 PM - Concourse Hall 150 & 151

Combinational Oncolytic Viruses

Paola Grandi, PhD, MBA. CG Oncology, Irivne, CA

01:30 PM – 03:15 PM - Room 502 AB

Co-Chair

David J. Segal, PhD. University of California, Davis, Davis, CA

01:30 PM – 03:15 PM - Room 502 AB

Co-Chair

Rachel Salzman, DVM. Armatus Bio, Delray Beach, FL

01:31 PM – 01:45 PM - Room 502 AB

Setting the Stage: Outlining the Ethical Dilemmas Involved in Pricing and Access

Rachel Salzman, DVM. Armatus Bio, Delray Beach, FL

01:45 PM – 02:00 PM - Room 502 AB

Pricing of and Access to Gene Therapies - Opportunities and Challenges

Sarah Emond, MPP. Institute for Clinical and Economical Review (ICER), Boston, MA

02:15 PM – 02:30 PM - Room 502 AB

The Promise and Challenges of Accessing Innovative High-cost Therapies: Angelman Syndrome

Allyson Berent, DVM, DACVIM. Foundation for Angelman Syndrome Therapeutics, New York, NY

02:30 PM – 02:45 PM - Room 502 AB

Assessing the American Health Care System: How Did We Get Here?

Aaron Seth Kesselheim, MD, JD. Harvard Medical School, MA

02:45 PM – 03:15 PM - Room 502 AB

Panel Q&A

Aaron Seth Kesselheim, MD, JD. Harvard Medical School, MA

02:45 PM – 03:15 PM - Room 502 AB

Panel Q&A

Sarah Emond, MPP. Institute for Clinical and Economical Review (ICER), Boston, MA

02:45 PM – 03:15 PM - Room 502 AB

Panel Q&A

Allyson Berent, DVM, DACVIM. Foundation for Angelman Syndrome Therapeutics, New York, NY

02:45 PM – 03:15 PM - Room 502 AB

Panel Q&A

Rachel Salzman, DVM. Armatus Bio, Delray Beach, FL

01:30 PM – 03:15 PM - Room 403 AB

Co-Chair

Donald B. Kohn, MD. Unversity of California, Los Angeles, Los Angeles, CA

01:30 PM – 03:15 PM - Room 403 AB

Co-Chair

Dan Manning, PharmD. Spark Therapeutics, PA