Original Agenda
We are actively working with our speakers to confirm their availability for the virtual event. Initial response from our speakers has been very positive, and we are optimistic we will have the new programs ready to share here soon.

Gene Therapy

Gene therapy, an experimental technique that uses genes to treat or prevent diseases, is experiencing a surge in interest and R&D investment. To date, there are over 2600 trials in 38 countries, with the majority of them in Phase I/II trials. With the heightened interest and the push to move quickly from bench to clinic, it is imperative for companies to have a better understanding of the implications of regulatory, commercialization, preclinical and clinical strategies on their gene therapies.

Meet the industry forerunners at CHI's inaugural Gene Therapy conference to gain insights into their strategies and program development.

Final Agenda

TUESDAY, september 1

Recommended Short Course*

SC10: CAR T Cell Therapy from A-Z - Detailed Agenda

*Separate registration required.

WEDNESDAY, september 2

7:15 am Registration and Morning Coffee

7:25 Women in Science Panel Discussion with Continental Breakfast - Learn More

Lucie Rochard, PhD, Liaison, Scientific & Entrepreneurial Initiatives; Director, Innovation Services, Massachusetts Biotechnology Council


Denise Aronson, Founder and CEO, Safety Partners, Inc.

Nora Mineva, PhD, CSO, Adecto Pharmaceuticals

Jennifer Chadwich, PhD, Vice President, Biologic Development, BioAnalytix, Inc.


8:40 Chairperson’s Opening Remarks

Knut Niss, PhD, CTO, Mustang Bio

8:50 KEYNOTE PRESENTATION: Clinical Development of Gene Therapies

Mike Singer, MD, PhD, Medical Officer, Division of Clinical Evaluation and Pharmacology/Toxicology, Center for Biologics Evaluation and Research, FDA

The exciting and rapidly-developing field of gene therapy poses unique challenges for FDA in facilitating the timely development and approval of new treatments that are both safe and effective. Clinical development programs for gene therapies differ in some important respects from those used for small molecule drugs. This presentation will discuss these challenges, and the approaches FDA uses to address them.

9:20 Translating Preclinical Responses to Clinical Relevance: Challenges of Gene Therapy

Carl Morris, PhD, CSO, Solid Biosciences

9:50 An International Collaboration: Towards the Standardisation of Gene Therapy

Yuan Zhao, PhD, Principal Scientist, Leader of Gene Therapy Section, Advanced Therapy Division/NIBSC, Medicines & Healthcare Products Regulatory Agency

Manufacturing hurdles, including changes in production sites and manufacturing processes, pose challenges to developers regarding reproducibility and comparability of results for gene therapy. Introduction of an international standard for gene therapy is especially important, given the usually orphan nature of the diseases to be treated, hampering the comparison of cross-trial and cross-manufacturing results. This presentation will discuss challenges and regulatory perspectives in quality control and standardization of gene therapy and an international effort in developing the 1st WHO International Standard for gene therapy products.

10:20 Coffee Break in the Exhibit Hall with Poster Viewing

10:30 Women in Science Speed Networking in the Exhibit Hall


11:05 Strategies for the Commercialization of Gene-Modified Cell Therapies

Knut Niss, PhD, CTO, Mustang Bio

This presentation will discuss several strategies to commercialize cell and gene therapy products. Particular attention will be given to the manufacturing of these products and how early planning can increase the speed to market and enable lower COGS. Both allogeneic and autologous therapies as well as gene therapy strategies will be discussed.

11:35 Evidence Generation and Principles for Articulating Value in Order to Achieve Patient Access for Gene Therapies

Phillip D. Carter, Head, U.S. Market Access, Orchard Therapeutics

12:05 pm Taking Gene Therapy Beyond Rare Diseases

Alex Bloom, PhD, VP, Regulatory Affairs and Quality Assurance, Gyroscope Therapeutics

To-date, gene therapy development has focused on orphan conditions. This talk will look at both the challenges and opportunities associated with moving gene therapies to large, non-orphan diseases.

12:35 Sponsored Presentation (Opportunity Available

1:05 Session Break

UnchainedLabs 1:10 Luncheon Presentation I to be Announced

1:40 Luncheon Presentation II (Sponsorship Opportunity Available)

2:10 Session Break


2:25 Chairperson’s Remarks

Chairperson to be Announced

2:30 DNA-Based Nanobody Delivery

Carlo Boutton, PhD, Head, Nanobody Explorative Technologies, Ablynx, a Sanofi company

Small Nanobodies® with their modular design show a different pharmacokinetic profile compared to conventional antibodies. This difference in exposure can be exploited by alternative delivery methods. DNA-based gene transfer of Nanobodies and biopharmaceuticals in general is an appealing alternative to conventional protein therapy. We demonstrate that multivalent Nanobodies encoded as DNA results in a stronger, longer-term and more localized exposure compared to conventional protein therapy.

3:00 Harnessing Novel CRISPR Systems for Genome Engineering

Jonathan Gootenberg, PhD, McGovern Fellow/Principal Investigator, McGovern Institute, Massachusetts Institute of Technology and Omar Abudayyeh, PhD, McGovern Fellow/Principal Investigator, McGovern Institute, Massachusetts Institute of Technology

3:30 The Zetasizer Ultra – A Novel Assay for Measuring Adeno Associated Virus (AAV) Particle Concentration Using MADLS

Johnathan Mehtala, Field Application Scientist, Malvern Panalytical

The high-resolution size capabilities of the Zetasizer Ultra have enabled a new assay to rapidly characterize (AAV) viral concentration. Utilizing (MADLS), Zetasizer Ultra can determine both AAV viral concentration and size in a single measurement that is cuvette-based, rapid, label-free, low volume, and non-destructive.

SVAR 3:45 Quantification of the Immune Response to AAV Mediated Gene Therapy

Michael Tovey, PhD, Managing Director, R&D, Svar Life Sciences

A highly sensitive iLite® reporter assay based on the establishment of a cell line stably transfected with a luciferase reporter-gene placed under the control of an AAV-responsive chimeric promoter, provides a highly sensitive, rapid and precise method for quantifying the immune response to a wide range of recombinant AAV vectors.

4:00 Refreshment Break in the Exhibit Hall with Poster Viewing

5:00 Problem-Solving Breakout Discussions - View All Breakout Discussion Topics

TABLE: Challenges During Gene Therapy Development for Neurological Diseases

Moderator: Gabriele Proetzel, PhD, Director, External Neuroscience Innovation, Neuroscience Drug Discovery Unit, Takeda Pharmaceuticals

  • Choice of animal models
  • Usefulness of reporter gene biodistribution studies
  • AAV capsid choices
  • Biopotency assay development

    TABLE: CQA Identification and Assessment in Gene Therapy Products

    Moderator: Lin Liu, PhD, Principal Scientist, Biologics Development, Sanofi

    • Identification of pCQAs for AAV based gene therapy products
    • Design structure-functional study specific to gene therapy CQA assessment
    • Role of forced degradation study in CQA assessment
    • Potency assay used for structure-function studies

    6:00 Taste of New England Networking Reception in the Exhibit Hall with Poster Viewing

    7:15 End of Day

    THURSDAY, september 3

    8:00 am Registration and Morning Coffee


    8:30 Chairperson’s Remarks

    Thomas Raj, PhD, Assistant Professor, Bioengineering, University of Illinois

    8:35 Gene and Enzyme Suppression in Cancer Therapy

    Brenda Laster, PhD, Professor, Nuclear Engineering, Ben-Gurion University

    We developed an anticancer drug and implanted it directly into solid tumors, suppressing the oncogenes and genes responsible for activation of telomerase. The drug is continuously available in the DNA promoter regions for long periods of time to prevent overexpression and reactivation. In 4 separate experiments, we implanted the drug, using, an intratumoral sustained polymeric system, directly into 100 mouse tumors, reducing the tumor volume 5-fold. Kaplan-Meier p-value was 0.0001.

    9:05 Therapeutic Applications of CRISPR/Cas9 in Rare Diseases**

    Seokjoong Kim, PhD, Executive Director, R&D Strategy and Strategic Alliances, ToolGen

    CRISPR/Cas9 is a disruptive genome editing tool for innovative gene therapies. In this talk, we will present our therapeutic genome editing strategies for Charcot-marie-tooth disease (CMT) and (likely) hemophilia. All programs are in animal-based efficacy validation steps (mid-to-late discovery).


    9:35 Presentation to be Announced


    10:05 Coffee Break in the Exhibit Hall with Poster Viewing and Poster Award


    11:05 Gene Therapy Development for Neurological Diseases

    Gabriele Proetzel, PhD, Director, External Neuroscience Innovation, Neuroscience Drug Discovery Unit, Takeda Pharmaceuticals

    With the approval of Zolgensma, gene therapy for neurological disorders have become a reality. This presentation will discuss the recent advances for in vivo gene therapy development for the neuronal diseases with their challenges and opportunities. The focus will be on adeno-associated virus (AAV), key aspects for preclinical development and how this is different from classical drug discovery. Discussed will be payload options, delivery and preclinical models critical for advancing gene therapy into the clinic and to the patient.

    11:35 Treatment of Neurodegenerative Diseases by Genome Editing Technologies

    Thomas Gaj, PhD, Assistant Professor, Bioengineering, University of Illinois

    Neurodegenerative disorders are the leading cause of disability in the aging population and predicted to soon surpass cancer and become the second leading cause of death worldwide. Our group aims to harness the highly versatile genome-modifying technologies to develop corrective gene therapies for such neurodegenerative conditions. This talk will highlight our most recent efforts on using recently emerged precision gene-editing tools to treat amyotrophic lateral sclerosis and Huntington’s disease, two devastating and relentlessly progressing neurodegenerative disorders.

    12:05 pm Targeted Gene Repression Using Engineered Zinc Finger Protein Transcription Factors as a Novel Therapeutic for Neurodegenerative Disorders

    Asa Hatami, PhD, Scientist II, Sangamo

    Aggregation of misfolded proteins into toxic species is implicated in the pathogenesis of many neurodegenerative proteinopathies, including Alzheimer’s disease, Parkinson’s disease, and prion disease. Lowering pathogenic protein expression at the DNA level using zinc finger protein transcription factors (ZFP-TFs) has the potential to slow or stop disease progression. We present data from iPSC-derived human neurons, rodents, and nonhuman primates demonstrating durable, highly specific targeted gene regulation following a single ZFP-TF administration.

    12:35 End of Gene Therapy


    **Presentations delivered via a live, interactive video conference platform.**


    Recommended Short Course*

    SC15: Introduction to Gene Therapy Products Manufacturing and Analytics - Detailed Agenda

    *Separate registration required.

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