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


Recommended Short Course*

SC12: Emerging Applications for Gene Editing, Base Editing and RNA Editing

*Separate registration required.


7:15 am Registration and Morning Coffee

7:25 Women in Science Panel Discussion with Continental Breakfast

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

Nora MinevaNora Mineva, PhD, CSO, Adecto Pharmaceuticals

Liu_TinaTina Liu, MBA, Co-Founder and CEO, Ally Therapeutics

Chadwich_JenniferJennifer 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, Vice President, Regulatory Affairs and Quality Assurance, RA and QA, 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 Measure AAV Quality Attributes with SEC-UV-MALS-dRI

John Champage, Northeast Regional Manager, Senior Applications Scientist, Wyatt Technology

Adeno-associated virus (AAV) is an attractive delivery vehicle in gene therapy attributed to its mild immune response and ability to deliver its genome into a wide range of host cells. In this presentation, we will discuss a method to measure the following three important AAV quality attributes (QAs): 1) total number of viral capsid particles; 2) relative capsid content (e.g., ratio of empty and full capsids); and 3) percentage of monomer or aggregates.

1:05 Session Break

1:10 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

2:10 Session Break


2:25 Chairperson’s Remarks

Juan Hernandez Bort, PhD, Head, Gene Therapy Technologies, Takeda Austria

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 Toward a Multi-Use Platform for the Production of Different Recombinant Adeno-Associated Virus Serotypes

Juan Hernandez Bort, PhD, Head, Gene Therapy Technologies, Takeda Austria

Recombinant adeno-associated viruses (rAAV) have become the vehicle of choice for many clinical studies and they are one of the preferred viral vectors for in vivo gene therapy applications. Nevertheless, achieving high titers while reducing product losses and impurities remains a challenge when different serotypes are produced, upon the same platform, due to innate biophysical-specific characteristics between some rAAV serotypes. In this talk, innovative approaches to develop a scalable and robust rAAV platform for different serotypes will be discussed.

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 Talk Title to be Announced 

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

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: Cell and Gene Therapy Development in Europe: Navigating the Regulatory Framework

Moderator: Alex Bloom, PhD, Vice President, Regulatory Affairs and Quality Assurance, Gyroscope Therapeutics

  • Clinical trial and GMO applications in the EU
  • Opportunities for accelerating ATMP development
  • Interacting with regulators in the EU and optimising scientific advice

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


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).

ProteinSimple9:35 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

Recommended Short Course*

SC15: Introduction to Gene Therapy Products Manufacturing and Analytics

*Separate registration required.

* The program is subject to change without notice, due to unforeseen reason.

Choose your language
Traditional Chinese
Simplified Chinese

Update History
Agenda,Sponsor updated
Agenda,Speaker,Sponsor updated
Agenda,Sponsor updated
Event Information updated
Sponsor updated

View By: