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.

Wednesday, September 2

CLINICAL DEVELOPMENT & STANDARDIZATION OF GENE THERAPY

9:05 am KEYNOTE PRESENTATION:

Clinical Development of Gene Therapies

Mike Singer, MD, PhD, Medical Officer, Division of Clinical Evaluation & Pharmacology, CBER, 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.

9:25 am

An International Collaboration: Towards the Standardisation of Gene Therapy

Yuan Zhao, PhD, Principal Scientist & Leader & Section Head, Gene Therapy, NIBSC

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.

Adam Hejmowski, Research Scientist, Research and Development, Pall Biotech

We have investigated the performance of membrane chromatography as a polishing step for purification of AAV5. We compare the performance for contaminant removal compared to commonly used resin based approaches to show the potential of membrane chromatography as a platform-able polishing step for AAV purification. Membrane chromatography can be loaded 40 times more quickly than conventional chromatography resins, which can lead to much higher productivities and greatly speed the purification step

10:10 am LIVE Q&A:

Q&A and Session Wrap-Up

Panel Moderator:
Yuan Zhao, PhD, Principal Scientist & Leader & Section Head, Gene Therapy, NIBSC
Panelists:
Mike Singer, MD, PhD, Medical Officer, Division of Clinical Evaluation & Pharmacology, CBER, FDA
Adam Hejmowski, Research Scientist, Research and Development, Pall Biotech
10:30 am Speed Networking Coffee Break - View our Virtual Exhibit Hall

COMMERCIALIZATION STRATEGIES AND CHALLENGES BEYOND RARE DISEASES

10:55 am

Taking Gene Therapy Beyond Rare Diseases

Alexis 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.

11:15 am

Strategies for the Commercialization of Gene-Modified Cell Therapies

Knut Niss, PhD, CTO, Mustang Bio, Inc.

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.

HARNESSING CRISPR FOR GENE THERAPIES

11:35 am

Harnessing Novel CRISPR Systems for Genome Engineering

Jonathan S Gootenberg, PhD, McGovern Fellow/Principal Investigator, McGovern Institute, Massachusetts Institute of Technology

RNA plays important and diverse roles in biology, but molecular tools to manipulate and measure RNA are limited. We demonstrate that RNA-targeting CRISPR effector, Cas13, can be engineered for mammalian cell RNA knockdown, binding, and RNA editing. Cas13 can be heterologously expressed in mammalian and plant cells for targeted knockdown of either reporter or endogenous transcripts, targeted RNA binding for transcript imaging, and programmable RNA editing. We also show that both Cas13 and Cas12 can be used for sensitive nucleic acid diagnostics and show that CRISPR diagnostics (CRISPR-dx) can be used for point-of-care COVID-19 detection. Our results establish CRISPR-Cas13 as a flexible platform for RNA targeting with wide applicability for studying RNA, diagnostics, and therapeutics.

Omar Abudayyeh, PhD, McGovern Fellow/Principal Investigator, Massachusetts Institute of Technology
12:00 pm LIVE Q&A:

Session Wrap-Up

Panel Moderator:
Knut Niss, PhD, CTO, Mustang Bio, Inc.
Panelists:
Alexis Bloom, PhD, VP, Regulatory Affairs and Quality Assurance, Gyroscope Therapeutics
Jonathan S Gootenberg, PhD, McGovern Fellow/Principal Investigator, McGovern Institute, Massachusetts Institute of Technology
Omar Abudayyeh, PhD, McGovern Fellow/Principal Investigator, Massachusetts Institute of Technology
12:20 pm Lunch Break - View our Virtual Exhibit Hall
1:15 pm Refresh Break - View our Virtual Exhibit Hall

VECTOR DESIGN, DELIVERY AND CHARACTERIZATION

2:25 pm

AI-Powered Design of Synthetic AAV Capsids 

Eric Kelsic, PhD, CEO & Co-Founder, Dyno Therapeutics

Pre-existing immunity is a major challenge for AAV gene therapy, preventing many patients from being treated by therapies under development today. Artificial intelligence (AI) presents new opportunities for overcoming such challenges, especially when applied to the design of synthetic AAV capsids. This approach combines three advanced technologies: i) next-gen library synthesis; ii) measurement via next-gen sequencing; and iii) library analysis powered by machine learning. Such a workflow can dramatically accelerate the search for synthetic capsids with superior targeting and immunological profiles relative to capsids derived from natural AAV isolates. I will review the technological advances that are pushing the field of AAV capsid engineering toward AI-powered methods, describe and explore the promise of this approach, and discuss anticipated challenges. In the near future, AI will revolutionize our ability to design safe, targeted, and robust delivery vectors for the treatment of many new genetic conditions, and importantly, enable all patients to benefit from such therapies.

2:45 pm

DNA-Based Nanobody Delivery

Carlo Boutton, PhD, Head, Nanobody Explorative Technologies, Ablynx NV, 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.

Jonathan 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.  

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

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.

3:30 pm LIVE Q&A:

Q&A and Session Wrap-Up

Panel Moderator:
Eric Kelsic, PhD, CEO & Co-Founder, Dyno Therapeutics
Panelists:
Carlo Boutton, PhD, Head, Nanobody Explorative Technologies, Ablynx NV, a Sanofi Company
Jonathan Mehtala, Field Application Scientist, Malvern Panalytical
Michael Tovey, PhD, Managing Director, R&D, Svar Life Science
3:50 pm Refresh Break - View Our Virtual Exhibit Hall
4:10 pm Problem Solving Breakout Discussions - Part A

This session provides the opportunity to discuss a focused topic with peers from around the world in an open, collegial setting. Select from the list of topics available and join the moderated discussion to share ideas, gain insights, establish collaborations or commiserate about persistent challenges.

TABLE 29: CQA Identification and Assessment in Gene Therapy Products

Lin Liu, PhD, CMC Dossier Development & Coordination, 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

 

4:40 pm Refresh Break - View Our Virtual Exhibit Hall
5:00 pm Problem Solving Breakout Discussions - Part B
5:30 pm Close of Day

Thursday, September 3

GENE THERAPY IN CANCER AND NEUROLOGICAL DISEASES

9:00 am

Translating Preclinical Responses to Clinical Relevance: Challenges of Gene Therapy

Carl A Morris, PhD, CSO, Solid Biosciences
9:25 am

Gene and Enzyme Suppression in Cancer Therapy

Brenda Laster, PhD, Prof Nuclear Engineering & Dir, Jerry J Cohen Radiobiology Lab, Ben Gurion Univ of the Negev

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.

9:45 am

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

Asa Hatami, PhD, Scientist II, Molecular Biology and Biochemistry, Sangamo Therapeutics

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.

10:05 am

Treatment of Neurodegenerative Diseases by Genome Editing Technologies

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

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.

10:30 am Coffee Break - View our Virtual Exhibit Hall
10:50 am LIVE Q&A:

Q&A and Session Wrap-Up

Panel Moderators:
Brenda Laster, PhD, Prof Nuclear Engineering & Dir, Jerry J Cohen Radiobiology Lab, Ben Gurion Univ of the Negev
Thomas Gaj, PhD, Assistant Professor, Bioengineering, University of Illinois Urbana Champaign
Panelists:
Carl A Morris, PhD, CSO, Solid Biosciences
Asa Hatami, PhD, Scientist II, Molecular Biology and Biochemistry, Sangamo Therapeutics
11:20 am Close of Gene Therapy





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