Cambridge Healthtech Institute’s Inaugural
Gene Therapy Bioproduction
Production, Quality Control and Analysis of Gene Therapies
Part of CHI's 8th Annual The Bioprocessing Summit
August 18-19, 2016 | Westin Boston Waterfront | Boston, MA

It’s exciting times for gene therapy – products on the market, IPOs and a long list of pharma collaborations. Pricing and reimbursement takes a majority of the headlines but equally important is finding a way to produce these therapies in a scalable, cost-effective and robust way, all the while developing a clear CMC and characterization profile that satisfies the regulators.

CHI’s Gene Therapy Bioproduction meeting takes a practical, case-study driven approach to the scale-up, quality control and analysis of gene therapies, tackling key topics such as AAV and lentivirus process development and scale-up, assay development and validation, and CMO management from early to late-stage development.

Final Agenda

Thursday, August 18

11:30 am Registration Opens

12:15 pm Lunch Available for Purchase in the Exhibit Hall

1:15 Dessert Refreshment Break in the Exhibit Hall with Poster Viewing

PROCESS DEVELOPMENT STRATEGIES FOR LENTIVIRUS-BASED PROCESSES

1:55 Chairperson’s Opening Remarks

Michael Kelly, Ph.D., Director, Asset Leadership, Gene Therapy, Biogen

2:45 Enabling Industrial Scale Production of Lentiviral Vectors for Gene Therapy

Kelly Kral, Ph.D., Senior Manager, Vector Process Development and Manufacturing, bluebird bio

Lentiviral vectors are an ideal platform for indications requiring long-term, stable expression, but the production processes have historically been limited by scale. This presentation will focus on the evaluation of platforms for the next-generation manufacturing process, with the guiding principles of preserving the comparability of the lentiviral product profile.

3:15 Discussion Panel: Gene Therapy Vector and Process Development

Panelists:

Dmitriy Lukashev, Ph.D., Principal Scientist, Novartis Institutes for BioMedical Research

Steven Howe, Ph.D., Head, Process Research, Cell & Gene Therapy Platform CMC, GSK

Kelly Kral, Ph.D., Senior Manager, Vector Process Development and Manufacturing, bluebird bio

Carol Knevelman, Ph.D., Senior Manager, Process R&D Department, Oxford Biomedica Ltd.

3:45 Refreshment Break

MANUFACTURING, COMPARABILITY & TECH TRANSFER

4:15 Lentiviral Vector Manufacturing Strategies to Support Cell and Gene Therapies

Carol Knevelman, Ph.D., Senior Manager, Process R&D Department, Oxford Biomedica Ltd.

Successful development and commercialization of gene and cell-based therapies is highly dependent on establishing robust and efficient cGMP manufacturing processes that meet the key challenges of scale, quality, cost of goods supplied (COGS), and sustainability. This talk will discuss new manufacturing modalities that are being developed for cGMP production of these vectors.

4:45 Demonstrating Comparability During Gene Therapy Tech Transfer

Scott Burger, M.D., Consultant, Advanced Cell and Gene Therapy Ltd.

Changing the manufacturing site (tech transfer) should always include an assessment of comparability, however the ability to demonstrate this varies between early and late development. This talk will discuss common pitfalls and mistakes and highlight key aspects of the comparability exercise.

5:15 End of Day

5:15 Registration for Dinner Short Course

Friday, August 19

8:00 am Registration Opens and Morning Coffee

CMC, QUALITY & POTENCY ASSAY DEVELOPMENT

8:25 Chairperson’s Remarks

Scott Burger, M.D., Consultant, Advanced Cell and Gene Therapy Ltd

8:30 Gene Therapies for All: How to Approach Industrialization of Gene Therapies on a Large Scale

Ben Locwin, Ph.D., MBA, MS, President, Healthcare Science Advisors

While precision medicine, personalized medicine, and gene therapies represent the future of targeted therapies for improved disease management and resolution, there are challenges of scale that must be faced in order to make GT viable for society. In this engaging discussion about the future of GT, best practices will be shown to increase the likelihood that gene therapies can successfully represent the next generation of medicine. Concepts covered will include Quality Risk Management, Quality by Design, CPPs, and CQAs.

9:00 Analytics for AAV Gene Therapy, Strategies for Phase I

Genine Winslow, Ph.D., Director of Research Analytics, Audentes Therapeutics, Inc.

Using case studies this presenation will look at an overview of CMC requirements for phase 1 CTA/IND applications, AAV CMC analytics challenges with emphasis on achieving comparable vector genome titers across sites, and a brief discussion of potency assays. The talk will also look at strategies for successful coordination of AAV analytics with CMOs.

9:30 Developing a Potency Assay Matrix for an AAV Gene Therapy Product

Barb Thorne, Ph.D., Consultant, Thorne Bio-Consulting LLC, Former Executive Director Process Development, Celladon

A case study will be presented on the potency matrix for a gene therapy product formerly under development, AAV1/SERCA2a. The approach was based on FDA Guidance on Potency Tests for Cellular and Gene Therapy Products, and the matrix included three complementary quantitative assays used for product lot release and stability testing as well as a challenging qualitative enzyme activity assay.

10:00 Networking Coffee Break

PROCESS DEVELOPMENT FOR AAV-BASED PROCESSES

10:30 A Commercially Viable Novel CMC Approach to Bioprocessing AAV for Rare Diseases

Reed Clark, SVP Mfg, Dimension Therapeutics

This presentation will look at creating a continuum for both upstream and downstream, viewing large scale manufacture from the very beginning and the main challneges associated with cost and scale-up.

11:00 Presentation to be Announced

11:30 From Initial Phase I Supply with CRO to Phase III CMO Supply

Jean-Philippe Combal, Ph.D., COO, GenSight Biologics, France

Jolyn Johnson, Ph.D., Upstream Processing Expert, Novasep

More rigorous product characterization, process development and regulatory hurdles for phase 3 or even commercial batches require highly skilled and critical transfer. Challenges and anticipation from rights to know-how transfer and regulatory impact requires significant perspectives to ensure large-scale cGMP and regulatory compliance. Viewpoint from both biotech and cGMP will be discussed.

12:00 pm Panel Discussion: Best Practice for Effective Gene Therapy CMO Transfer and Management with External Partners

Jolyn Johnson, Ph.D., Upstream Processing Expert, Novasep

Jean-Philippe Combal, Ph.D., COO, GenSight Biologics, France

12:30 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own

1:15 Session Break

PROCESS DEVELOPMENT & SCALE-UP STRATEGIES

1:25 Chairperson’s Remarks

Scott Burger, M.D., Consultant, Advanced Cell and Gene Therapy

1:30 rAAV Vector Production in the Baculovirus/Sf9 platform and in HEK293 Suspension Cell System

David Dismuke, Director, Vector Production, Voyager Therapetuics

2:00 Strategies to Deliver Scalable and Reliable Lentiviral Vector Biomanufacturing

Jeffrey Bartlett, Ph.D., Senior Vice President, R&D, Calimmune

One important consideration in designing processes for the clinical production of lentiviral vectors is minimizing lot-to-lot variation. With a mind towards developing a scalable, reliable, cGMP-compliant biomanufacturing process for lentiviral vectors we have established the Cytegrity stable cell line system and have defined key regulatory parameters to facilitate cGMP production and regulatory approval using this system.

2:30 Manufacturing Models for Commercial-Scale Gene Therapy

Scott Burger, M.D., Consultant, Advanced Cell and Gene Therapy

3:00 Panel Discussion: Commercializing Gene Therapies

Panelists:

David Dismuke, Director, Vector Production, Voyager Therapetuics

Jeffrey Bartlett, Ph.D., Senior Vice President, R&D, Calimmune

Topics to be covered include:

• Pricing - from process to reimbursement

• Scaling up - unit operations, platforms, analytics

• Gene therapy supply - from rare disease to larger indications

• Facility design

3:30 Close of Conference