ADVANCED THERAPIES XCHANGE
EAST COAST
Boston
April 3, 2023

Welcome to hubXchange’s Advanced Therapies Xchange 2023, East Coast, bringing together executives from pharma and biotech to address and find solutions to the key issues faced in cell and gene therapeutics.

Discussion topics will cover Cell Tx Development, Gene Tx Development, C&G Tx Bioprocessing and C&G Tx Manufacturing.

Take advantage of this unique highly interactive meeting format designed for maximum engagement, collaboration and networking with your peers.

Venue Details: Hilton Boston Woburn Hotel, 2 Forbes Road, Woburn MA 01801

SNAPSHOTS OF DISCUSSION TOPICS

Track 1: Cell Tx Development

Enhancing CAR-T cell efficacy
Alternatives to autologous- allogenic and in vivo cell therapies
The promise and challenges of PSC-CAR-NK cell therapy

Track 2: Gene Tx Development

Current landscape of non-viral vector technologies
Selecting the optimal route of administration and AVV serotype for neurological applications
Strategy, technology, and challenges to meeting speed and quality requirements during early phase development

Track 3: C&G Tx Bioprocessing

Improving the upstream GT bioprocessing

Track 4: C&G Tx Manufacturing

Future of autologous cell therapy
Tackling the challenges of AAV vector manufacturing

Full Xchange Agenda

Click on each track for detailed agenda

Cell Tx Development

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation
Applying principles of the 4th Industrial Revolution to accelerate novel therapeutic development

Digitalize data and meta dat
Digitalization of “Work” “Flow”
Integrate & streamline operations from research to development to manufacturing; and real-world data collection

Vasu Rangadass

President & Chief Executive Officer, L7 Informatics

Vasu Rangadass, Ph.D., President and CEO at L7 Informatics, Inc., is a leader in enterprise-scale digital transformation and automation of scientific processes. Previously, Dr. Rangadass was the Chief Strategy Officer at NantHealth following its acquisition of Net.Orange, the company he founded to provide an enterprise-wide platform to simplify and optimize care delivery processes in health systems. Prior to Net.Orange, Vasu was the first employee of i2 Technologies (currently Blue Yonder), which later became a global company that revolutionized the supply chain market through innovative approaches based on Six-Sigma principles, operations research, and process optimization.

09:05 – 10:05

Enhancing CAR-T cell efficacy

Next generation approaches for cell therapy manufacturing focused on improving response
Better CAR: Current approaches to CAR design, construction and T cell engineering
Multidimensional analyses that create insights CAR T cell attributes associated with improved response
Approaches to data analyses: can product understanding help identify opportunities to identify manufacturing improvements?

Max Tejada

Executive Director of Analytical Development, Kite Pharma (Giliead)

Max Tejada is a scientist with 20+ years of experience at Genentech, Gilead, AstraZeneca and Kite where he has led teams focused on the development, validation, and transfer of bioassay or immunoassays for therapeutic proteins, monoclonal antibodies, antibody drug conjugates and cell-based cancer immunotherapies. He has led analytical and Chemistry Manufacturing and Controls (CMC) teams for investigational oncology and immunotherapy programs and has been a contributing author on numerous regulatory filings in both early and late clinical phases. Max obtained his Ph.D. in Biochemistry from Queen’s University in Kingston, Ontario, Canada. He was a post-doctoral fellow in the laboratory of Dr. Napoleone Ferrara at Genentech.

10:10 – 10:40

1-2-1 Meetings/Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

11:20 – 12:20

Beyond development and into the clinic: how do we mitigate the impacts of the narrow cell viability window to make cell therapies viable at scale, as treatments in hospitals across the world?

How to improve the viability of cell therapies post-cryopreservation
Important considerations for extending cell viability at room temperature – during transport, and importantly, in the clinic
How may the quality of excipients affect the performance of the final cell therapy product?

Brian MacDonald

Head of US Business Development, Albumedix

Brian is a passionate business development expert, with significant experience in identifying and facilitating collaborations in the life science space. Supported by a near four-decade portfolio of experience in recombinant albumin at Albumedix, Brian works with customers developing advanced therapies to support solution finding that gets their advanced therapies from the lab, into production, and deployment.

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation
Financial and other considerations for early phase cell and gene therapy manufacturing: Build, Buy or Blend?

This talk aims to compare the various options for early phase clinical manufacturing for cell and gene therapies. The traditional option of Build (internalizing manufacturing) and the factors to consider for facility build out, cGMP readiness and routine operations will be discussed, specific to cell and gene therapy operations. The factors and risks to consider with the traditional option of Buy (outsourcing to CDMO) will be discussed. The Blend approach via Azzur Cleanrooms on DemandTM and the acceleration advantages will be discussed. A financial comparison with a model mRNA therapy process/facility for all three options will be included in this presentation.

Ravi Samavedam

Chief Innovation Officer, Azzur Group

Ravi Samavedam is a Biochemical Engineer with more than 20 years of experience in the pharmaceutical and biotechnology industries. Ravi joined Azzur in 2012, and prior to his appointment as CINO of Azzur Group, Ravi was the President and COO of Azzur Cleanrooms on Demand™ and previously served as General Manager of the Boston-area consulting practice. Prior to Azzur, Ravi has been part of the technical operations and quality/validation departments at Baxter, Amgen, and Shire.

Ravi is an expert in phase-appropriate cGMP implementation, process validation, supplier management, and technology transfer. Azzur Cleanrooms on Demand™ offers flexible early-phase cGMP manufacturing options designed to mitigate risk and increase speed to clinic. In addition to his work with Azzur, Ravi is an active member of industry and peer groups, and he is a frequent speaker at industry events.

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:35 – 16:35

Alternatives to autologous-allogenic and in vivo cell therapies

Key challenges of autologous CAR-T cell therapy (in vitro activation and production, target selection, solid tumors, etc)?
Challenges and opportunity for allogeneic CAR-T cell therapy
Targeting tumor heterogeneity by modular CAR-T technology
T cell sources (PBMC, UCB, iPSC)
Could other types of cell therapies, CAR-NK, CAR-Macrophage, be a good alternative solution for CAR-T (better safety profile, less GvHD, no MHC, TCR)?

Qingcong Lin

Chief Executive Officer, Biocytogen Boston

Qingcong Lin is SVP of Biocytogen and CEO of Biocytogen Boston Corporation, focusing on Biocytogen global business development on antibody discovery, RenMab/RenLite licensing and therapeutic antibody co-development, in addition to providing services on gene-targeting and humanized mouse models, using cutting-edge ESC and CRISPR-Cas9 technology and preclinical pharmacology, especially antibody in vivo efficacy and toxicity assessment using immune-check point target, cytokine, cytokine receptor, and TAA-humanized mice. Prior to joining Biocytogen, Dr. Lin was SVP of Shenogen Pharma Group. Dr. Lin has extensive expertise in multiple therapeutic areas, including cancer, CVMD, neurology, immunology & inflammation and various technologies, including genetically engineered mouse models, therapeutic antibody engineering, affinity maturation and optimization. Prior to joining Sheongen, Dr. Lin worked at Pfizer as a principal research scientist II and group leader of the antibody engineering group. Before joining Pfizer, he worked at Wyeth and Harvard Medical School as Director of Gene Modification Lab of Harvard-Partners Center for Genetics and Genomics. Dr. Lin received Ph.D. from Albert-Einstein College of Medicine and completed his postdoctoral training at Harvard Medical School.

16:35 – 16:45

Afternoon Refreshments

16:45 – 17:45

The promise and challenges of PSC-CAR-NK cell therapy

Promise: CAR-NK cells from pluripotent stem cells (PSC) will potentially provide an inexhaustible cell source of allogenic/off-the-shelf therapeutics for all patients worldwide.
Challenges: Tumorigenic potential from undifferentiated PSCs, CMC and manufacture; and limited sustainability in vivo.
Discussion points: Differentiation process and QA/QC; scalable manufacture; and gene editing strategy

John Lu

President and CEO, HebeCell

John Lu, PhD, MPH, is the President and CEO of HebeCell Corporation, focusing on the development and pluripotent stem cell (PSC)-CAR-NK cells for the treatment of human diseases. Before establishing HebeCell, he was the Senior Director of Research at Advanced Cell Technology/Ocata Therapeutics for 11 years. John has more than 20 years of experience in stem cell biology and regenerative medicine as well as >10 year experiences in cancer research. John is the inventor of more than 20 patents in stem cell field: in an analysis of global stem cell patent landscape by Nature Biotechnology in 2014, John’s patent application and citation ranked No. 7 and No. 5, respectively.

17:45 – 18:45

Evening Drinks Reception

Gene Tx Development

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation
Applying principles of the 4th Industrial Revolution to accelerate novel therapeutic development

Digitalize data and meta dat
Digitalization of “Work” “Flow”
Integrate & streamline operations from research to development to manufacturing; and real-world data collection

Vasu Rangadass

President & Chief Executive Officer, L7 Informatics

09:05 – 10:05

Current landscape of viral and non-viral vector technologies

Key challenges and future for viral vectors
Recent advancement in non-viral vectors, key challenges, and prospects.
Key considerations when selecting vectors – viral versus non-viral, for various gene therapy modalities?

Kausiki Datta

Manager, External Innovation and Alliance Management
PTC Therapeutics

Kausiki Datta received her Ph.D. from Louisiana State University and completed her post-doctoral research at University of Oregon. She then joined Hoffman La-Roche where she worked on developing small molecule antivirals to treat Influenza and HBV. Kausiki subsequently moved to Novira Therapeutics where she worked on developing direct-acting antiviral for HBV which advanced to Phase 1 clinical trial. She then moved to the Center for NeuroGenetics at University of Florida as Research Assistant Professor where she served as the principal investigator for developing high-throughput platforms for screening compound libraries to treat ALS and Myotonic Dystrophy. She subsequently completed her MBA from Rutgers Business School and joined PTC Therapeutics where she leads multiple search and evaluation efforts within the research team. Kausiki has authored multiple peer-reviewed publications and has secured competitive grant funding from ALS Association and Wyck Foundation for her research. She has successfully initiated, developed, and led many industry-academic collaboration programs for advancing translational science to speed drug development.

10:10 – 10:40

1-2-1 Meetings / Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation
Financial and other considerations for early phase cell and gene therapy manufacturing: Build, Buy or Blend?

Ravi Samavedam

Chief Innovation Officer, Azzur Group

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:35 – 16:35

Selecting the optimal route of administration and AVV serotype for neurological applications

Option to penetrate the blood brain barrier
What in vitro and in vivo preclinical tools are useful?
Challenges and limitation for direct delivery to CNS
What do we know that AAV can and cannot achieve?

Gabriele Proetzel

Senior Director, External Neuroscience Innovation Neuroscience Drug Discovery Unit, Takeda

Gabriele Proetzel, PhD, Senior Director, External Neuroscience Innovation in the Neuroscience Drug Discovery Unit at Takeda Pharmaceuticals, based in Cambridge, MA, focuses on gene therapy therapeutic approaches for neurological disorders.

Prior appointments were at The Jackson Laboratory, Scil Proteins, Deltagen Inc., and Boehringer Mannheim/Roche. Gabriele earned her Master’s degree in Life Sciences from the Ludwig-Maximilian University of Würzburg, Germany, and a Ph.D. from University of Cincinnati, Ohio studying homologous recombination in embryonic stem cells to develop mouse models for disease. She completed her postdoctoral at the Max Planck Institute for Biophysical Chemistry in Göttingen, Germany.

16:35 – 16:45

Afternoon Refreshments

17:45 – 18:45

Evening Drinks Reception

C&G Tx Bioprocessing

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation
Applying principles of the 4th Industrial Revolution to accelerate novel therapeutic development

Digitalize data and meta dat
Digitalization of “Work” “Flow”
Integrate & streamline operations from research to development to manufacturing; and real-world data collection

Vasu Rangadass

President & Chief Executive Officer, L7 Informatics

09:05 – 10:05

Improving the upstream GT bioprocessing

Early assessment of manufacturability to determine if design modifications are required.
Genetic modification of the cell line to do more work.
Time and place; are the component parts in the right place at the right time. “

Joseph Rabinowits

Senior Vice President of Process science and Innovation, Kriya Therapeutics

Joseph Rabinowitz is the Senior Vice President of Novel Technologies at Kriya, joining Kriya from Pfizer where he was Senior Director of Capsid Development in the Rare Disease Research Unit where he led a team generating AAV viral libraries. Dr. Rabinowitz brings over 25 years of experience in AAV biology and vector development. Prior to Pfizer, he was faculty at Temple University working on gene therapy in heart disease. Dr. Rabinowitz received his PhD in genetics from Case Western Reserve University and completed his postdoctoral work at UNC Chapel Hill in Dr. Samulski’s lab on AAV biology and gene delivery. Dr. Rabinowitz received his undergraduate degree in microbiology from the University of Arizona, Tucson.

10:10 – 10:40

1-2-1 Meetings/Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation
Financial and other considerations for early phase cell and gene therapy manufacturing: Build, Buy or Blend?

Ravi Samavedam

Chief Innovation Officer, Azzur Group

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

16:35 – 16:45

Afternoon Refreshments

16:45 – 17:45

Strategy, technology, and challenges to meeting speed and quality requirements during early phase development

Cell culture and purification process development and technology to support early development
Fill finish technology
Analytical testing strategy
Development and FIH material comparability

Anjil Giri

Director, Gene Therapy CMC Development, PTC Therapeutics

Anjil Giri, Ph.D. is the Director of CMC Development at PTC Therapeutics, Inc. In this role, Dr. Giri is responsible for development and execution of CMC strategy for PTC’s portfolio of Gene Therapy Programs. He has 8 years of experience in Drug Development and has been involved in development and commercialization of two traditional biologics and more recently a Gene Therapy Product at PTC. He is passionate about developing innovative manufacturing processes and strategies and bringing life-changing therapies to patients with rare diseases. He earned his Ph.D. in Chemical Engineering from the Johns Hopkins University and completed his B.S. in Biological Systems Engineering from the University of Idaho.

17:45 – 18:45

Evening Drinks Reception

C&G Tx Manufacturing

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation
Applying principles of the 4th Industrial Revolution to accelerate novel therapeutic development

Digitalize data and meta dat
Digitalization of “Work” “Flow”
Integrate & streamline operations from research to development to manufacturing; and real-world data collection

Vasu Rangadass

President & Chief Executive Officer, L7 Informatics

09:05 – 10:05

Adapting analytics and quality control in cell therapy manufacturing to ensure adequate safety, purity and potency of final products

Multiple technologies for in process testing in cell therapy manufacturing
Cell therapy manufacturers Identifying potential issues early on in the manufacturing process and taking corrective actions to mitigate any risks by conducting in process testing, cell therapy manufacturers can
Using this approach to help to improve the consistency and quality of the final product and ensure that it meets the required specifications and standards for safety and efficacy

Bo Yan

Associate Director, Beam Therapeutics

Bo Yan leads a team in developing characterization methods and bioanalytical assays for a broad range of molecules (such as gRNA, mRNA, protein, LNP, and AAV), to support research, development, and GMP release of ex vivo and in vivo base editing therapy. Prior to joining Beam, Bo worked on characterization of biologics; small molecule separation and structural elucidation; nanoparticle characterization and biodistribution; and biomarker discovery. He has authored over 50 peer reviewed papers.

10:10 – 10:40

1-2-1 Meetings/Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

11:20 – 12:20

Defining gene-editing nucleases and plasmid DNA manufacturing strategies

Optimization & Development: Invest upfront to ensure downstream manufacturing efficiencies. Defining the balance between speed & time investment
Facility & Quality Requirements: Understanding your CMO’s facility infrastructure & quality systems to ensure suitability from toxicology through to late-clinical and commercial
Analytics & Documentation: Defining appropriate analytics and documentation for phased-appropriate manufacturing to meet regulatory requirements
Scale-Up: Build a strategy and CMO partnership that bridges to large-scale manufacturing and validation capabilities

Lexan Lhu

Associate Director, Business Development, Akron Biotech

Lexan Lhu brings nearly a decade of experience in providing technical support for single-use technologies and reagents in cell and gene therapy. Facilitated scale-up of RD methods into closed-system manufacturing processes. He also influenced the fluid paths of several commercialized cell therapy manufacturing platforms.

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation
Financial and other considerations for early phase cell and gene therapy manufacturing: Build, Buy or Blend?

Ravi Samavedam

Chief Innovation Officer, Azzur Group

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:00 – 15:30

Poster Session
Manufacturing of Autologous Novel T Cell Therapies Using the Cocoon® Platform Automated System

The poster highlights the challenges involved in manufacturing autologous cell therapies, specifically engineered T-cell therapies such as CAR-T immunotherapies. While CAR-T therapies have shown efficacy, they can cause adverse events, and their success in solid tumors is limited.

The presentation outlines an alternative approach that activates T-cells via native T-cell receptors, which has shown greater tumor antigen recognition. The process was translated and developed on the Cocoon® Platform Automated System.

The poster demonstrates the feasibility of translating a T-cell therapy from a manual process to an automated process using the Cocoon® Platform, which enables robust manufacturing, reduces costs, and accelerates the development of cell therapies to meet patient needs.

Soumendra Chatterjee

Clinical Application Scientist, Lonza

Soumendra Chatterjee is a Clinical Application Scientist with Lonza, Inc. working in process development for novel CAR-T cell therapy manufacturing. He graduated from The Pennsylvania State University with a graduate degree in Molecular Biology and a special focus in Biomedical Engineering. He will be sharing his insights on advancements in CAR-T cell therapy manufacturing.

15:35 – 16:35

Future of autologous cell therapy

Centralized manufacturing vs. onsite manufacturing. Which one is best?
How can CAR T cell Manufacturing become scalable?
What is the future of short manufacturing pipelines?
What are the alternatives to viral transduction of T cells?

Alberto Nobili

Alberto Nobili, Senior Director Strategy and Portfolio, Bayer US

Alberto Nobili, Ph.D. is a scientist and entrepreneur with focus on Cell & Gene Therapy (CGT) technologies. He is currently Senior Director for Portfolio and Strategy at Bayer Co.Lab US where he identifies and monitors startups for the Bayer CGT incubator. He is co-founder of Dynamic Cell Therapies, co-founder and vice-president of 42N Advisors Benefit Corp, and member of Mass Medical Angels. Dr. Nobili completed his postdoctoral studies at the Dana-Farber Cancer Institute (Harvard Medical School) and he obtained his PhD in Protein Engineering at the University of Greifswald (Germany). He is author of 12+ peer-reviewed articles and 5+ submitted patents.

16:35 – 16:45

Afternoon Refreshment Break

16:45 – 17:45

Tackling the challenges of AAV vector manufacturing

What are the current challenges facing AAV vector manufacturing?
How to overcome scale-up challenges? Is it all about the transfection complex?
Is there a role for AI and other technologies in addressing these manufacturing challenges or is this more about resolving the fundamentals?

Itzcoatl Pla

Head of Gene Delivery Process and Analytical Development, Bristol Myers Squibb

Itzcoatl Pla has more than 20 years of experience in the biopharmaceutical industry, ranging from process control, process development, process characterization, large-scale clinical and commercial manufacturing, establishing new experimental and technical teams, transferring processes to and from third-party manufacturers, process validation and life-cycle management and licensure by various Health Authorities. Itzcoatl currently leads the Gene Delivery, Process and Analytical Development organization, based in Seattle, WA. Itzcoatl joined Bristol-Myers Squibb at Devens, MA in July 2013 as the Director for Biologics MS&T Drug Substance, Global Capabilities and later Parenteral MS&T in New Jersey, having previously worked initially for Abbott Laboratories and Abbvie in Massachusetts, Singapore and Puerto Rico. Itzcoatl holds a B.S. in Biochemical Engineering from the Instituto Tecnológico de Veracruz, in Mexico, and a PhD in Chemical and Biomolecular Engineering from Cornell University in Ithaca, NY.

17:45 – 18:45

Evening Drinks Reception

ADVANCED THERAPIES XCHANGEEAST COASTBoston April 3, 2023

SNAPSHOTS OF DISCUSSION TOPICS

Full Xchange Agenda

Cell Tx Development

Gene Tx Development

C&G Tx Bioprocessing

Partners

ADVANCED THERAPIES XCHANGE
EAST COAST
Boston
April 3, 2023