Advanced Therapies Xchange
East Coast
Boston - March 14, 2022

Welcome to hubXchange’s Hybrid Advanced Therapies Xchange 2022, 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.

Please note: This will be a HYBRID meeting. Participants can join in-person and virtually. All Covid protocols will be adhered to.

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

Cell Tx Development

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote Presentation: AML in a Patient with Sickle Cell Disease: A Case Study of Cross Functional and Inter-Agency Collaboration in Gene Therapy Development

Case of AML in a patient with sickle cell disease treated with lovo-cel gene therapy
Cross-functional collaboration to understand the root cause of the AML
Interorganizational collaboration to implement risk mitigation strategies
Lifting the clinical hold and clinical outcomes

Richard Colvin

Chief Medical Officer, Bluebird Bio

Richard Colvin, MD, PhD, has served as chief medical officer of bluebird since March 2021. Rich joined bluebird in 2018 and was medical lead of the thalassemia program and led clinical research and development prior to becoming CMO. Prior to bluebird, Rich was an executive director in translational medicine at Novartis where he led early-stage anti-infective drug development programs. Rich clinically trained at the Brigham and Women’s Hospital and remains on faculty at Harvard Medical School. He sees patients in the Infectious Diseases clinic at MGH. Rich received his MD and PhD from Duke University School of Medicine.

9:05 – 10:05am

Efficiently demonstrating clinical effect and value for One-And-Done Therapy Programs

How to demonstrate efficacy for therapies with slow onset and long duration to regulators, prescribers and payors
When is placebo control required? Desirable?
When is a run-in design sufficient?
Tokenization, external and synthetic control groups: pros and cons

Brendon Boot

Senior Medical Director
BlueRock Therapeutics

Brendon Boot BA MBBS FRACP is a clinician-scientist with 18 years of experience in clinical trial design and implementation. He was an investigator in 37 clinical trials in Australia, France, and the USA (Mayo Clinic, Brigham and Women’s Hospital, Massachusetts General Hospital and Harvard Medical School).
Brendon joined industry in 2014, serving as the Medical Director of high-profile programs at Biogen (Alzheimer: aducanumab), Voyager Therapeutics (Parkinson; VY-AADC01), and SSI Strategy (Huntington’s disease, FTD, immunology). He has conducted and analyzed pre-clinical and phase 1-4 programs in neurology, immunology, and rare disease covering small molecule, immunotherapy, gene therapy, antisense oligonucleotide, cell therapy and medical device therapeutics. In 2019, he joined BlueRock Therapeutics as the clinical lead for the DA01 Parkinson’s disease cell therapy program.

10:10 – 10:40am

1-2-1 Meetings/Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

11:20am – 12:20pm

Take back stability control! How can we overcome the narrow cell viability window?

How to improve viability of stem cells post-cryopreservation
Important considerations for extending cell viability at room temperature
How can the quality of excipients affect performance of the final cell therapy product?

Brian MacDonald

Head of Business Development (US), Albumedix

Brian has extensive experience in the pharmaceutical and biopharmaceutical spaces with areas of focus in sales and business development. Leading program development efforts and building relationships across distribution channels, manufacturing to support pharmaceutical development in small and large molecule programs for various biologic and ATMP clients. With a more recent focus in the CRO and CDMO areas focusing on viral and vaccine clients as well as early and late stage ATMP clients developing clinical and commercial programs with providing cGMP manufacturing solutions for the more challenging drug substance, drug product and final packaging/cold chain programs within the advanced therapy space.

12:20 – 1:20pm

Networking Lunch

1:25 – 2:25pm

Challenges and differences in the development of cell-based therapies

Therapeutic effects of MSCs, extracellular vesicles and secretome
Alternatives to autologous: Allogeneic and in-vivo CAR-NK / T cell therapies
Phase-appropriate characterization and alignment with the regulatory path
Using analytical data to provide manufacturing solutions

Ziv Kirshner

Director of Advanced Analytical Sciences
Noveome Biotheraputics

Ziv Kirshner, is the Director of Advanced Analytical Sciences at Noveome Biotheraputics, Inc. Noveome is advancing its ST266 platform biologic, a multi-targeted secretome containing many factors crucial to neuroprotection, the modulation of inflammation, cell recovery and healing. Ziv has extensive experience in the development of multiple cell and gene therapies across all stages of development and for a wide range of diseases. He was a lead scientist in Lonza Biologics and a scientist in Multi-Gene Vascular Systems Ltd. He holds a PhD in Pharmaceutical Sciences from the University of Pittsburgh and is dedicated to studying the mechanisms underlying the beneficial effects of complex and cell-secreted biologics.

2:30 – 3:00pm

1-2-1 Meetings / Networking Break

3:00 – 3:30pm

1-2-1 Meetings / Networking Break

3:30 – 3:45pm

Afternoon Refreshments

4:20 – 5:20pm

Cell therapy for solid tumors: challenges and opportunities

Finding the tumor
Trafficking CAR T cells to the tumor site
Overcoming hostile tumor microenvironment
Production logistics and financial accessibility issues

Jun Ren

Scientific Director
Dynamic Cell Therapies

Jun Ren is a Scientific Director at Dynamic Cell Therapies (formerly Raqia Therapeutics), a cell therapy company dedicated to developing next-generation CAR T-cell therapy for the treatment of solid and liquid tumors. Jun has a multidisciplinary background and deep expertise in CAR T-cell therapy, immuno-oncology, and tumor microenvironment. Prior to joining Dynamic Cell Therapies, he worked at Massachusetts General Hospital and Harvard Medical School, where he developed multiple therapeutic strategies overcoming resistance to cancer immunotherapy and provided mechanistic insights in support of clinical trials. Jun received his Ph.D. from the University of Wisconsin-Madison, followed by postdoctoral training at Massachusetts General Hospital.

5:20 – 6:20pm

Drinks/Canape Reception

Gene Tx Development

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote Presentation: AML in a Patient with Sickle Cell Disease: A Case Study of Cross Functional and Inter-Agency Collaboration in Gene Therapy Development

Case of AML in a patient with sickle cell disease treated with lovo-cel gene therapy
Cross-functional collaboration to understand the root cause of the AML
Interorganizational collaboration to implement risk mitigation strategies
Lifting the clinical hold and clinical outcomes

Richard Colvin

Chief Medical Officer, Bluebird Bio

9:05 – 10:05am

Concurrently optimization of manufacturing processing with clinical development

Coordination of clinical development with CMC
Optimizing the CMC process via clinical data
Functional releases assays coordinating with clinical assay

Richard Colvin

Chief Medical Officer
Bluebird Bio

10:10 – 10:40am

1-2-1 Meetings/Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

11:20am – 12:20pm

Exosome biology in cell and gene therapy

Approaches to the robust analysis of exosomes.
Future trends for exosomal biomarkers.
Exosomes bioanalysis

Justin Devine

Chief Medical Officer & Co-Founder, Synexa Life Sciences

Justin is a medical doctor, immunologist and pharmacologist and a co-founder and CMO of Synexa.

His primary focus is to understand our clients’ objectives in new drug development and to implement biomarker strategies that bring real insight to the challenges of clinical development and the immunological underpinning of health and disease..

12:20 – 1:20pm

Networking Lunch

1:25 – 2:25pm

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.

2:30 – 3:00pm

1-2-1 Meetings / Networking Break

3:00 – 3:30pm

1-2-1 Meetings / Networking Break

3:30 – 3:45pm

Afternoon Refreshments

5:20 – 6:20pm

Drinks/Canape Reception

C&G Tx Bioprocessing

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote Presentation: AML in a Patient with Sickle Cell Disease: A Case Study of Cross Functional and Inter-Agency Collaboration in Gene Therapy Development

Case of AML in a patient with sickle cell disease treated with lovo-cel gene therapy
Cross-functional collaboration to understand the root cause of the AML
Interorganizational collaboration to implement risk mitigation strategies
Lifting the clinical hold and clinical outcomes

Richard Colvin

Chief Medical Officer, Bluebird Bio

9:05 – 10:05am

Building essential characterization data for bioprocesses and translation

Preclinical data packages for success
Frontiers in novel analytics, characterization assays, and metrology
Regulatory and safety considerations

Michael Look

Senior Principal Scientist
Pfizer

Michael Look is a Senior Principal Scientist at Pfizer Inflammation and Immunology where he works on discovery and preclinical development of immune tolerance therapies. Michael is experienced in developing novel therapeutic modalities including drug delivery systems, nanotechnology, and biotherapeutics. He holds a PhD in biomedical engineering from Yale University and MBA from University of Massachusetts Amherst.

10:10 – 10:40am

1-2-1 Meetings/Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

12:20 – 1:20pm

Networking Lunch

1:25 – 2:25pm

Challenges in gene therapy potency assay development

Phase appropriate strategies for assessing potency of gene therapy products
Development of in vitro potency assays
Approaches for assessing mechanism of action
Use of relative potency assays

Jim Richardson

Senior Director, Analytical Development
Interius Biotherapeutics

Jim Richardson leads the analytical development group at Interius. Trained as a virologist, Jim has over 25 years experience in developing viral products for the prevention and treatment of disease. Jim comes to Interius from Altimmune, where he was Director of Scientific affairs leading efforts to identify and evaluate strategic opportunities. Jim also led the cell and gene therapy standards development efforts at US Pharmacopeia. In previous roles at Advanced BioScience Laboratories and Foundation Fighting Blindness, he led translational science activities for the development of vaccines and biologics to prevent and treat infectious and retinal diseases. Earlier in his career, Jim held positions responsible for performing viral clearance and adventitious agent testing at Viromed Biosafety as well as AAV vector development and characterization at Genovo/Targeted Genetics. Jim earned his Ph.D. in Biomedical Sciences at the Mount Sinai/Icahn School of Medicine.

2:30 – 3:00pm

1-2-1 Meetings / Networking Break

3:00 – 3:30pm

1-2-1 Meetings / Networking Break

3:30 – 3:45pm

Afternoon Refreshments

4:20 – 5:20pm

Challenges in cell therapy potency assay development

Potency assays for autologous and allogeneic products
Phase appropriate assay design
GMP readiness and qualification/validation strategy
Reference standard selection

Bo Yan

Associate Director, Analytical Sciences
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.

5:20 – 6:20pm

Drinks/Canape Reception

C&G Tx Manufacturing

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote Presentation: AML in a Patient with Sickle Cell Disease: A Case Study of Cross Functional and Inter-Agency Collaboration in Gene Therapy Development

Case of AML in a patient with sickle cell disease treated with lovo-cel gene therapy
Cross-functional collaboration to understand the root cause of the AML
Interorganizational collaboration to implement risk mitigation strategies
Lifting the clinical hold and clinical outcomes

Richard Colvin

Chief Medical Officer, Bluebird Bio

9:05 – 10:05am

Cell Tx Development Topic-
Tackling immunogenicity of cell therapies

Factors contributing to immunogenicity for autologous, allogenic, or in vivo reprogramming cell therapies; delivery system/formulation & payload
Parameters to be optimized or managed bed side to avoid unwanted immune response
Preclinical models to assess immunogenicity and their clinical utility impacting safety, efficacy, and dosing frequency
Monitoring immunogenicity in clinic for Immuno-oncology and other disease area

Dharini Shah

Senior Research Director, Immunology
Tidal Therapeutics, a Sanofi Company

Dharini Shah, Ph.D. is a Senior Director, Immunology for Tidal Therapeutics, a Sanofi company developing novel mRNA-based approach for in vivo reprogramming of immune cells. Multi-disciplinary scientist with 17 years’ experience building teams and leading projects from inception to clinical candidate for immuno-oncology, autoimmune and rare immune metabolic disorders. She started her industry career at Boehringer-Ingelheim in translational sciences and also delivered new drug concepts. Transitioned to Pfizer and led projects for immune checkpoint inhibitor. Prior to Tidal, at CoMET she led research for inborn error of metabolism and expand platform for immune disorders that was acquired by VectBio AG. She earned her Ph.D. at Eppley Cancer Research Institute (UNMC), followed by postdoctoral training at Massachusetts Institute of Technology.

10:10 – 10:40am

1-2-1 Meetings/Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

11:20am – 12:20pm

Overcoming ancillary material closed systems challenges – strategies and solutions to functionally close the incorporation of ancillary materials into the cell therapy manufacturing process

Are you currently experiencing ancillary material challenges with closed systems? If so, how are you overcoming these challenges?
What ancillary materials are most critical to move into closed system formats?
What are your closed systems container material requirements? Form-fit-function
Which quality attributes are most important? For example, particulates specifications, other required tests and/or regulatory requirements

Robert Margolin

Vice President, Sales, Akron Biotech

Robert brings 14 years of business leadership and sales experience in the cell and gene therapy market, with a focus on business development and corporate strategy and execution. His experience ranges across cell therapy drug product and ancillary material manufacturing. Robert’s deep knowledge of clinical and commercial operations has driven process development, scale-up, and design and configuration of electronic systems and logistics platforms for each stage of development. Additionally, Robert played an instrumental role in the formation and growth of the Alliance for Regenerative Medicine, serving as its vice president of communications, along with launching several of the field’s preeminent conferences and events.

12:20 – 1:20pm

Networking Lunch

1:25 – 2:25pm

Building vs. outsourcing manufacturing capacity

Advantages and disadvantages of in-house manufacturing
Selecting and successfully managing CMOs
Strategies for transitioning from outsourced to internal manufacturing

Damien Hallet

Vice President – CMC
Affini-T Therapeutics

Damien Hallet has more than 20 years of international biotech and biopharmaceutical industry experience working on the development on various biologic medical products (cell & gene therapeutics and therapeutic proteins). Prior to Affini-T, Damien was the Head of CMC Technical Operations and Strategic Planning at Precision Biosciences where he contributed to the development and successful IND submissions of their first four allogeneic CAR-T cells programs. Prior to joining Precisions Biosciences in 2017, Damien served 4 years as VP of CMC at Heat Biologics where he was responsible for all aspects of CMC development and manufacturing of their allogeneic whole cell cancer vaccines. Earlier in his career, Damien served in various process development management positions at UCB Pharma (UK), Sanofi (Research Centre, Paris) and Centelion SAS (a subsidiary of Aventis Pharma dedicated to Gene Therapy). Damien received his Master of Advanced Studies in Industrial Microbiology from Ecole Doctorale de Toulouse, and a Biochemical Engineering degree from INSA de Toulouse, France.

2:25 – 3:00pm

1-2-1 Meetings / Networking Break

3:00 – 3:30pm

1-2-1 Meetings / Networking Break

3:30 – 3:45pm

Afternoon Refreshment Break

3:45 – 4:15pm

Poster Session: Your future cell therapy manufacturing success-story: The Lonza Cocoon® with integrated magnetic separation

The Lonza Cocoon® is a closed, automated platform for end-to-end cell therapy manufacturing. As manufacturing processes evolve, Lonza continues to upgrade the Cocoon® Platform with new functionalities. The recently released “Integrated, in-line magnetic cell separation” will allow the automated isolation of T cells as a unit operation within the system, resulting in standardized cellular input material and greater control over closed cell therapy manufacturing processes.

Peter Yates

Associate Director, Business Development & Clinical Applications, Lonza

Peter Yates, PhD, is currently Associate Director of Business Development & Clinical Applications for Lonza’s Personalized Medicine Business Unit. His focus is on the Cocoon® Platform, a closed and automated cell therapy manufacturing solution. Peter earned a doctorate in Genetics, Molecular, and Cellular Biology – Microbiology and Molecular Immunology from University of Southern California. He has been with Lonza since early 2021.

4:20 – 5:20pm

Future of autologous cell therapy

Will alternatives to autologous CAR cell therapy such as allogeneic/hypoimmune or in vivo/bedside gene therapies be as effective as centrally manufactured autologous cell therapy?
- what new issues arise with non-autologous cell therapies such as lack of persistence, inability to redose, safety concerns, etc?
Decentralized manufacturing:
- Who owns the therapy and pays for the clinical development?
- Will these systems enable hospitals to own the therapies themselves and use under practice of medicine (discuss various regulatory agency outlooks)?
- Who pays for the physical plant and FTE resources to run the systems in decentralized model?
- Will such systems enable 3rd party vendors to set up tertiary centers such as dialysis centers to conduct therapies?

David Peritt

Founder and CSO
Lupagen

David Peritt is a cellular immunologist by training with substantial industry experience in biologics and cell therapy. He was formerly Chief Technology Officer at Sigilon Therapeutics, a Flagship Pioneering company, until soon after they went public in late 2020.

Prior to this he was Program Lead for Cell and Gene therapy at Pfizer, Head of Biosimilars Biology and scientific lead for strategic investment in cell therapy for Hospira. For a decade, David also led research efforts at several divisions of Johnson & Johnson including Centocor and Therakos as well as experiences at ImClone Systems, USDA, Walter Reed Army Medical Center and the Sharrett Cancer Institute.

David received his Ph.D. and post-doctoral training in immunology from the University of Pennsylvania and the Wistar Institute. He also serves as an adjunct professor, Vice Chairman MBP Advisory Board at Northwestern University McCormick School of Engineering. He advises for several cell and gene therapy companies.

5:20 – 6:20pm

Drinks/Canape Reception

Advanced Therapies Xchange
East Coast
Boston - March 14, 2022

Cell Tx Development

Gene Tx Development

C&G Tx Bioprocessing

C&G Tx Manufacturing

Partners

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Advanced Therapies XchangeEast CoastBoston - March 14, 2022

Cell Tx Development

Gene Tx Development

C&G Tx Bioprocessing

C&G Tx Manufacturing

Partners

Advanced Therapies Xchange
East Coast
Boston - March 14, 2022