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
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
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.
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
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.
- 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?
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.
Networking Lunch
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
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.
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
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.
Gene Tx Development
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
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.
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
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.
Exosome biology in cell and gene therapy
- Approaches to the robust analysis of exosomes.
- Future trends for exosomal biomarkers.
- Exosomes bioanalysis
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..
Networking Lunch
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?
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.
C&G Tx Bioprocessing
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
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.
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
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.
- 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
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.
4:20 – 5:20pm
- Potency assays for autologous and allogeneic products
- Phase appropriate assay design
- GMP readiness and qualification/validation strategy
- Reference standard selection
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.
C&G Tx Manufacturing
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
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.
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
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.
- 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
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.
Networking Lunch
- Advantages and disadvantages of in-house manufacturing
- Selecting and successfully managing CMOs
- Strategies for transitioning from outsourced to internal manufacturing
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.
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.
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
- 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?
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.