Advanced Therapies Xchange
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
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.
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
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
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.