Editor’s picks: Advanced Therapies Week 2023

Cell therapy Industry developments Manufacturing & scale-up Upcoming & past events US and Canada

Phacilitate’s Advanced Therapies Week will return once again to the Miami Beach Convention Center (17–20 January 2023; FL, USA), and is bringing you a range of fascinating talks from industry experts covering all aspects of gene and cell therapy, as well as plenty of networking opportunities and the first edition of the Advanced Therapies Awards.

RedMedNet and Regenerative Medicine will of course be right there covering all the latest updates, and if you’re there in person you can find us at booth 118 – do come over and say hello to the RegMedNet team!

Read on for some of the talks and themes we’re most excited to see at Advanced Therapies Week:

Putting the patient first

  • What is the public and patient perception of cell and gene therapies? (18th January 2023, 11:00): Courtney Horvath (Novartis; Basel, Switzerland) will share her experience of frontline care in pediatric oncology after her son was diagnosed with stage 1, low-risk lymphoma and is joined by other speakers including Timothy Hunt (Alliance for Regenerative Medicine; Washington D.C., USA) and Teonna Woolford (Sickle Cell RED; MD, USA) in this session chaired by Ryan Leahy (Phacilitate; London, UK) to discuss patient perspective on these therapies. Mona Jhaveri (Music Beats Cancer; Washington D.C., USA) and Sven Kili (Antion Biosciences; Geneva, Switzerland) will join all the speakers in a closing panel with a Q&A.
  • Working with patient advocacy organizations to inform development (19th January 2023, 11:00): In this session chaired by Sharon Hesterlee (Muscular Dystrophy Association USA; IL, USA), Tessa Rowe (Tensentric; CO, USA) will discuss how human factors can be used in medical devices, Gwen Nichols (Leukemia and Lymphoma Society; NY, USA) will give a presentation on supporting blood cancer patients with advocacy, research and education, and Anne Rowzee (US FDA; MD, USA) will talk about the ways in which the FDA incorporates patient perspectives.
  • How do we best serve rare and small patient populations (20th January 2023, 11:00): Discover the scientific advances that have made gene therapy possible for a few rare diseases, how the small samples sizes available for rare diseases can limit the development of drugs and medical devices, and how a motivated community started a company launching the first in human allele-specific oligonucleotide (ASO) for Angelman syndrome. This session is chaired by Christian Chabannon (Institut Paoli-Calmettes; Marseille, France) with speakers Wilson Bryan (FDA), Brad Carlin (PharmaLex – USA; MA, USA), Allyson Berent (Foundation for Angelman Syndrome Therapeutics; IL, USA) and Sharon Hesterlee (Muscular Dystrophy Association USA). Tom Whitehead (Emily Whitehead Foundation; PA, USA) will join the speakers in a closing Q&A.

New technologies in gene therapy

  • The future of viral vectors (18th January 2023, 11:00): Nicole Paulk (University of California, San Francisco; CA, USA) chairs this session, which will feature presentations from Li Ou (Genemagic Bio; MA, USA), James Miskin (Oxford Biomedica; Oxford, UK) and Hanna Lesch (Exothera; Charleroi, Belgium). Learn about adeno-associated virus (AAV) gene therapy, the latest lentiviral vector platform processes and improving safety when manufacturing viral vectors with smart optimization tools.
  • Innovative delivery technology for gene and cell therapy (19th January 2023, 11:00): Pierre Boulas (Biogen; MA, USA), Justin McCue (Avectas; MA, USA), Leila Mekkaoui (Autolus; London, UK) and Farzin Fazaneh (ViroCell Biologics; London, UK) will present at this session chaired by Vincent Ling (Takeda; Osaka, Japan). They will discuss the importance of integrating the drug delivery and development strategy as early as possible when developing gene therapies, producing healthy and highly functioning gene-edited immune cells, accelerating vector manufacturing for early-phase clinical testing and enhancing safety and efficacy with programmable vectors.
  • Editing the future: gene editing tools and technology (19th January 2023, 15:30): Hear about the benefits of editing in ex vivo cell therapy, the evolution of new CRISPR technologies in gene therapy and the challenges of applying these technologies in clinical spaces and much more in this session chaired by Jian Irish (Metagenomi; CA, USA). This will feature presentations from Raisa Lowe (Arbor Biotechnologies; MA, USA), David Yoder (Aldevron; ND, USA), Chris Brown (Metagenomi) and Matthew Cato (Mission Bio; CA, USA), with Marco Ruella (University of Pennsylvania; PA, USA) joining the speakers for a closing Q&A.

Supply chain and technical operations

  • What does a global supply chain look like for CGT? Defining end-to-end logistics (18th January 2023, 11:00): Maia Hansen (Athersys; OH, USA) chairs this session, which will include presentations discussing how supply chains for cell and gene therapy need to be scaled up to meet a commercialization pipeline, supply chain logistics and practical advice on bringing these therapies to patients. The speakers include Kent Thorup (QuickSTAT; USA) and Jennifer Fenwick (WorldCourier; USA).
  • Think locally, source globally: Are your products region-fixed or trans-continental? (18th January 2023, 15:30): Troels Jordansen (Glycosystem Therapeutics; Oss, Netherlands) is joined by speakers Beth Gardner (Beam Therapeutics; MA, USA), Robert Newman (FujiFilm Irvine Scientific; CA, USA), Jeff Liter (Luminary Therapeutics; MN, USA) and Amy DuRoss (CSafe; OH, USA). Their presentations will explore choosing recombinant proteins for cell and gene therapy, advancements in patient access and reaching patients at a sufficient scale.

Find out more about Advanced Therapies Week and register here >>>