Advanced Therapies Week Countdown: 5 Agenda Highlights Not to be Missed

Ryan Leahy
16 December 2022
Cell Therapy
Clinical Trials
Gene Therapy
Patient Access & Engagement
Phacilitate’s Advanced Therapies Week 2023 is almost upon us, and we know that this is the way to start off the year right – think of it as a late Christmas present for cell and gene therapy knowledge leaders!

This year, I’ve been asked what are the not-to-be missed sessions across the ATW agenda, and boy, was that a tough ask. Without a doubt this agenda delivers on several key promises we made in our research and outreach – to highlight the patient voice within your development journey, to platform disruptive technologies that will transform your workflows, and to explore the latest innovations in breakthrough cell and gene therapy therapeutics.

1. Putting Patients First: Patient Advocacy, Engagement and Patient-Centric Development for CGT Therapeutics Developers, Manufacturers and All Elements of the Advanced Therapies Commercial Journey

A major consideration for the Advanced Therapies Week and a core goal approaching the 2023 agenda was the inclusion of patient organizations – not just to remind us why we are here and who eventually receives these therapies, but to ensure that the patient voice is recognized and implemented throughout the entire process from translation through manufacturing through to commercialization. Cell therapy in particular is a patient-determined process, where the source material often comes directly from the patient they are treating; cell and gene therapies are also targeting the most difficult indications with particularly high-risk patients.

We have four core sessions that revolve around the patient voice with significant input from the advocacy organizations, developers and the FDA, but have also implemented this throughout all workflows of the agenda.

– Public and Patient Perceptions of Cell and Gene Therapy

– Patient-Centered Development in the Commercialization Journey of Advanced Therapies

– Working With Patient Advocacy Organizations

– How Do We Best Serve Rare Disease Patients?

Find out more. Join us at Advanced Therapies Week 2023 >>

2. What’s Next for Cell and Gene Therapies?

A major emphasis for the meeting is to be future-looking, and this is particularly pertinent within our manufacturing, commercialization and supply chain tracks (not to mention New and Enabling Technologies!) – but what about the drug products and therapeutic indications?

Several key sessions look to what developers are looking to target for the future treatments with cell and gene therapy, including sessions focused on:

– Therapeutics for AutoImmune Indications (Kyverna Therapeutics, Cabaletta Bio, Jura Bio)

– Treatments for Neurological Disorders (AviadoBio, Taysha Gene Therapies, Aspen Neuroscience)

– Gene Therapies Entering the Mainstream (for treatment of indications such as heart disease)

– Future cell therapies – Allogeneic, Off-the-Shelf, NK Cell based therapeutics (AdaptImmune, ATARA Bio, Glycostem, Wugen, Alloplex, Tevogen)

3. How Are We Approaching the Cost, Pricing and Payment of Cell and Gene Therapies?

With advanced therapies once again boasting the most expensive drug in the world (With UniQure/CSL’s Hemagenix, weighing in at $3.5 million per dose), and a year fraught with difficulties when it comes to entry into the European pricing market, a critical question hangs over cell and gene therapies: how are we determining the value of these medicines, and how are we ensuring both market and patient access to these breakthrough products?

A critical session within the ATW agenda is “Paying For Cures”, which looks at success stories in the cell and gene therapy space from Novartis, bluebird bio and Orchard Therapeutics, and explores the essential revolutions in novel pricing and reimbursement for these expensive but life-altering medicines.

4.The Workforce Conundrum: Building the technical, scientific, commercial and leadership workforce necessary for the furtherance of cell and gene therapies

The advanced therapies space has a major sticking point – where do you source the skilled personnel necessary throughout the various workflows? The problem isn’t just facing developers; it also effects manufacturers, regulators and clinical areas.

Advanced Therapies Week is tackling this particular issue across multiple sessions throughout the agenda, including our “The People Behind the Cures” session, led by Angela Justice, Chief People Officer for TCR2 Therapeutics, which looks to cover recruitment, opportunity and the importance of diversity in cell and gene therapies. Our “Enhancing Technical Knowledge in CGT” session brings together an expert panel of bioprocessing training and workforce development professionals to explore how we bolster both the training and sourcing of talented personnel within the manufacturing and supply chain areas where experts are sorely lacking. As well as this, our Opening Plenary will touch on the requirements necessary for leaders in the CGT field, and our Roundtable discussion on “Building and Retaining Excellent Teams” with Matt Li of Tome Bio speaks to the important conversation around retention of talent, as well as their positioning within critical workflows.

5. Advances That Improve Cost-of-Goods and Expedite Time-to-Patient

Central tenets of the Advanced Therapies Week (and more broadly, the Phacilitate Network) have always been “Advancing The Next Pillar of Medicine”. A critical component of this and a central focus for the ATW meeting has classically been Manufacturing, Supply Chain, and novel, disruptive technologies that will transform the space.

Expect to see important launches of critical products that will revolutionize cell and gene therapy workflows, all with the goal of reducing cost of goods and improving quality and safety of drug products. Within sessions like “Operational Excellence” and “Building Better Cell Therapy Processes” expect to see developers wrestle with key considerations in improving the process development and manufacturing of CGTs, while sessions that focus on digitalization, automation and closed systems explore how these technologies can be properly implemented and transform the way we develop, manufacture and deliver the therapies of the future.

Join us at Advanced Therapies Week >>