Advanced Therapies Week Superplenary: CGT – A Supercharged Industry

Anna Osborne
17 January 2023
Cell Therapy
Gene Therapy
It’s that time of year again were cell and gene therapies leading experts, enthusiasts and extraordinary people descend upon Miami for the annual Advanced Therapies Week – for a positive and productive start to the new year among colleagues old and new.
Welcome Back!

This year’s event in Miami is noticeably grander than last year’s (and that’s not just because of the better weather), with 2000 attendees and 180 exhibitors, a brand new shiny new track in the 6-track agenda, and a new larger-scale Superplenary theatre – all signs of the rapid growth the advanced therapies industry is experiencing!

To kick off the week of sharing, networking and learning, we were welcomed by Phacilitate Vice President Kimberly Barnes, who begins with a pat on the back for everyone in the room.

It has been a record-breaking year of CGT approvals, with Kim pinpointing 2022 as the year when the commerciality of cell and gene therapies became apparent.

Before we dive into addressing the challenges, obstacles and issues we see in this fast moving and novel area of science, that collaborative meetings such as Advanced Therapies Week are so great for highlighting, it is important to acknowledge there is a lot to celebrate and a lot to be proud of!

Superplenary Chair Anthony Davis joins Kim in celebrating the past year’s successes, sharing that 2022 saw a total of 9 commercial CGT approvals, 5 in the US and 5 in Europe. When compared to 2021’s 5 approvals it is clear cell and gene is heading towards a commercial field.


Ahead of The Game

First up to join Anthony from our Superplenary super esteemed panel was Peter Zandstra, Professor at the University of British Columbia.

“What motivates many of us is the beauty of cellular technology and the uncertainty and ability to play with that technology, that was, in the industry’s infancy, undefined” shares Peter.

In the early days when this field was just starting out so much was unknown, we are now at a point says Peter, were it has become very exciting. We can now start taking the complexity out of these systems and look at engineering to optimize manufacturing, reduce costs and improve overall efficacy.

The technology used, and the cell analysis that can be carried out, in cell therapy production has reached a very powerful level, but this is not yet matched by the data on safety and efficacy required by regulators.

Data is only useful if it can be acted on. This poses the question: ‘Do regulators understand the characterization of advanced therapies?’

So, how can we help ‘demystify’ these therapies to the regulators and to the public, so as to not let all the complex work behind them go to waste?

We are at a point where we can officially say that the production of cell therapies has gone from impossible to possible. However, the same can’t be said for their cost.

Over to The Patients

Next joining the (in her instance, virtual) stage was Gwen Nichols, Chief Medical Officer, Leukemia & Lymphoma Society (LLS). As a non-for-profit business, LLS supports advanced therapies from research to clinical development, through to educating clinicians and supporting the patient’s in their experiences.

Whilst the charity spreads its funds across the different stages of discovery and development depending on current demands it also provides help directly to individual patients. “I see us as a physician extender – not competitive of resources,” says Gwen.  For example, offering CAR-T travel funds to patients who don’t have access to a CAR-T facility, and providing those who are newly diagnosed with Lymphoma or Leukemia, with free resources.

Anthony presents Gwen with two thought-provoking questions, as someone in a unique position who has worked in this industry across clinical, commercial, and charitable fields.

If she were to go back in time what would she say to herself as a hematologist in clinical practice – “I would have asked patients in much more detail about their needs. A great prescription if the patient can’t fill it is meaningless.”

And if she went back to herself when she worked at Roche – “Continue to stick to my guns about what are the most exciting therapies going forward.”

Two important takeaways we can all gain from these answers; never underestimate the power of the patient and trust in the process.


The Good, The Bad, and The Cost of Cell and Gene Therapies

To shed some light on perhaps the biggest issue facing advanced therapies right now – cost – Anthony was joined for conversations with Michael Meyers, Managing Director of HC Wainwright & Chief Executive officer of Cell BT, and Steven B. Miller, Senior Vice President and Chief Medical Officer, Express Scripts.

First of Michael puts all of our minds at rest- – ‘There is hope’ he declares.

All jokes aside, Michael explains how he believes there is great potential for the current costing crisis plaguing cell and gene therapies to be tackled as ‘we are committed across different channels’. It is going to take collaboration right from early research days, through clinical and up to commercial stages, to make this a possibility.

Michael offers an alternative perspective for looking at the risk accompanying cell and gene therapies – ‘As a commodity that can be priced.’ Pricing this risk can have the benefit of attracting an investor pool who are looking for this level of risk.

A better method and a universal understanding for pricing these particular risks need to be put in place, with a detailed outlook on returns, for them to be better placed in the competitive market.

Michael continues to discuss the need for government backstops. It is important that we don’t leave insurers, payers and patients, on their own.

We have to face the issues surrounding costs head-on and with a pragmatic mind-set, as what will always sit peripheral to science and technology is finance.

Finally the panel was joined by Steven of Express Scripts, which serves 125 million patients over 30 countries. He notes that they are not just a payer, they also have a distribution arm, making direct deliveries, saving money and resources.

As the industry looks towards the next chapter, aiming to make cell and gene therapies more accessible to patients on a large scale, it is important to take look at the resources we have available to us.

Steven shares that he believes that one-third of the money put into the healthcare system is wasted. If this waste can be identified and reallocated, imagine the applications this could have in contributing to patient costs for life-changing advanced therapies.

If we can make these therapies more robust, in terms of procedure, product, and financial plan then there is hope that we can solve this problem and we will no longer be making life-saving therapies, with advanced technologies, that we can’t afford.


Reasons to be Cheerful: Cell and Gene

Anthony ended the session with a reference to his favorite song; Reasons to be Cheerful Pt.3. 

I think this premise is extremely important –  stepping back and taking a look at how far the industry has come, despite the challenges we still very much face today, and celebrating the industries collaborative work is what Advanced Therapies Week is all about.

I will leave you with the panel’s reasons to be cheerful, and I wonder what we will be celebrating next year….

Peter: ‘Our understanding and match to clinical efficacy is getting closer together.’

Gwen: ‘We now understand the microenvironment better and understand why some therapies don’t work for everyone, cost will go down if we aren’t using treatments on patients that it won’t work in’

Michael: ‘Yes I agree; our understanding of the tumor microenvironment has greatly progressed. We also have some really cool technologies coming along and also the ability to now start focusing on production and costs of goods.’

Steven: ‘When we look at the number of approvals we had this year and attendees here today at Advanced Therapies Week, we are still in our infancy but this is going to be a robust industry.’