Everything was a little different at this year’s (January 25-28) Advanced Therapies Week: from the new venue (Miami Beach Convention Centre), to the COVID-19 precautions, to one less track (stem cell talks were removed) and the replacement of the boat cruise networking event with a beach networking event, the conference was slightly altered from what regular attendees are accustomed to.
Nevertheless, there was a lot to like about this year’s version. The “superplenary,” which kicked off the talks, featured Tom “Emily’s dad” Whitehead who was as gracious and moving as he has been in the past. (If you’re not already familiar with Emily’s story of being the first patient to receive experimental CAR T-cell therapy in a clinical trial back in 2012, it has a very happy ending for Emily and her family, but also for the broader patient community and industry. That clinical trial led to the first approved cell-based gene therapy. More on this below.)
The “superplenary” was chaired by Dr. Rob Allen of Dark Horse Consulting. He was joined on stage by Lisa Ward, the mother of a son who passed away from Diffused Intrinsic Pontine Glioma (DIPG), a deadly, rare pediatric brain tumour. Ms. Ward and her son Jace were advocates for DIPG and funding rare diseases, and she continues this important mission. The patient advocates were well received. At another talk, a member of the audience stood up to thank Mr. Whitehead for his presence at the conference and for reminding the delegates why our industry is dedicated to getting therapies into the hands of patients.
We also heard from Shephard Mpofu, Chief Medical Officer, Novartis Gene Therapies. Novartis was the first company to receive FDA-approval for a CAR T-cell therapy, back in August 2017. Dr. Mpofu spoke about the challenges of reimbursement and the need to reduce cost of goods sold (COGS), and the importance of untangling the manufacturing cost from the value of the product. If you’d like to delve deeper into what he said, I recommend you read this article in BioProcess International.
The final speaker invited to the stage was Craig Shepherd, Senior Managing Director, Blackstone Life Sciences. He cautioned that the pandemic has revealed the chemistry, manufacturing and controls (CMC) risk and emphasized that it is “unrivaled” in cell and gene therapy manufacturing. Read more about this important topic here. CCRM has written about CMC in the context of ways to maximize a cell and gene therapy budget.
Despite changes to this year’s conference, the first plenary stuck to a popular formula: Janet Lynch Lambert, Chief Executive Officer of Alliance for Regenerative Medicine (ARM), presented the state of the industry and Susan Nichols, Chief Executive Officer of Propel Biosciences, offered her top 10 highlights of 2021.
According to ARM, 2021 saw long-term growth and short-term volatility in the form of record-breaking investment; gene editing rising in prominence; venture capital ascending; and it was a “down year” for public equities. As of Q3, there were 1,307 active therapeutic developers worldwide, 2,261 global clinical trials with 1,000+ sponsored by industry and slightly more sponsored by academia and government, and over US$23 billion[1] was raised. Six new products received FDA approval, with half designated as RMAT. ARM expects 2022 to be a big year for rare disease approvals with gene editing continuing its “march to the clinic.” Keep an eye on ViaCyte/CRISPR, Beam and Verve, and expect to see data from Caribou, Intellia and others. For future predictions, see this slide from Ms. Lynch Lambert’s presentation.
Alliance for Regenerative Medicine slide, Advanced Therapies Week 2022
In alignment with Craig Shepherd, ARM expects CMC to be a key sector challenge, but also dosing and delivery, and policy and reimbursement, which continue to cast a shadow over the industry.
Susan Nichols began her top 10 talk by reminiscing on how far this conference (and the industry) has come. When she attended her first conference organized by Phacilitate, there were 10 vendors and about 100 delegates. I was told by one of the organizers that 1,100 people registered for this year’s event – hence the move to a larger venue.
Here is Ms. Nichols assessment of 2021 highlights, in reverse order.
10. Autologous CAR-T process improvements – the process is speeding up
9. Gene editing platforms land significant investment – Prime Medicine emerged from stealth mode with $315 million; Metagonomi received $175 million; and, Chroma Medicine received $125 million.
8. Advances in mRNA-based medicines – moving beyond COVID-19 vaccines
7. In vivo gene editing enters the clinic
6. Market/patient access – see details below
Slide from Susan Nichols’ presentation at Advanced Therapies Week 2022
5. Expansion of CGTx-focused end-to-end service providers – news from Charles River (see Cognate, Vigene and Hemacare), Danaher, Resilience (see Swiftscale Biologics, bluebird bio and Ology Bioservices), Thermo Fisher Scientific and Sartorius.
4. Workforce development opportunities – we know talent shortages are affecting the industry, resulting in increased competition for people; more job mobility; remote/relocation opportunities; and, talent development.
3. AAV adverse events
2. Investor focus expands tools and technologies
1. CAR T advances in line of treatment – Ms. Nichols says she gets “goosebumps” at the thought of CAR T treatments moving from fourth or fifth line of treatment to second or even first line standard of care. I think that’s something we can all get excited about.
For more details behind the highlights above, read 2021 CGT moments: First place for second line CAR-T.
In memory of Jace Ward, here he is with his mother, Lisa, and neuro-oncologist Dr. Michael Prados. The video is a poignant reminder of what inspires our industry. (I will post a second blog, with more coverage from Advanced Therapies Week, in the coming weeks.)
[1] All dollar figures are U.S., unless specified.
Stacey Johnson
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