CGT 2023: A Look Ahead
By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1
Once a month, my Life Science Connect editorial colleagues and I meet to discuss the most pressing topics in our respective coverage areas to learn from each other and to address any overlaps (hint: there are always many overlapping issues). Fresh off our most recent meeting, I thought I’d share with you what we covered and what are just some of the topics and trends Cell & Gene will continue to cover into next year.
PDUFA VII’s Impact of CGTs
By 2030, 60 CGTs are expected to reach regulatory approval. One of the biopharmaceutical industry's most closely watched regulatory developments is the FDA’s reauthorization of the Prescription Drug User Fee Act (PDUFA) or PDUFA VII, as it will force the FDA and CBER to have the capacity to accommodate the 60 aforementioned therapies. PDUFA VII will cover the fiscal years 2023-2027, and it places greater oversight emphasis on cell and gene therapies. The Act affects clinical trial inclusion requirements, manufacturing, etc. The reauthorization of the PDUFA Act allows FDA to collect fees from companies that produce certain human drug and biological products, including gene and cell therapies. PDUFA VII has the largest cell and gene package, and there is language in the Act specifically for rare diseases. Part of the regulatory problem to date (and COVID didn’t help) is that there are only so many skilled people in the regulatory agencies to determine a therapy’s approval, and thanks to PDUFA VII, CBER hopes to be better staffed.
In the coming weeks, BridgeBio’s Chief Regulatory Affairs Officer, Adora Ndu, will be my guest on Cell & Gene: The Podcast to discuss where we are with PDUFA VII’s reauthorization and what it means for CGT companies. Subscribe now to Cell & Gene: The Podcast wherever you get your podcasts so you don’t miss the episode when it airs.
The Capacity Crunch
If you’re reading this, you’re well aware of the on-going issues with planning for manufacturing capacity. As more therapies move from the bench to the clinic, the challenges unique to cell and gene therapies present problems in the capacity planning domain. Automation and AI’s impact on manufacturing will be key topics in the months, and well, years to come. In fact, Kite Pharma’s Head of Vector Manufacturing Quality, Will Junker, and I discussed the near-term future of capacity on Episode 32 of Cell & Gene: The Podcast. Junker and I talk about the various must-haves for a scale-up strategy. And we also will continue to see more of the acquisition and expansion announcements in manufacturing capacity to which we’ve become accustomed.
Genome Editing
2023 may bring us the first-ever approval of a CRISPR gene-editing therapy from Vertex and CRISPR Therapeutics. The FDA granted exagamglogene autotemcel (exa-cel) a rolling review for the potential treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Next month, Vertex plans to submit its biologics licensing application (BLA) for exa-cel for rolling review. The company expects to complete the submission by the end of Q1 2023. Then there’s bluebird bio’s $2.8M Zynteglo, the non-CRISPR-based gene therapy approved to treat beta thalassemia. In general, you can expect additional coverage on the different modalities of AAV gene therapy (i.e. gene replacement, gene editing, knockdown, vectorized antibodies, etc.).
Auto, Allo; Oncology, and Beyond
We can hope for breakthroughs in solid tumor oncology, and we should expect advancements in cell therapy beyond oncology (i.e. autoimmune and cardiovascular diseases, etc.). And, COGS and the financial feasibility of generating small-batch CGT products for rare and ultra-rare diseases with limited patient populations will be top coverage areas as well. Will the breakthrough in allogeneic therapy approach come along in 2023? In his recent article, Mark Gergen, CEO of Poseida explained that in order for allogeneic therapies to become a reality, we need to look at multiple factors including up-front efficacy, durability, safety, and access as well as health economics (i.e. cost). The end goal should be to make transformative allogeneic CAR-T cell therapies available to as many patients who can benefit from them as possible. Autologous cell therapies are proven, and all eyes are on allogeneic cell therapies for the foreseeable future.
Talent Acquisition, Retention, and Training
Attracting and retaining top talent is not the sexiest topic in the CGT universe, but it’s definitely one of the most important. For a market that is expected to be worth $5.5 billion by 2026, the CGT sector struggles with recruiting and retaining enough experienced candidates needed to bring life-saving treatments to market. Why is that, and what can you be doing now to hire and keep skilled professionals in highly sought-after roles? Indeed, we have seen the emergence of novel science leading to even more exciting clinical opportunities. With research and breakthroughs occurring at such a fast pace, expertise is being developed alongside the therapy itself. Rest assured biotechs will have recruitment and retention as a top priority in 2023 and beyond.
Supply Chain
There are countless challenges that go along with supply chain, manufacturing, and distribution that exist to make sure one-time doses are available when and where they are needed. There are many solutions to cover, but innovative technologies and strategies are key to unclogging the supply chain as it stands. Where and if it makes sense, manufacturing processes should go from open and manual to closed and automated.
Truth be told, I could keep you here all day line-iteming the myriad of hot topics that will continue to make our headlines. But, what are you most worried about? What CGT-related concerns keep you up at night? Tell me what they are by emailing me directly at erin.harris@cellandgene.com, and we will continue to deliver timely, data-driven content.