This year, the Cell and Gene Meeting on the Mesa was a hybrid event held in Carlsbad, CA from October 12-14. Each year this event connects executives and key decision-makers in the cell and gene therapy industry. As the first in-person meeting for many in the life sciences, the excitement was palpable. First and foremost, it was great to talk to actual people again instead of zoom screens! Every room at the venue was abuzz with conversation, as business professionals, directors, and executives discussed ideas and potential collaborations.
The conference was organized by the Alliance for Regenerative Medicine (ARM), an organization that is dedicated to supporting the advancement of safe and effective regenerative medicines. They noted how the association has changed in recent years, with a growing number of members and a marked effort to address topics pertinent to the rapid development of advanced therapeutics.
The company presentations and panel discussions were extremely informative. Here are a few takeaways:
1. The Industry is Thriving
From the various company presentations, it was apparent that the cell and gene therapy industry is making great progress. A variety of in vivo and in vitro therapies are in development and address several indications — including lysosomal disorders and diseases of the central nervous system (CNS), heart, and eye. Most companies had several candidates in clinical trials, with more in development.
2. Enabling Technologies are Bolstering Progress
Cell and gene therapy developers were not the only companies at the conference. Several biotechs were present that support the development of next-generation therapeutics through enabling technologies. For instance, some companies have automated platforms that can make molecules or optimize protocols for scaling and manufacturing.
Others offer services aimed at analyzing therapeutic products. In fact, Mission Bio falls into this category with our Tapestri Platform! Our CEO, Yan Zhang, gave an exciting talk about how Tapestri can co-measure the DNA and cell-surface protein in 1000s of individual cells — combining multiple assays into one. With this powerful system, therapeutic products can be characterized thoroughly and quickly.
Learn more here.
Mission Bio CEO, Yan Zhang, giving a talk at Cell and Gene Meeting on the Mesa 2021
3. Clarity with Patients is Imperative
One of the sessions addressed patient education and the need for better transparency and clear communication. The panel discussed how it was incumbent on clinicians to explain the experimental nature of clinical trials to patients in order to manage their expectations. Even for approved therapies, developers and clinicians have a responsibility to communicate what they can and can’t deliver.
The need for clear and consistent terminology was another issue. For instance, one of the panelists stressed that the term “cure” should be used with caution, especially since a given therapy may have variable effects across patients. The language that people in the industry use influences the expectations of patients and their families. It must be used thoughtfully.
4. Despite Hurdles, The Future is Bright
It was evident from this meeting that professionals in the cell and gene therapy space are passionate about improving the lives of patients. Yet, there are still challenges in the development of these therapeutics. One obstacle involves delivering in vivo gene therapies to target tissues and cells. This hurdle is being addressed by improving vector systems like adeno-associated viruses (AAV) and lipid nanoparticles and adapting gene-editing systems to fit into smaller cargo spaces.
Other challenges lie in ensuring the safety and efficacy of novel therapeutics while still advancing them through the development pipeline quickly. This is where new enabling technologies can be used to ensure that the next generation of therapeutics is successful. All in all, the future is bright for cell and gene therapies. As was evident at this year’s Meeting on the Mesa, there is both excitement and tremendous hope for the next wave of genetic medicines and the lives they will change.
