New Platform for mRNA Platforms
Regulators from various countries will convene in Washington, D.C. in April 2024 to contribute to the development of the mRNA and RNA therapeutics field
The Alliance for mRNA Medicines (AMM) has announced that a meeting of global regulatory leaders will take place in Washington, D.C from April 4–5, 2024. The main focus will be on evaluating the advantages of a possible unified regulatory pathway across various organizations such as FDA, NMPA, MHLW, EMA, PMDA, ICH, stakeholders, and others. Every jurisdiction may take different paths, but it is important that they first establish a common framework for messenger RNA (mRNA) current good manufacturing practice (CGMP) compliance.
The meeting aims to explore the potential growth and appearance of the tree trunk. Practically speaking, using just one tree trunk as a metaphor may not be the most effective approach. It might be more helpful to consider multiple trunks, like a copse, thicket, spinney, or grove. There are various types of structural modalities, each with its own unique functions. Whether it's self-amplifying RNA (saRNA) or circular RNA (circRNA), the methods of handling and manufacturing can differ greatly. Just like with quantities, potency profiles, or ADME considerations, the same applies when talking about a generalized vaccine versus personalized oncology vaccines, gene editing, CAR-T, antibodies, or protein replacement therapies.
However, Peter Marks, director of the Center for Biologics Evaluation and Research at FDA, has consistently expressed his support for the idea of a collaborative regulatory pathway for mRNA platforms. In an interview with Pharmaceutical Technology, Marks highlighted the success of COVID-19 mRNA vaccines, with billions of doses administered, as evidence of the platform's safety and effectiveness in combating viral respiratory diseases. We expect that further research will broaden the application of mRNA vaccine technology.
At present, the production of mRNA vaccines is classified under broad categories that encompass genetic immunogens. These categories draw from the principles established for plasmid DNA vaccines and gene therapy vectors. The World Health Organization's (WHO) guidelines for vaccine development are considered a fundamental and comprehensive framework that applies to both plasmid DNA and RNA-based vaccines. These guidelines cover aspects such as quality, safety, and efficacy. The upcoming meeting is well-timed. EMA is seeking input on a guideline for regulating mRNA vaccines, including those for influenza and other diseases.
There is currently a growing movement towards applying successful strategies from infectious disease treatment, specifically the abundance of data collected during the COVID-19 pandemic response, to develop hybrid or combination therapy approaches. "Establishing agreed-upon templates as a foundational starting point," states Clay Alspach, the executive director of AMM, will help expedite innovative and potentially revolutionary treatments.
Additionally, Alspach highlighted the recent approval of ARCT-154 by Japan's MHLW. This sa-mRNA COVID-19 vaccine is intended for adults 18 years and older, serving as both an initial vaccination and a booster. According to Alspach, there is a need for better coordination and optimization of second generation approaches, including self-amplifying and various novel emerging methods, in their manufacturing processes. Regulatory compliance plays a significant role in that picture.
Andy Geall, the first board chair of AMM and CEO of Replicate Biosciences, emphasizes the need for increased international dialogue on the science and regulations surrounding rapidly advancing and converging technologies. He stressed the importance of a collective endeavor to inform the public about the upcoming changes, as they are happening faster than anyone is accustomed to. This is not your typical pharmaceutical development. This is happening quickly, with a wide range of approaches and technologies. We should prioritize providing accessible education to the public, governments, and regulators to make this information more understandable for them. In addition to addressing vaccine disinformation, Geall discussed the magnitude and potential of upcoming innovations, mentioning the dedicated individuals working in the field of rare diseases. We have various advancements in the field of medical research, including gene editing, gene therapy, and the development of vaccines for infectious diseases, oncology, and even early stages of research in neurology. It may seem intimidating at first, but it's just unfamiliar territory for those who aren't familiar with science. It's important to effectively convey the reasons why all of this is incredibly beneficial and has the power to bring about significant positive changes.
Source: EMA.europa.eu
