Niazi Sarfaraz K, Magoola Matthias
College of Pharmacy, University of Illinois, Chicago, IL 60612, USA.
DEI Biopharma, Kampala P.O. Box 35854, Uganda.
Int J Mol Sci. 2024 Nov 28;25(23):12797. doi: 10.3390/ijms252312797.
Recombinant therapeutic and vaccine proteins have revolutionized healthcare, but there remain challenges, as many are awaiting development due to their slow development speed and high development cost. Cell-free in vivo ribosomes offer one choice, but they come with similar constraints. The validation of in vivo messenger RNA (mRNA) technology has been accomplished for COVID-19 vaccines. The bioreactors inside the body, the ribosomes, deliver these proteins at a small cost, since these are chemical products and do not require extensive analytical and regulatory exercises. In this study, we test and validate the final product. A smaller fraction of the recombinant protein cost is needed, removing both constraints. Although thousands of in vivo mRNA products are under development, their regulatory classification remains unresolved: do they qualify as chemical drugs, biological drug, or gene therapy items? These questions will soon be resolved. Additionally, how would the copies of approved in vivo mRNA protein products be brought in, and how would they be treated: as new drugs, generic drugs, or new biological drugs? Researchers are currently working to answer these questions. Regardless, these products' cost of goods (COGs) remains much smaller than that of ex vivo mRNA or recombinant products. This is necessary to meet the needs of the approximately 6.5 billion people around the world who do not have access to biological drugs; these products will indeed serve the dire needs of humanity. Given the minor cost of establishing the manufacturing of these products, it will also prove financially attractive to investors.
重组治疗性蛋白和疫苗蛋白已经彻底改变了医疗保健行业，但仍然存在挑战，因为许多产品由于开发速度缓慢和开发成本高昂而有待开发。无细胞体内核糖体提供了一种选择，但也有类似的限制。体内信使核糖核酸（mRNA）技术已在新冠疫苗方面得到验证。体内的生物反应器，即核糖体，以较低成本生产这些蛋白质，因为它们是化学产品，不需要大量的分析和监管工作。在本研究中，我们对最终产品进行测试和验证。所需的重组蛋白成本比例更小，消除了这两个限制因素。尽管有成千上万种体内mRNA产品正在研发中，但其监管分类仍未确定：它们属于化学药物、生物药物还是基因治疗产品？这些问题很快将得到解决。此外，已获批的体内mRNA蛋白产品的仿制品将如何引入，又将如何处理：作为新药、仿制药还是新生物药？研究人员目前正在努力回答这些问题。无论如何，这些产品的商品成本（COGs）仍远低于体外mRNA或重组产品。这对于满足全球约65亿无法获得生物药物的人群的需求至关重要；这些产品确实将满足人类的迫切需求。鉴于生产这些产品的成本较低，对投资者来说在财务上也将具有吸引力。
