CRISPR Gene Therapies Advance Amid Safety, Scalability Challenges

Recent advances in genome-editing technologies such as CRISPR and RNA-based regulation are transforming gene and cell therapies by improving precision, efficiency, and responsiveness. Tools like Cas9, Cas12a, base editing, and prime editing have broadened the scope of genetic manipulation, but selecting the best methods remains complex, prompting the development of high-throughput evaluation and machine learning to optimize guide RNA efficacy. Concurrently, studies on cellular stress responses reveal how certain messenger RNAs evade sequestration during stress, ensuring essential protein production, which informs understanding of RNA's regulatory roles. Despite the promise, the cell and gene therapy field faces challenges including regulatory uncertainties highlighted by Sarepta's Duchenne muscular dystrophy treatment and the FDA's fluctuating leadership, contributing to market volatility. Advances in RNA regulation, inspired by natural cellular mechanisms like splicing and folding, offer new avenues for precise control of therapeutic gene expression beyond traditional promoter reliance. Synthetic biology is further enabling the engineering of modular biological systems that can dynamically respond to cellular environments, potentially enhancing the safety and efficacy of future gene therapies.