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Moderna is aiming to build a geneediting franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare geneticdiseases and other conditions.
Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases.
The arrival of COVID-19 brought precious few positives, but one that emerged for the pharmaceutical industry was the emergence and validation of mRNA vaccine technology to prevent disease. Pfizer already has a head start on competitors in the field, having achieved the first US FDA approval for an mRNA vaccine, alongside BioNTech.
The RNA Revolution: From mRNA Vaccines to RNA Editing. The power of its real-world application was demonstrated by the resounding success of the mRNA COVID-19 vaccines. The mRNA COVID-19 vaccines have showcased the speed, versatility, scalability and flexibility that mRNA-based interventions offer.
In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light. Given their precise targeting, gene therapies have largely been focused on oncology indications followed by rare geneticdiseases.
RNA-based therapies hold the potential to offer new treatment options for diseases with limited or inadequate therapeutic alternatives. GeneEditing CRISPR-Cas9 and related gene-editing technologies continue to advance, and today they are widely used to study and develop therapeutic approaches for a broad range of human diseases.
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