In the coming days curative therapies will change the face of the healthcare system. More significantly, patients that previously had to rely on ongoing medication could be cured through specific, time-limited courses of treatment, which will transform their lives.
Knowing the potential of the curative therapies in the future healthcare systems, big players like Pfizer, Moderna, AstraZeneca have become increasingly involved in the curative therapeutics space and are increasingly developing novel technologies like – Synthetic mRNA, Gene Therapy and DNA sequencing. “Ties to innovation are close to the prospects of clinical success and subsequent investment, and there is cause for optimism.
The science in the field of regenerative medicine is hitting the make-or-break point over the next 2 years. If trials go well, we could see things take off like they have in the CAR-T [chimeric antigen receptor T cell] or oligonucleotide space,” explained Anthony Sun, partner at Aisling Capital.
In this article, let's take a look at the key novel curative therapy technologies that will shape the future of the healthcare industry.
Recombinant adeno-associated virus (rAAV) gene therapy
Recently, Pfizer rare disease is actively working on the (rAAV) gene therapy. In the paper named “Pfizer & Gene Therapy” the company pointed out that this approach works by targeting the missing or non-functional gene in an individual’s DNA, adding or replacing it with a functioning gene that, in turn, produces a functioning protein.
The rAAV gene therapy uses rAAC vectors which serves as a custom-made vehicle that can be infused into the body to deliver a functional gene to a specific target tissue–such as the liver or muscle–depending on the disease. When the vector reaches its target cell, the functioning gene is transferred and used as a blueprint to produce the missing or non-functional protein.
This is different from other approaches being investigated, like gene editing techniques, such as CRISPR, in which the functioning gene is integrated into a patient’s chromosomes. This approach has the potential to directly target cells with consistent treatment. It is a technology that can be standardized, potentially streamlining the manufacturing and regulatory path to medicine approval, mentions the paper.
Synthetic mRNA
Currently, Moderna therapeutics are actively involved in developing synthetic mRNA to cells to cure all manner of disease. The firm has developed proprietary technologies and methods to create mRNA sequences that cells recognize. Using mRNA as a drug opens up a breadth of opportunities to treat and prevent disease. mRNA medicines can go inside cells to direct protein production, something not possible with other drug approaches.
It has the potential to treat or prevent diseases that today are not addressable – potentially improving human health and impacting lives around the world.
Prime Editor Nuclease (PEn)
With an ambition to cure genetic disease, AstraZeneca is approaching to develop novel methods of promoting gene repair. In its recent paper, “Harnessing DSB repair to promote efficient homology-dependent and -independent prime editing” the firm showed its advances in DNA insertion techniques including the use of non-homologous end joining (NHEJ) – the primary pathway that human cells use for daily DNA repair.
The new approach to targeted DNA insertions builds on a recently developed prime editing technology and uses prime editor nuclease (PEn). This combines the DNA cutting SpCas9 nuclease enzyme with a reverse transcriptase – an enzyme that catalyzes transcription of RNA into DNA.
This approach can utilize a spectrum of efficient DNA repair mechanisms including NHEJ which robustly introduces programmed small insertions at DNA double strand breaks.
AstraZeneca’s next step is to move the PEn research from the in vitro to the in vivo setting. For this, it will be able to draw on the well-established expertise in novel delivery systems, such as lipid nanoparticle technology.
The firm is also building on the concept of localized NHEJ to develop novel methods to promote gene insertions in its therapeutic genome editing projects.
What's next?
In the future, increase in the curative therapies will increase the quality of life of the patients. Not to mention, the established revenue system of the healthcare industry will also be affected tremendously.
High initial sales caused by the treatment of large backlogs will lead to distinct first-mover advantages and large fluctuations in production volumes. To adhere to changing market dynamics, the pharmaceutical companies need to review their drug pipelines, portfolio management practices, and launch plans (marketing, sales, manufacturing) to accommodate the different properties of curative treatments, so they can proactively push for a first-mover position or adapt their strategies if that isn’t possible.
Lastly, developing the new capabilities across the healthcare system will be essential to ensuring that new therapies can be brought to market and implemented in clinical practice in an efficient and sustainable manner, prioritizing high-value treatments to the benefit of patients.
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