Entering the Age of Genomic Medicine

MRNA PLATFORMS, CRISPR, STEM CELLS, GENE THERAPIES, PERSONALIZED MEDICINE & SOME OTHERS……IT IS A DECADE OF MEDICAL INNOVATIONS

The gene-editing tool which was discovered only about 7 years ago, has shown potential to be used in treating wide-range of diseases like cancer, HIV as well as haematological diseases.

Gene editing aims at modifying a specific disease-causing genetic sequence using molecular scissors to mitigate the effects of the disease variant. A common example is the CRISPR-Cas9 approach.

Scientists have been successful in extracting and treating the faulty genes from the stem cells and insert them inside the human body again which alleviates the symptoms. This is a breakthrough in curing the diseases by the means of genomics which will provide the necessary boost to the many diseases.

For instance, Lieber Congenital Amaurosis is the most common type of blindness in children which has no treatment as of now. But, a leading brand in genetic technologies, Editas Medicine Inc., is working on a CRISPR therapy where the respective cells will be edited on a genetic level to restore their function before a child loses the sight completely. The increasing innovations and advancements in genetic engineering will further boost the market in the next decade.

The Stem Cell Revolution

Discovered nearly 30 years ago in 1988, stem cell technology has failed to transform the field of regenerative medicine — until now. After delivering only one treatment during three decades of development, stem cell technology could finally become the all-purpose tool for repairing the body, thanks to rapid and precise genome editing techniques such as CRISPR. Several obstacles in stem cell technology – high costs, safety concerns, and bioethical considerations – are beginning to fall away. In particular, the introduction of induced pluripotent stem cells (iPSCs) eliminated many of the initial bioethical concerns stirred by the source of stem cells. Now, advances in genome-editing is accelerating the pace of progress.

Personalized medicine in the era of genomics

Overall, the challenge that researchers and physicians face is still immense nowadays. The purpose of personalized medicine is to combine modern medicine with molecular advances in order to target patients separately and improve the efficacy and effectiveness of the therapeutic approach

Cutting-edge biochemical advances including single-nucleotide polymorphisms (SNPs), genotyping, and biochips have made personalized medicine a reality justifying the use of the terminology in the last few decades.

Genetic vaccines

Thirty years ago, researchers for the first time injected mice with genes from a foreign pathogen to produce an immune response.

By 2019, academic laboratories and biotechnology companies all over the world had dozens of promising mRNA and DNA vaccines for infectious diseases, as well as for cancer in development or in phase 1 and phase 2 human clinical trials. When COVID-19 hit, mRNA vaccines in particular were ready to be put to a real-world test. The 94% efficacy of the mRNA vaccines surpassed health officials’ highest expectations.

DNA and mRNA vaccines offer huge advantages over traditional types of vaccines, since they use only genetic code from a pathogen – rather than the entire virus or bacteria. Traditional vaccines take months, if not years, to develop. In contrast, once scientists get the genetic sequence of a new pathogen, they can design a DNA or mRNA vaccine in days, identify a lead candidate for clinical trials within weeks and have millions of doses manufactured within months. This is basically what happened with the coronavirus.

Gene-based vaccines also produce precise and effective immune responses. This makes these vaccines better able to respond to mutations, and it also means they could be capable of eliminating chronic infections or cancerous cells.

The beauty of the messenger RNA vaccines is that they use a relatively cheap technology that instructs our cells to make a viral protein that hopefully will result in the body making broadly neutralizing antibodies against the virus we are targeting. But they will advance the field. This approach is going to take us beyond what we’ve been able to achieve so far.

The hopes that gene-based vaccines could one day provide a vaccine for malaria or HIV, cure cancer, replace less effective traditional vaccines or be ready to stop the next pandemic before it gets started are no longer far-fetched. Indeed, many DNA and mRNA vaccines against a wide range of infectious diseases, for treatment of chronic infections and for cancer are already in advanced stages and clinical trials.

I believe their proven effectiveness against COVID-19 will usher in a new era of vaccinology with genetic vaccines at the forefront.