Date : 05/08/2023

Relevance: GS Paper 3 – Science and Technology- Developments and their Applications and Effects in Everyday Life – Biotechnology, Genetic Therapy.

Keywords: Genetic Engineering, Protein Nanoparticle, mRNA, Crispr, Cancer Treatment, Genetic Therapy.

Context:

Recently, Aera Therapeutics, a cutting-edge biotech company, revealed a revolutionary protein nanoparticle designed to efficiently deliver a wide range of genetic medicines throughout the human body.

Key Highlights:

Certain approaches circumvent the delivery challenge entirely by extracting cells from the patient's body, modifying them in a laboratory setting, and subsequently re-administering them back to the patient. However, this strategy is associated with time-consuming procedures, high costs, and can be physically demanding for the patients.

What is Genetic Therapy?

Gene therapy is a medical technique aimed at modifying a person's genes with the purpose of treating or curing diseases. These therapies are being researched extensively for the treatment of various conditions such as cancer, genetic disorders, and infectious diseases. It's important to note that not all medical procedures that involve genetic modifications fall under the category of gene therapy.

Gene therapies operate through several mechanisms, which include:

1. Substituting a disease-causing gene with a healthy version of the gene.
2. Inactivating a malfunctioning disease-causing gene.
3. Introducing a new or altered gene into the body to aid in the treatment of a specific disease.

What are the limitations of present Genetic Therapy?

Currently available genetic therapy technologies face limitations in their ability to target and fix the genome in various parts of the body. The areas where cures are more feasible include the liver, eyes, and blood. The challenge lies in the fact that our bodies have evolved to keep harmful substances out of our cells, which makes it difficult for medicines to penetrate and deliver their therapeutic effects.

Traditional approaches for genetic medicines involve using viral vectors, which are essentially empty virus shells, and lipid nanoparticles, which are fatty bubbles that encapsulate genetic material. However, these methods have restrictions on their delivery capabilities, with lipid nanoparticles mainly being effective in delivering to specific areas like the liver and eyes.

Moreover, there are limitations on the cargo capacity of these delivery systems. Some genes, particularly those required for treating certain diseases, are too large to fit inside a virus or lipid nanoparticle. For instance, it can be challenging to package the instructions for creating CRISPR tools into a usable lipid nanoparticle due to size constraints.

As a result, researchers are continuously working to overcome these challenges and develop more advanced and efficient delivery systems to extend the reach of genetic therapies and treat a broader range of diseases effectively.

How will Aera's New Protein Nanoparticle be helpful?

Aera Therapeutics is capitalizing on a recent breakthrough related to a class of human proteins that have origins as relics of ancient viruses that infected humans in the distant past. One of these proteins was found to assemble into a virus-like protein shell capable of storing RNA necessary for self-replication.

Taking inspiration from this discovery, Feng Zhang, a scientist from the Massachusetts Institute of Technology (MIT), recognized the potential to leverage this system for delivering specific genetic material. Zhang's lab conducted experiments on the human genome to identify other proteins that could form protective shells and investigated their potential for RNA transfer. In 2021, they successfully demonstrated that one of these proteins, known as PEG10, could be repurposed for delivering gene-editing tools.

This groundbreaking work laid the foundation for Aera Therapeutics, which is now leveraging this knowledge to develop advanced genetic therapies using the protein nanoparticle technology based on PEG10. By harnessing these naturally occurring proteins with the ability to transfer genetic material, Aera aims to revolutionize the field of gene therapy and make significant strides in treating various diseases.

Way Forward:

If Aera Therapeutics achieves its goals, the capsid packages based on these self-assembling proteins could potentially deliver gene therapies to various locations within the body.

Researchers have identified around 50 of these proteins so far, and they believe there may be more waiting to be discovered. The capsids formed by these proteins come in different sizes, making them suitable for different purposes, such as crossing the blood-brain barrier or accommodating larger genetic material.

Given the advancements in protein engineering, Aera's scientists could potentially tailor the capsids to target specific organs or tissues effectively. Currently, the company's primary focus is on reaching the brain, heart, and muscles, as these areas hold great promise for the application of genetic therapies, opening up new possibilities in medicine.

An advantage of using these proteins is that they are naturally present in our bodies, which might reduce the risk of triggering an immune response. However, the technology is still several years away from being ready for use as an actual drug.

Regardless of whether Aera Therapeutics succeeds in turning this technology into something commercially viable, the biotech industry should take note of the potential and possibilities that this research offers for advancing the field of gene therapy and revolutionizing the treatment of various diseases.

Conclusion:

Absolutely, the field of genetic engineering has seen significant progress over the past two decades, but there is still much room for innovation and breakthroughs. The discovery and potential application of these self-assembling proteins by Aera Therapeutics represent a fresh and exciting approach to gene therapy. It offers the possibility of delivering genetic medicines to previously challenging locations in the body and opens up new avenues for treating various genetic diseases.

As we continue to advance in our understanding of genetics and molecular biology, novel technologies like this have the potential to bring about a revolution in genetic engineering. The millions of individuals living with genetic diseases eagerly await transformative treatments, and innovative research like Aera's could pave the way for more effective, targeted, and safer therapies.

By challenging traditional methods and pursuing fresh approaches, researchers can usher in a new era of genetic medicine, bringing hope and improved quality of life to countless patients worldwide.

Source – Livemint