Stunning breakthroughs in biotechnology are transforming healthcare at an unimaginable speed. Remarkable technologies like CRISPR-Cas9 offer hope for treating incurable conditions, regenerative medicine may soon enable printing organs for transplantation, and that is just the beginning. - BY Karen White

Significant events in the biotech industry are demonstrating remarkable new ways to approach medicine and healthcare, tackling problems that seemed insolvable not so long ago and enhancing patient outcomes. Technically, the biotech revolution began in the 1990s with the development of technologies like recombinant DNA technology that allows gene manipulation and genome sequencing, which provided the groundwork for personalized medicine. With advanced technologies, including artificial intelligence (AI), medical researchers are using the foundation of previous discoveries and making leaps and bounds in areas like regenerative medicine, CRISPR-Cas9, and gene editing to treat specific disorders like sickle cell disease and vaccine development.

Regenerative Medicine: Closing Gaps in Natural Human Repair Processes

Regenerative medicine focuses on repairing, replacing, regenerating, or engineering human cells, tissues, or organs to restore or establish normal function. The goal is to heal or replace human organs and tissues damaged by injury, disease, or defect through tissue engineering that enhances the natural repair processes. Regenerative medicine harnesses the body’s natural healing process or creates organs and tissues in a laboratory for transplantation.

This field of medicine embraces several approaches. Stem cell therapy utilizes the ability of stem cells to differentiate into different types of cells. Embryonic and adult stem cells have been used in therapy, but adult stem cells are limited as to their differentiation potential. Induced Pluripotent Stem Cells (iPSCs) return adult stem cells back to embryonic stem cells. Breakthroughs in applying iPSCs technology are being used in disease modeling, gene therapy, drug discovery, and cell replacement therapy.

Stem cell therapy is only one area of regenerative medicine. It also includes research in gene therapy. A recent breakthrough was the use of a novel gene therapy approach that gave five children born deaf the ability to hear. The 10 FDA-approved gene therapies to date are expected to change lives, but the expectation is that 30-50 additional therpaies will be approved by 2030. They are used in different ways, including treating rare diseases. For example, the Utah Program for Inherited Neuromuscular Disorders is using gene therapy to treat infants before they are three weeks old for Spinal Muscular Atrophy, once a leading cause of death, and is conducting a pilot study on using gene therapy to correct mutations in the gene that cause collagen VI related muscular dystrophy.

Regenerative medicine uses cellular therapies that transplant cells to treat damaged tissues due to leukemia or repair damaged cartilage. Another breakthrough is engineering substances that interact with biological systems for medical purposes. Scaffolds are structures made from biomaterials that protect fibroblasts and adult stem cells and promote the migration of the cells into areas of damaged tissues, where they proliferate to form new tissues. Technologies such as nanoparticles have advanced this area of biotechnology.

An exciting recent focus is 3D bioprinting, in which complex tissue structures are created in layers. It involves computer-aided design printing of a patient’s living cells and biomaterials to build tissue-like structures that can one day be used for organ transplants. This process is used for 3D bioprinting various tissues and organs like the heart, blood vessels, bone, skin, cornea, and more.

Gene Editing and CRISPR-Cas are Saving Lives

Gene editing, also called genome editing, is a set of technologies that lets scientists change DNA. Genetic material can be removed, added, or altered. One of the breakthrough gene editing technologies is CRISPR-Cas9. It was adapted from the naturally occurring genome editing system involved in the immune system and virus infections. It is a complex process that is becoming important in preventing and treating human disease. Casgevy is the first FDA-approved therapy using CRISPR-Cas9 to treat sickle cell anemia in patients over 12 years old who are experiencing recurring organ damaging and painful events. Blood stem cells are modified using CRISPR-Cas9 technology, which cuts DNA in the cells of targeted areas and enables DNA editing. The edited blood cells are transplanted back into the patient, where they attach and multiply within the bone marrow. This increases the production of a type of hemoglobin that drives oxygen delivery and prevents the sickling of red blood cells. Clinical trials are underway to test gene editing as a therapy for other conditions and diseases. They include other blood disorders, type 1 diabetes, cancer, and HIV/AIDS.

Putting New Vaccines to Work

Biopharmaceuticals came into the limelight during the pandemic. The recent advent of mRNA technology led to the development of a new class of vaccines, including those used to fight COVID-19. The mRNA vaccines use the body’s cellular activity to produce a viral protein. The immune system is trained to recognize and fight the virus. Of importance is the fact vaccine development has a much more compressed timeline so that they can be manufactured quickly in response to new viruses. The frontier in vaccine development is nanotechnology, with small particles engineered to carry vaccine antigens directly to immune cells. Novel vaccines that use innovative approaches, like DNA vaccines, to inject DNA-encoded vaccine antigens into the body to stimulate the immune system, are also being developed. Researchers are also working on plant-based vaccines and viral-like particle vaccines.

Enter Artificial Intelligence

AI touches most industries today, including biotechnology. Pharmaceutical company professionals feel AI will help put more data to work through resource sharing. Multiomics will advance life sciences by supporting high-cell number multiomics assays targeting a combination of RNA, DNA, and proteins. Niven R. Narain, President and CEO of BPGBio, said, “…the emphasis is shifting from the mere use of AI to the substantive impact it has in propelling the discovery and development of groundbreaking pharmaceuticals and diagnostics…” There is a belief AI will support the development of blood based biomarkers and genetic screening for better diagnosis and treatment of early-stage Alzheimer’s Disease. These are just a few examples of how AI and machine learning are transforming biopharmaceuticals. AI also supports personalized medicine, like integrating genomic data with clinical information for personalized treatment plans. AI is being integrated into wearable technology. AI algorithms are used in bioprocess optimization, molecular modeling, and predictive analytics to assess the efficacy and safety of drug candidates and identify suitable candidates for clinical trials. The list could go on, but as AI technology continues to evolve, its integration with biotechnology promises to drive further innovation and breakthroughs, ultimately improving healthcare, agriculture, and environmental sustainability.

Breathtaking Speed of Change

The latest breakthroughs in biotechnology hold immense potential for transforming medicine and improving human health. Gene editing technologies are paving the way for precise genetic therapies. Regenerative medicine is making strides with stem cell therapies and tissue engineering, offering hope for treating previously incurable conditions. Meanwhile, advancements in vaccine development are revolutionizing our ability to respond to infectious diseases and even cancer. As research progresses, these innovations will lead to more effective treatments and improved patient outcomes worldwide.