On November 22, 2022, the Food and Drug Administration (FDA) approved the first gene therapy of its type for individuals diagnosed with hemophilia B. This inherited disorder causes people not to produce enough of a protein that causes blood clotting.
In the past, the standard treatment was a time-consuming infusion of this protein. Even with regular visits, people could still suffer from a potentially life-threatening bleeding incident.
CSL Behring developed a gene therapy that uses a modified virus to deliver a copy of the missing protein to the liver so that the body starts making more of it.
Researchers have found in its clinical trials that a single infusion of this new treatment method can protect patients for at least eight years.
Advancements in Biotechnologies Are Creating Numerous Treatments
Precision genomics creates opportunities for companies to develop precision medicines and new delivery methods. ReCode is working on non-viral options that could one day send corrective genes to the spleen, lungs, or central nervous system.
ReCode plans to start safety trials on its approach with individuals diagnosed with cystic fibrosis and primary ciliary dyskinesia in 2023. [1]
One of the primary reasons these genetic advancements are available in biotech is the decreased cost of mapping the human genome. The arrival of ultra-rapid sequencing allows a sick patient to arrive at a hospital and receive a diagnosis of a rare condition in as little as five hours for just $100. [2]
Although the potential for this technology is massive, the immediate effect is that cancer patients can have individual tumor cells sequenced to determine their most effective treatment options.
Transplant Technology Breakthroughs That Happened in 2022
The first successful transplant patient receiving a genetically modified pig heart survived for two months. It was the only option for the individual diagnosed with terminal heart failure. The treatment team reviewed the autopsy for signs of organ rejection and found none. [3]
His recovery was so profound that he went from being bedridden on a heart-lung bypass machine to signing “America the Beautiful” while watching the Super Bowl.
“We are grateful for every innovative moment, every crazy dream, every sleepless night that went into this historic effort,” said David Bennett, Jr., the patient’s son. “We hope this story can be the beginning of hope and not the end.” [4]
This science is called xenotransplantation. With more than 100,000 people in the United States and thousands more globally needing a kidney or a heart, more porcine organ modification studies are expected to bring advancements in 2023.
“In the coming decades, it is likely we will see more internet-like revolutions in which technology steals a march on politics,” writes Yuval Noah Harai. “AI and biotechnology might soon overhaul our societies and economies, and our bodies and minds, but they are hardly a blip on our political radar.” [5]
In the past, xenotransplantation largely failed because the human body rapidly rejects the animal organ. With gene editing, scientists add human genes while removing the ones that trigger a hyper-rejection to help with the acceptance process.
Base Editing Technology Offers Hope
Base editing technology breakthroughs are creating cures for previously incurable diagnoses. A teenager from Leicester was diagnosed with T-cell acute lymphoblastic leukemia in 2021, treated with all conventional therapies, and nothing worked. She was given a terminal diagnosis.
She became the first patient enrolled in a new clinical trial focusing on base editing, a technology invented six years ago. The teen’s treatment team used the tool to engineer a new T-cell that could hunt and kill the cancerous cells in her body. After starting with a healthy donor, scientists made three edits. [6]
The first was to disable the targeting mechanism so that they wouldn’t attack the child’s body. Then a chemical marking, removed before an “invisibility cloak”, was given to them to prevent chemotherapy drugs from killing them.
In just six months, the teen’s incurable cancer became undetectable. [7]
Biotech Is Just Beginning to Explore This New Medical Field
Looking back at 2022, it could be the year when future generations start discussing the beginning of the biotech revolution. We’ve seen some of the first glances that an incredible new medical era could offer.
We are looking at the rewriting of the different genetic codes that life offers to treat and cure incurable diseases.
Although time is necessary to maximize the impact of this current research, we will continue building on the early successes with innovative approaches that hopefully evolve into a better standard of care for everyone.
[1] https://www.businesswire.com/news/home/20230110005385/en/ReCode-Therapeutics-Announces-Strategic-Investment-from-the-Cystic-Fibrosis-Foundation-to-Accelerate-Development-of-Novel-mRNA-Therapy ; [2] https://www.science.org/content/article/100-genome-new-dna-sequencers-could-be-game-changer-biology-medicine ; [3] https://www.umms.org/ummc/news/2022/transplant-of-genetically-modified-pig-heart-into-human-patient ; [4]https://www.npr.org/2022/03/09/1085420836/pig-heart-transplant ; [5] https://www.ynharari.com ; [6] https://www.ukri.org/news/world-first-use-of-base-edited-cells-to-treat-incurable-leukaemia/ ; [7] https://www.bbc.com/news/health-63859184
