Gene Editing sounds like something from a sci-fi movie, however, it is available in real-time and is making waves. This past month, a Massachusetts-based company, Beam Therapeutics, announced a breakthrough in its gene editing trials with human DNA.
The company reported it had seen positive results in its clinical trials for a rare genetic disorder which affects the liver and lungs. Through gene editing, Beam Therapeutics has been able to correct this mutation at a DNA level.
This incredible breakthrough is significant as it highlights the many possibilities of intervention in human DNA-correlated disorders and conditions.
The New York Times praised the breakthrough, with Dr. Kiran Musunuru, a gene therapy researcher from the University of Pennsylvania Perelman School of Medicine quoted saying: “This is the beginning of a new era in medicine.”
DNA-level Intervention
This specific clinical trial by Beam Therapeutics involved nine patients affected by the condition. All were suffering from alpha-1 antitrypsin (AATD) deficiency. This can lead to severe liver and lung complications.
The treatment administered to them was called Beam-302, and the patients were given a single infusion of the intravenous therapy, with no adverse side effects. Beam Therapeutics is now planning to evaluate the therapy at even higher doses.
The Beam Therapeutics website defines ‘base editing’ as ‘an emerging class of investigational precision genetic medicines designed to overcome the limitations of existing approaches and expand the potential of genetic medicine’.
But what is genetic medicine and gene editing?
Gene editing is medicine at its finest, most precise application. To explain, it allows for scientists to literally change an organism’s DNA. Much as editing a piece of writing involves taking out words and transforming sentences, gene editing involves altering, adding, and removing genetic material. The hyper-personalized, super-focused nature of the intervention means that precise modifications to the genetic make-up of living organisations.
One of the methods used in gene editing is called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). This powerful tool involves making precise changes to the DNA of living organisms.
CRISPR is a natural system originally discovered in bacteria, where it helps them defend against viruses. Scientists adapted this into a biotech tool which allows researchers to target and modify specific DNA sequences in plants, animals, and even humans.
It is usually used in combination with a protein called Cas9, which acts like molecular scissors to cut DNA at specific points.
Researches Anticipate Breakthroughs Across Various Conditions
Genetic editing has various applications and is currently being explored as a treatment for sickle cell anemia and muscular dystrophy.
It has also been considered as a treatment include certain neurological disorders; among these Huntington’s Disease, Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s Disease (targeting associated risk genes like APOE4) and Epilepsy (genetic subtypes).
Gene editing for the treatment of inherited eye diseases is also undergoing clinical trials. One of these eye diseases is Leber Congenital Amaurosis (LCA), which is the cause of childhood blindness. Trials focused on the treatment for this condition have used CRISPR injected directly into the eye to correct mutations of the CEP290 gene.
Gene editing has also been used to enhance immune cells (like T cells), for the purposes of attacking cancerous tumours. CRISPR-modified CAR-T cell therapies for the treatment of Leukemia and Lymphoma are currently in clinical trials.
Other groundbreaking clinical trials are underway for the treatment of HIV. Gene editing is being explored as a means to knock out the CCR5 gene which the virus uses to enter the immune cells.
Recent statistics show that the gene editing market is forecast to exceed R40 billion by the year 1934. The global gene editing market was valued at $9.3 billion in 2024, with North America leading the CRISPR gene editing market. The Asia-Pacific gene editing market is set for the fastest growth.