A new and exciting type of medical treatment called ‘in vivo cell therapy’ is now being tested for the first time in humans. Unlike traditional cell therapies that are made outside the body and then re-injected into patients, these new therapies work by delivering genetic instructions directly into the patient’s own cells. This is done using a harmless carrier, such as a modified virus or a tiny fat bubble called a lipid nanoparticle (LNP). These carriers act like delivery trucks, transporting the genetic instructions to the cells where they are needed.
The field is moving quickly, with big pharmaceutical companies showing strong interest. Just in the past year, AstraZeneca bought a company called EsoBiotec for $1 billion, Eli Lilly acquired Orna Therapeutics for $2.4 billion, and other major companies like AbbVie and Bristol Myers Squibb also made large purchases. These investments suggest that experts believe this new approach could become a widely used treatment in the future.
Right now, most of these therapies are still in the early stages of testing, with some in preclinical research and others in the first phase of human trials. One example is a new in vivo CAR-T therapy being studied by AstraZeneca. Early results from a small group of patients with multiple myeloma (a type of blood cancer) showed promising results: four out of five patients responded to the treatment, and three achieved complete remission. While these results are encouraging, more testing is needed before these therapies can become widely available.
You might hear these treatments called ‘in vivo cell therapies,’ but experts say they are more accurately described as a type of gene therapy. Unlike gene therapy, which often fixes damaged genes, these new therapies aim to give cells entirely new functions so they can act as treatments themselves. For example, some of these therapies could train immune cells to fight cancer more effectively.
One of the biggest advantages of in vivo therapies is that they could simplify the entire treatment process. Traditional cell therapies require collecting a patient’s cells, modifying them in a lab, and then returning them to the patient—a process that is complex, expensive, and time-consuming. In contrast, in vivo therapies could work by delivering the genetic instructions directly into the patient’s body, turning their own cells into the treatment. This could make therapies more accessible and affordable for patients.
However, there are still challenges to overcome. For example, ensuring the right dose is delivered to the correct cells without causing harmful side effects such as immune reactions is a major hurdle. Different types of carriers, like viruses or LNPs, have their own strengths and weaknesses. Viruses can permanently alter cells but may also cause unwanted reactions, while LNPs can be easier to control but may target the wrong cells or trigger immune responses.
Some companies are finding creative solutions to these problems. For example, one company called Lentitek has developed a way to prevent genetic instructions from being accidentally cut or damaged during the manufacturing process. Another company, Lupagen, has created a system that allows for the modification of cells outside the body in a controlled way before returning them to the patient, blending aspects of both in vivo and traditional cell therapies.
Experts believe that in vivo therapies could revolutionize medicine by making treatments more widely available and easier to produce. However, they also note that these therapies are unlikely to replace traditional cell therapies entirely. Instead, both types of treatments will likely play important roles in the future of medicine. The next year will be critical as more data from clinical trials becomes available, helping us better understand the potential and limitations of these groundbreaking treatments.