Oscar Segurado, MD, Ph.D., Chief Medical Officer at ASC Therapeutics, focuses on clinical development of gene therapies for rare diseases.

We are at the dawn of synthetic medicine, which will forever change how doctors and patients approach healthcare. In fact, simply understanding the basics of synthetic medicine could help many doctors and patients make informed medical decisions.

With this in mind, this article will look at the ways that synthetic medicine could transform healthcare and how those in the biopharmaceutical and healthcare industries can help ensure that it reaches its potential.

Synthesizing Data From Digital Sources

Since any disease is a dynamic process, it is crucial to continuously track all variables contributing to the severity and resolution of the condition. Historically, tracking an individual’s vital signs requires them to be in a medical facility.

With synthetic medicine, healthcare synthesizes the profile of a patient and disease based on digital information collected from any individual at any location through mobile devices and wearable sensors. This approach, integrated with therapeutic interventions and other datasets, can help doctors to precisely monitor and even predict a patient’s disease trajectory.

Along with data collection, the use of machine learning and artificial intelligence will be crucial to identify patterns that are simultaneously unique to a patient while providing a helpful profile to help doctors apply to future advances in medicine.

In our all-encompassing digital world, medicine at the practice and academic levels is leveraging these powerful digital tools to incorporate the collection, assimilation, interpretation and dissemination of data. This digitalization of information may be especially beneficial to the way patients interact with doctors and how doctors interact with each other. This is digital medicine.

Synthesizing Data Leading To Precision Medicine

Modern medicine relies on the collection, synthesis and interpretation of millions of data points obtained from patients and healthy individuals. Health and disease can be generalized, standardized and quantified. This vast amount of data is structured at the individual and disease level, incorporating demographics, clinical and biological data.

These biological markers of disease, so-called biomarkers, can be obtained from imaging, as well as from biological proteins that are circulating in the bloodstream or present in tissues or organs. In a typical medical visit, the doctor will consider a patient’s description of signs and symptoms, conduct a physical examination and review imaging and laboratory tests to decide on the next steps.

In the future, personal and clinical data will be anonymized and incorporated into a data repository, potentially a digital avatar, that will precisely guide doctors to prescribe the right medical intervention and optimize decision-making for patients with similar conditions. This is precision medicine.

Synthesize Genes Leading To Functional Cures

Once the genomic code was unraveled, scientists rapidly learned how to synthesize DNA or RNA sequences from human or non-human genes. This remarkable feat forms the basis for several gene therapies that can provide a “functional cure” for hard-to-treat or incurable diseases.

In the space of hereditary neurodegenerative disorders, there is an approved gene therapy in the U.S., EU and several other countries based on the synthesis of a defective gene, which is delivered to patients with spinal muscular atrophy. These patients would usually die within the first few years of their lives, but most treated patients can reach up to 10 years with normal physical and mental development after this gene therapy.

In the space of hereditary coagulation disorders, there is a gene therapy approved in the EU and under review in the U.S based on the replacement of the defective gene responsible for hemophilia A, a severe and lifelong condition driven by the absence of a factor essential for the coagulation of the blood. These patients can now avoid repeated visits to doctors and hospitals to receive replacement therapy for their missing coagulation factor.

In both examples above, a biological transporter, a harmless virus, was also synthesized to take the missing gene to the neurons (spinal muscular atrophy) or to the liver (hemophilia A), which become biofactories synthesizing indefinitely the missing genes. This is synthetic biology.

Synthesizing A Future Of Personalized Healthcare

The combination of digital advances, precision medicine and synthetic biology are the key drivers of patient-centric and personalized medicine. Having data continuously updated and contextualized with attributes from similar patients and population health analytics is revolutionizing medicine.

The patient will be at the center of all activities by healthcare providers and institutions, and they will be empowered to know about their disease and to become part of the healthcare solution. The patient will realize that prevention and incorporating individualized guidelines for physical and mental health are crucial elements of well-being.

But high tech shouldn’t replace high touch. The patient needs a personal relationship with the doctor. This is personalized medicine.

With millions of data points collected from patients in clinical studies and clinical care, the biopharmaceutical industry can further the adoption of synthetic medicine by focusing on patient-centricity and decentralization. Patients should be considered partners, not just “study subjects.” They should be actively incorporated into clinical development, including study designs. Programs should also collate input from several angles and perspectives, not just from “centers of excellence.” Any medical facility should be able to contribute data, thus providing a decentralized approach to the clinical development of novel therapies.

Patients, healthcare providers and the biopharmaceutical industry are synthesizing a better future—let’s embrace it.