What is the microbiome?
The microbiome refers to the trillions of micro-organisms, and their gene products, which colonise the human body. The bacteria which colonise the human gastro-intestinal tract – known as the gut microbiome – play an important role in health and disease.
The gut microbiome is a collection of around 2kg of bacteria and there are more microbial cells in the gut than in the entire human body. The volume of genetic information contained with these bacteria dwarfs that of their host – the gut microbiome is thought to consist of 500x more genes than the human genome.
All of this genetic information has function. Many of these genes are required for primary metabolic functions, survival and interaction with other organisms in the gut. However, crucially, many of them have functions within the human host itself.
How will it impact medicine?
The gut microbiome is commonly understood to influence gastrointestinal diseases such as infectious diarrhoea.
However, gut bacteria can also impact diseases in remote locations of the body, through modulation of the human immune system, metabolism and even neurological function. This is not simply an ecological effect - it is an evolved functionality that allows bacteria to interact with and modulate the systems of the body.
Understanding and leveraging this precise functionality offers a new approach to the treatment of a broad range of diseases, from cancer to asthma and conditions of the CNS.
Live Biotherapeutics are recognized new class of medicines, which have the potential to transform the way in which we treat many diseases.
The US FDA defines a Live Biotherapeutic Product (LBP) as a biological product that:
1) contains live organisms, such as bacteria;
2) is applicable to the prevention, treatment, or cure of a disease or condition of human beings; and
3) is not a vaccine
U.S. Department of Health and Human Services, Food and Drug Administration (FDA), Center for Biologics Evaluation and Research (CBER), 2012 (updated 2016) - Early Clinical Trials with Live Biotherapeutic Products: Chemistry, Manufacturing, and Control Information: Guidance for Industry
4D pharma's LBPs are single strains of gut commensal bacteria which have been originally isolated from healthy human donors. These are grown from cell banks at our in-house cGMP-certified manufacturing facility, encapsulated for oral administration and selective delivery to the gut where they exert their therapeutic effects.
Each strain is selected for its particular functionality relevant to mechanisms of disease, in the same way a small-molecule or biologic is selected to perform a specific role in combating a disease.
A revolutionary new class of drug
The development of Live Biotherapeutics offers a new way to create safe, convenient and effective treatments for patients. It also presents an opportunity to treat diseases that existing therapies are unable to effectively address. Live Biotherapeutics exert their therapeutic effects in a variety of ways. Some act on the same or similar pathways as existing medicines. Others act in novel ways, providing potential solutions for diseases with few current treatment options. The development of Live Biotherapeutics provides an opportunity to treat many diseases in an entirely new way.
Attractive safety profile
Toxicity is a significant obstacle in the development of new medicines. The side effects associated with existing medicines are a concern for both patients and clinicians, and are often the reason for termination of development of a new drug, or can results in sub-optimal treatment regimens or poor patient compliance.
Our Live Biotherapeutics are non-engineered strains of human commensal bacteria, originally isolated from healthy donors. As a result, 4D pharma's LBPs are expected have attractive safety profiles.
This allows us to rapidly accelerate our therapies into the clinic, conducting first-in-human studies in patients to generate clinically relevant data much earlier than with traditional drug types, significantly shortening the drug development timeline. Inherently favorable safety and tolerability also significantly reduces one of the major early clinical development risks for any new drug, de-risking our drug candidates.
Live biotherapeutics interact with human biological systems (immune, metabolic, neuronal, endocrine) through a wide range of mechanisms.
Although host-microbe interactions are typically initiated in the gut, the resulting changes in downstream pathways are diverse and produce systemic activity with effects in distal areas of the body - from tumors, to the lungs, and even the central nervous system (CNS).
4D pharma investigates and characterizes the interactions of specific strains of bacteria with host pathways, including the identification and validation of bacterial effector molecules. Find out more - MicroRx®