At its core, biotechnology is harnessing biological and molecular systems to develop products and processes. There are three fields of biotech: agricultural biotechnology, which includes the development of genetically modified crops; industrial biotechnology, which includes the production of chemicals, paper and textiles; and medical biotechnology. So what is biotechnology in business and how has it developed?
Advances in AI and Machine Learning significantly lowers the cost of developing and testing new biotech products.
Since the human genome was first sequenced in 2003, the cost of sequencing has come down dramatically and our knowledge of what the sequence means has increased significantly.
This has helped drive advances and investment in biotechnology fields such as pharmacogenomics which uses information in a person’s genes to inform how they will react to different drugs and therapeutics as well as gene therapy and immuno-oncology.
Why does biotechnology matter to business?
In the biotech sector, companies that make the most effective use of the modern data tools are going to be successful while, in the long run, big pharma could be threatened by patent expiration and rising competition unless they invest.
What are the main themes around biotechnology?
The rapid evolution of big data technologies, including AI and Machine Learning, will have a major impact on the biotechnology sector; the need to process and analyze the vast volumes of data generated both in the research phase, and subsequently in the application phase means that the effective exploitation of advances in data science will become an increasingly significant differentiator.
Cloud computing offers small firms the ability to get access to computing power at a scale that was, until relatively recently, only available to the largest organizations. Cloud computing allows small organizations to compete with much larger competitors where it comes to the processing and analysis of data on a large scale.
Robotics and 3D Printing
The building of automated manufacturing systems and the creation of novel devices and machines has been made easier, quicker, and less expensive thanks to innovations in the fields of robotics and 3D Printing. Indeed, 3D printing is already being directly applied to biotech in the form of bio-printing human tissue and human organs.
Immuno-oncology (IO) aims to harness the body’s own immune system to target cancer. These therapies have the potential to revolutionize the way that we treat cancer and aim to provide long-lasting survival for patients, by training the immune system to recognize and kill cancer, even after remission.
The field of immuno-oncology involves the development of therapies that can harness the body’s ability to generate and sustain, naturally or artificially, an effective immune response against cancer. The majority of these mechanisms revolve around priming and boosting the immune system via antigen-presenting cell (APC), T-cell, or innate cell stimulation, reducing immunosuppression in the tumour environment by regulating inhibitory pathways, and enhancing adaptive or innate immunity. Such active immunotherapies contrast with passive strategies consisting of the administration of immune system components helping to provide a temporary antitumoral effect.
Active immunotherapy stimulates the immune system by presenting antigens in a way that triggers an immune response, while passive immunotherapies attack a tumour directly, and do not directly engage the patient’s immune system to target a specific antigen. Immuno-oncology products are active immunotherapies. There are four main types:
- Checkpoint inhibitors
- Cancer vaccines
- Oncolytic viruses
- CAR-T cell therapy
Checkpoint inhibitors are antibodies that block proteins that prevent the immune system attacking cancer cells.
Normally, when cancerous cells develop or your body has an infection, the immune system, particularly a type of cell called T-cells, recognizes it using proteins on the cell surface and kills the infectious agent or cancerous cell.
There are also proteins that help to turn the immune response off, for example, once an infection has been cleared, to prevent the immune system from attacking healthy cells. The proteins that turn on and off the immune response are known as checkpoint proteins.
Cancer cells can combat the immune response by activating inhibitory proteins, meaning that the T-cells no longer attack and kill the cancer cells.
Checkpoint inhibitor drugs were first approved for treatment of patients with metastatic melanoma (a type of skin cancer), but they are now approved for use in many other cancers including non-small cell lung cancer, renal cell carcinoma, head and neck squamous cell carcinoma and Hodgkin’s lymphoma.
Cancer vaccines are not the same as vaccines that are used to prevent disease.
Cancer vaccines are also known as therapeutic vaccines and they work by boosting the body’s immune system to fight cancer.
The vaccines are designed to recognize specific proteins on the surface of cancer cells which help the immune system to recognize and attack the cancer cells.
Currently, there is only one therapeutic cancer vaccine approved by the FDA (Food and Drug Administration, the US federal agency responsible for approving therapeutic drugs): Valeant Pharmaceutical’s Provenge for metastatic prostate cancer.
However, there are many types of cancer vaccines that are being investigated in clinical trials, with particular promise when combined with other types of cancer immunotherapies.
Oncolytic viruses are a type of virus that selectively infect and destroy cancer cells but leave healthy cells intact.
These have advantages over traditional cancer treatments such as chemotherapy as they are more selective for cancerous cells than these therapies.
Oncolytic viruses occur naturally, but they can also be made in the laboratory by modifying other viruses.
As well as directly killing cancer cells, oncolytic viruses have now been suggested to also stimulate the immune system and recruit it to kill cancer cells.
This is because when a virus infects a tumour cell, it hijacks the cell’s machinery to replicate the virus many times and, eventually, the cell bursts open.
This releases the viruses inside that are then able to infect more cells, but it also releases cell contents. These contents include tumour antigens which can be recognized by the immune system, alerting them to the presence of a tumour.
CAR-T cell therapy
CAR-T cell therapy uses a patients’ own immune cells to treat their cancer.
It is a type of immunotherapy called adoptive cell transfer, of which there are several types, but CAR-T cell therapy has been the most successful in clinical development so far.
The cancers that have seen the most success with regards to CAR-T cell therapy have been largely blood cancers such as acute lymphoblastic leukaemia (ALL) and, in 2017, the FDA approved a CAR-T cell therapy for children with ALL called Kymriah, as well as one for adults with advanced lymphomas. Kymriah (manufactured by Novartis) has had very promising results with 81% cancer remission of patients for whom other treatments have not worked.
Despite these promising results, CAR-T cell therapy is not without its problems. Firstly, the currently approved CAR-T cell therapies come at a huge cost (Kymriah costs $475,000 per patient) and at present, the range of cancers that can be targeted by CAR-T cell therapy is small, limited largely to blood cancers. As with other current cancer drugs and treatments, CAR-T therapy can cause side effects, the most frequent being cytokine release syndrome. Cytokines are small signalling molecules that are released by T-cells and help to stimulate and direct the immune response. During cytokine release syndrome these molecules are released very rapidly and in huge numbers, which can result in high fever, low blood pressure and difficulty breathing. The biotech industry is currently trying to find ways to combat and solve these issues.
Researchers are also now expanding CAR-T research into looking at targeting solid tumours. This is difficult for two reasons: one is that identifying unique antigens expressed on the surface of solid tumours has been challenging, and the second is that solid tumours have a microenvironment surrounding them that is designed to mute the immune response.
This article was produced in association with GlobalData Thematic research. More details here about how to access in-depth reports and detailed thematic scorecard rankings.