How Do I Invest In The Gene Therapy And Genetic Engineering Sector? – Gene therapy is the use of gene editing technology to repair, replace or correct the body. The first approved study of gene therapy was conducted by the National Institutes of Health (NIH) in 1989 and provided the first evidence that human cells could be genetically modified without harming patients. To date, approximately 2,600 clinical trials and six gene therapy products have been approved in different countries.
Although most of the current studies are still in phase I or phase II (about 77% of the total), there are more than 100 studies worldwide that are in phase III or IV. This suggests that there may be dozens of gene therapy product approvals in the coming years. Last year was a landmark year for gene therapy with two leading chimeric antigen receptor T cell (CAR T) therapies: Novartis’ Kymriah
How Do I Invest In The Gene Therapy And Genetic Engineering Sector?
Given the impressive recovery rates and recent regulatory approvals, it’s no surprise that the gene therapy market has also grown tremendously, with $7.5 billion raised by advanced stem cell and gene therapy companies in 2017.
Cell And Gene Therapy: The Next Frontier In Pharmaceutical Services
Cell and gene therapy is being touted as the future of medicine, and it is revolutionizing the current healthcare paradigm, from patient care to the logistics of drug delivery.
For gene therapies to work, genetic material must be inserted into cells to treat diseases, which is most effectively achieved using a vector delivery system. Viruses are good vectors for carrying genetic material because they have evolved to deliver just that—deliver genes by infecting cells. The viral vectors used in gene therapy have been modified so that they do not cause infectious disease to the patient. The viral vectors most commonly used in gene therapy are retroviruses, adenoviruses, adeno-associated virus (AAV) and lentiviruses. Although non-viral approaches to gene therapy delivery are being explored, viral vectors remain the most popular approach, with two-thirds of clinical trials to date being delivered via viral vector.
Gene therapies based on CAR T cells have been able to achieve incredible remission rates and have shown success where other treatments have failed.
Gene therapies can be delivered by injecting the vector directly into the patient (in vivo), or the vector can be delivered into specific cells collected from the patient’s blood or tissue (ex vivo). The vector is introduced into the patient’s cells by a method called transduction. Cells modified by ex vivo methods are then grown in a cell culture before being re-injected into the patient.
We Need To Ground Truth Assumptions About Gene Therapy
Non-viral approaches offer many advantages, including greater gene delivery, simpler production, and fewer biosafety concerns. However, they have been shown to be less effective in delivering genetic material, and in some cases the therapeutic benefits are short-lived. Recent developments in non-viral methods increase the interest of this approach.
Perhaps the most talked about gene therapies are cell-based gene therapies for cancer immunotherapy called CAR-T. Novartis’ Kymriah
Examples of this type are cell-based gene therapy. These cell-based gene therapies have been able to achieve incredible remission rates and have shown success where other treatments have failed. Cancer is by far the largest category of indications under investigation, as 65% of clinical gene therapy trials are in this area. The second most popular symptom category is hereditary monogenetic disease with 11.1%, followed by infectious diseases (7%) and heart diseases (6.9%) after the first four symptoms.
Due to clinical success and increased investment in the market, many gene therapy companies are looking to develop and commercialize their lead drugs. Since the commercialization of gene therapy is relatively new – with only six approved products worldwide – there are still several issues to consider when it comes to how to sustainably manufacture these products at scale and deliver them to patients. This task is complicated by the fact that until recently it was not entirely possible to develop production technologies designed solely with gene therapy in mind. Furthermore, there is no “one size fits all” approach, as gene therapy products are complex and can be produced in many different ways using different vectors and cell lines. Finally, ex vivo treatments in which harvested patient cells are genetically modified may also include a cell culture component. These autologous therapies, which use the patient’s own cells, increase the complexity of the preparation. The logistics of collecting and distributing cells to and from patients can be difficult and often require tight lead times. And of course, all this must be achieved at the lowest possible cost, while still maintaining strict quality standards to ensure patient safety and regulatory reliability.
Spark Therapeutics To Invest $575m In New 500k Square Foot State Of The Art Gene Therapy Innovation Center On Drexel’s University City Campus
Gene therapy companies should also assess whether their current intellectual property portfolio gives them enough freedom to work in commercial production. IP visibility around critical process elements, including unit operations, must be understood and evaluated as soon as possible. Developers can then identify obstacles and find alternatives early in the development process. This saves developers time and money.
It sounds scary, but there are valuable resources, and with the 2017 approvals, a final and successful regulatory and manufacturing path has been opened for other companies. In this article, we have combined many years of experience with experts in various fields of gene therapy to create a list of key ideas and best practices.
In order to make therapeutic products available to the public, products must demonstrate efficacy and safety. With biologic gene therapies, regulatory authorities require sustained effectiveness and long-term safety. Data demonstrating sustained effectiveness will convince payers and providers that these potentially more expensive treatments are better options for patients than traditional treatments. Payers are likely to want to see data outside of clinical trials to justify long-term product coverage.
For gene therapies that provide little improvement over the standard of care, payers expect the price to be commensurate with the level of improvement. For gene therapies that offer a transformative improvement over a standard treatment, such as a cure, payers want to see long-term data with a large group of patients to justify the high initial costs and overall economic benefit of these treatments.
History Of Gene Therapy
Gene therapy production is an important part of whether or not gene therapy is successfully commercialized. Can the product be produced in such a quantity and quality that it meets the demand? Should it be made at a price that is affordable for patients? These are just some of the questions that need to be addressed.
It is important that stakeholders engage in process development and manufacturing programs as early as possible in the development phase to keep up with the rapid movement of gene-based products into the clinical landscape. A key balance must be struck between investing too early in manufacturing technology before the product is fully characterized and the risk that the manufacturing process will not yield the correct product. Conversely, investing too late means trying to build a process that may not meet needs, which can be very expensive and risky.
Gene therapy companies also need to understand what regulatory avenues are available to them based on whether their product addresses an unmet need or a serious, life-threatening condition. Thus, there are several shortcuts that may be available to gene therapy developers. If a product is introduced on a fast schedule, this will affect production schedules and therefore must be considered in process design, growth, quality and continuous inspection.
Viral vector systems are by far the most widely used methods for delivering therapeutic gene products due to their infectious nature and ability to deliver specific genes into a cell. In general, therapeutic DNA is delivered using viral vector systems based on adeno-associated viruses (AAV), lentivirus (LV) and adenoviruses (Ad). Among these, AAV vectors are the most commonly used delivery vehicles in gene therapy indications. According to market experts, AAV is used in almost 50 percent of the 483 gene therapy trials currently underway.
Gene Therapy: Behind The Scenes
However, the main obstacle to translating promising studies through clinical evaluation to bring these drugs to the market is the demand for the large numbers of viral vectors needed for this. Point vector production is one of the biggest challenges facing the industry today. Key elements in the development of large and optimized production are harvesting and cleaning strategies as well as analytical tools to monitor quality characteristics to ensure a safe and effective product. For the purpose of this article, we will focus on the production of viral vectors.
Many pre-clinical processes for making viruses are currently based on academic protocols, where scale and quality are paramount. It is important to determine whether the current process is scalable for clinical and commercial production and whether quality requirements can be met. Gene therapy developers may have varying perceptions of how mature their manufacturing processes actually are. Good manufacturing practice (GMP) requirements apply to raw materials, cell substrates and process consumables