For decades, medical science has been foraging for permanent cures for a large array of diseases. During the last decade, great strides have been made in the field of genetic technologies, which might have the potential to fundamentally disrupt the human evolutionary process and bring forth unprecedented benefits to the human race. Simply put, science fiction is on its way to becoming a reality.
Gene editing is one such emerging technology that is mounting enormous curiosity in the medical world through its ability to alter and modify the DNA of a species for a specific purpose. In simple words, gene editing is similar to how an author edits or corrects a book or how an artist amends an art piece. The technology is not new to the world; it is already being used in agriculture to create genetically-modified animals and plants.
Gene editing in humans is deemed to have the potential to deliver permanent cures, in potentially one-time treatments, to a myriad of diseases across various therapeutic areas. This includes chronic diseases, genetic diseases, neurology, and oncology, which have limited cures or do not currently have any cure at all.
However, as of 2022, no gene editing treatments have been approved by the FDA for commercial purposes, but more than 45 patients (as of December 2021) with sickle cell disease and beta-thalassemia have been treated in experimental clinical trials, indicating the treatment’s potential. More gene editing treatments are underway in Phase I and II clinical trials and can be expected to enter the clinic in the next few years.
The majority of companies in gene editing are focused on research and development of novel CRISPR/Cas9-based treatments, due to the technology’s simplicity, versatility, and cost-effectiveness. Although low in number, a number of drug-developing companies, including several early-stage disruptors, are leveraging other older yet evolving technologies such as TALEN and ZFN owing to fewer observed off-target effects and greater efficiency (in certain gene applications such as heterochromatin—the gene responsible for Fragile X syndrome and sickle cell anemia) compared to CRISPR.
On the other hand, gene editing SaaS platforms remain the smallest segment, with a relatively low number of startups in the space due to most companies choosing to use internally developed gene editing platforms and technologies at present. However, demand for these platforms could increase as gene editing becomes commercialized, and more pharma and drug developers plan to enter the space quickly, using pre-built gene editing platforms.
Most companies across the space fall under the seed stage, with only a limited number of startups in the early stage. This reflects the industry’s current nascent stage, with no FDA-approved, commercially available treatments; most companies are either in the preclinical or early clinical trial stages.
The incumbent involvement in the industry is predominantly focused on the CRISPR/Cas9 segment, which includes prominent big pharma companies such as Merck, AstraZeneca, and Bayer.
Major big pharma companies such as AstraZeneca, Pfizer, Bayer, Eli Lilly, and Bristol-Myers Squibb are involved in the CRISPR/Cas9 drug developers segment owing to the high potential and increasing popularity of the technology.
The most common method used by incumbents to enter the gene editing space has been through partnerships and research collaborations with startup companies. Although the number of incumbents with in-house gene editing capabilities is low, a few prominent big pharma incumbents such as Novartis, Merck, and AstraZeneca have initiated in-house R&D initiatives over the last few years in order to stay at the forefront of gene editing.