Gene Therapy Market | Gene Augmentation | Market Analysis | 2035

1. Gene Therapy Market | Gene Augmentation Market Analysis | 2035 Over the last two decades, there have been several breakthroughs related to the development of gene therapies. In 2020, Libmeldy, an ex vivo gene therapy received approval for the treatment of metachromatic leukodystrophy. Today, there are a variety of gene-focused corrective strategies that have been developed for the treatment of a wide range of clinical conditions caused due to genetic aberrations, such as adenosine deaminase severe combined immunodeficiency (ADA-SCID), β-thalassemia, biallelic RPE65 mutation-associated retinal dystrophy, critical limb ischemia, head and neck squamous cell carcinoma, melanoma, peripheral artery disease and spinal muscular atrophy. Considering that such therapies are designed to address the root cause (at the genetic level) of a disease, a number of contemporary drug / therapy development initiatives are focused on gene correction and genome editing-based therapeutic interventions. Further, the growing need for treatment options that can cure a clinical condition, as opposed to those intended for treatment of disease-related symptoms, has created a demand for more effective gene manipulation approaches. Genome editing is a technique used to modify a single gene or a set of genes within the genome of an organism by altering the nucleotide sequence, using specialized molecular tools, such as artificially engineered nucleases or molecular scissors. The applications of this technology are vast, and it is often used to develop gene therapies. It is worth highlighting that the discovery of endonucleases and development of affiliated DNA splicing technologies played a key role in establishing genome editing as a promising field of research. Other key advances in the field include clustered regularly interspaced palindromic repeats (CRISPR), zinc finger nuclease (ZFN) and transcription activator-like effector nucleases (TALENs). Major genome editing techniques are described below:

2. Gene therapies are gradually changing the face of modern healthcare. These new regenerative and immunotherapy treatments have the potential to improve the practice of medicine and provide therapy solutions to patients who currently have limited or no treatment options. For the commercial success of such therapies, an effective manufacturing platform and a robust supply chain model is imperative. It is worth mentioning that a sustainable supply chain can actually enable further reductions in cost of goods and, in turn, prices of such therapies. GENE THERAPY MARKET: OVERALL MARKET LANDSCAPE The current market landscape features over 1,150 gene therapy candidates being investigated by more than 250 developers across different stages of development for the treatment of a myriad of disease indications; till date, 11 gene therapies have been commercialized. It is worth mentioning that three gene therapies received approval in 2020 across different regions; these include (in reverse chronological order of the month of approval) Luxturna (in Canada; developed by Sparks Therapeutics) Libmeldy (in Europe; developed by Orchard Therapeutics) andZolgensma (in Japan; developed by Novartis). Most of the therapies (70%) are currently in the early stages of development (preclinical / discovery), indicating that this domain has significant opportunity in the mid-long term.

3. The growing pipeline and the increasing demand for effective treatment using gene therapies, has spurred the establishment of many companies in the last decades. It is worth mentioning most of the companies engaged in the development of gene therapies were established post 2015 (42%). In fact, more than 40 gene therapy developers have been established, during the period 2019-2021. Notable examples of companies established in 2021 include (in alphabetical order) AAVantgarde Bio, Axovia Therapeutics, Nuntius Therapeutics and Opus Genetics. Also, the gene therapy market is currently dominated by the presence of small players (60%), followed by mid-sized firms (30%). Examples of well-established players in this domain include (firms established prior to 1990, more than 5,000 employees) Allergan, Amgen, Astellas Pharma, Bayer, Biogen, Boehringer Ingelheim, CSL Behring, Eli Lilly, Intas Pharmaceuticals, Janssen Pharmaceuticals, Pfizer, Roche, Takeda Pharmaceutical and UCB. During our research, we were able to identify 339 gene therapy products that have either been approved or being developed in clinical stages for the treatment of various diseases. It is evident from the figure that most of the gene therapies (53%) currently use AAV vectors as a preferred delivery vehicle.

4. In addition, 65% of such therapies are presently modified using the gene augmentation approach, followed by those based on oncolytic immunotherapy (18%). It is worth mentioning that oncolytic therapies use genetically modified viruses that are capable of selectively targeting and killing cancer cells. In case of gene therapies, these viruses act as vehicles responsible for delivering the therapeutic agent to the cancer site. Moreover, most of the gene therapies have received orphan drug designation (168), followed by those awarded with fast track designation (70), and rare pediatric disease designation (59).