Nucleic Acid Based Gene Therapy Market Global Report 2020-30

Executive Summary

The nucleic acid-based gene therapy market consists of sales of nucleic acid-based gene therapy products and services. Gene therapy is used for correcting defective genes that are responsible for disease development. Moreover, nucleic acid-based therapeutics are used to treat genetic disorders and diseases for which there exists no permanent cure such as anaemia, sickle cell, cystic fibrosis, diabetes, and thalassemia.

The global nucleic acid based gene therapy market was worth $557.47 million in 2019. It is expected to grow at a compound annual growth rate (CAGR) of 10.00% and reach $816.18 million by 2023.

The nucleic acid-based gene therapy market covered in this report is segmented by technology into anti-sence and anti-gene, short inhibitory sequences, gene transfer therapy, nucleoside analogs, ribozymes, aptamers, others. It is also segmented by application into oncology, muscular dystrophy/ muscular disorders, rare diseases.

Rising financial support by the government and the companies is projected to drive the demand for nucleic acid based gene therapy. Nucleic acid therapeutics are analogs of naturally occurring acids or proteins responsible for genetic expression. The traditional therapies do not have any cure for the treatment of diseases such as cystic fibrosis, hemophilia, sickle cell anaemia, thalassemia, and diabetes. Genetic profiling and molecular target identification form the backbone of these classes of drugs. Nucleic acid medication has greater potential for the treatment of these diseases, as they target the genetic basis of diseases and have a permanent cure. Rising financial support by the government and the companies dealing in the market for gene therapy is expected to contribute to increasing the demand for nucleic acid-based gene therapies. For instance in October 2019, the USA National Institutes of Health (NIH) announced plans to invest $100 million for the next four years to cure HIV and sickle cell disease with gene therapies. The government support for gene therapies will contribute to the growth of the market.

Stringent regulations imposed on gene therapies raises the price of gene therapies, which in turn hinders the demand for nucleic acid-based gene therapies. The excessive regulatory oversights create an expensive and elongated route for approval increasing the expenses. According to Foundation for Economic Education (FEE), unlike other drugs approved or regulated by the Food and Drug Administration (FDA), gene therapies are not only subject to the regulatory structure of FDA, but also the Recombinant DNA Advisory Committee and Office of Biotechnology Activities. Also, as estimated by FEE, an approved gene therapy drug cost nearly $5.0 billion, which is five times higher than that of the average cost of FDA approval. The high cost of gene therapeutics drugs places them beyond the financial reach of the populace. This scenario is anticipated to restrain the market growth of the nucleic acid-based gene therapy market.

In May 2018, Vectalys, a leading biotechnology company specializing in the manufacturing of high-quality solutions for gene delivery and FlashCell, a company engaged in developing non-integrated lentiviral delivered RNA Therapeutics announced the merger to create Flash Therapeutics, a privately held gene therapy company developing cell and gene therapeutics. The new company Flash Therapeutics is expected to focus on the development of RNA therapeutics based on LentiFlash, a non-integrative lentiviral delivery technology for incurable diseases.

Major players in the market are Wave Life Sciences, Imugene, Caperna, Phylogica, Protagonist Therapeutics, Benitec Biopharma, EGEN, BioMedica, Transgene and Copernicus Therapeutics.

• Celsion Corporation
• Wave Life Sciences
• Imugene
• Caperna
• Phylogica
• Protagonist Therapeutics
• Benitec Biopharma
• EGEN
• BioMedica
• Transgene
• Copernicus Therapeutics

Methodology