Nucleic acid-based therapeutics are used to target genes responsible for either the expression of a disease causing proteins or to correct the decreased protein expression in diseases where the absence of the protein contributes to a disease state.
Nucleic acid-based therapeutics are target deficiencies or dysfunctions at the molecular level and are targeted therapies. These are used specifically to target genetic diseases and disorders for which there exists no permanent cure such as thalassemia, sickle cell anemia, hemophilia, cystic fibrosis, diabetes etc. The nucleic acid-based therapeutics are based on accurate target identification and genetic profiling and the human gene project has formed the backbone of these class of drugs. As such nucleic acid-based medication have one of the most versatile and revolutionary potential.
The critical market driver for nucleic acid-based therapies is the poor cure rates for genetic diseases with traditional drugs. Other market drivers include increasing understanding of the human genetics, growing capabilities of mapping human tissue molecular targets, rising power of software’s to mimic the human molecular entities such as receptors etc.
Despite these achievements the market for nucleic acid-based medication is growing at a rate not justifiable with their potential. The lack of complete understanding of human genetics and molecular targets is the most critical constraints. The death of Jesse Gelsinger, as a result of a gene therapy in 1999 exemplifies the dangers associated with the therapy. Other restraints are ethical issues such as the changes at the genetic level and genetic contamination, extremely prohibitive regulatory requirements and high treatment cost of the nucleic acid-based therapy etc.
The global market for Nucleic Acid-based Therapeutics is expected to reach around USD 741.98 million by the end of the forecast period and is expected to grow at a CAGR of ~6.8%.
Some of the key players in this market are: Wave Life Sciences Ltd., Copernicus Therapeutics Inc., Imugene, Caperna, Phylogica, Protagonist Therapeutics, Benitec Biopharma, EGEN (Expression Genetics), Benitec Biopharma, BioMedica (Oxford BioMedica), Transgene and others.
Segments:
Global nucleic acid-based therapeutics market has been segmented on the basis of technology which comprises anti-sense and anti-gene, short inhibitory sequences, gene transfer therapy, nucleoside analogs, ribozymes, aptamers and others. On the basis of applications; market is segmented into monogenetic disorders which is further sub segmented into thalassemia, sickle cell anemia, hemophilia, cystic fibrosis etc. and multi-genetic disorders which is sub segmented into cancer, diabetes, neurodegenerative diseases, cardiovascular diseases etc. On the basis of end users; market is segmented into hospitals, academic & research institutes.