Monogenetic Disease Therapy Market Size, Share, Growth Report 2025 Monogenetic Disease Therapy Market: Introduction Monogenetic diseases result from the mutation or change in the DNA sequence of one gene. A mutated gene hinders the normal functioning of the protein present in its composition and hence results in various types of monogenetic disorders such as cystic fibrosis, sickle cell disease, Gaucher disease, SCID and others. The monogenetic disease therapy market is expected to grow during the forecast period, due to a rise in the incidence of monogenetic diseases, coupled with advancement in treatment technologies. Request for sample copy of this report @ https://www.insightsandreports.com/request-sample/252 Market Dynamics: The monogenetic disease therapy market is expected to grow during the forecast period, due to the rising prevalence of monogenetic diseases globally. According to the World Health Organization (WHO), the global prevalence of all monogenetic diseases at birth is approximately 10/1000. Increased research activities and clinical trials on monogenic diseases and its therapeutic treatments are further anticipated to drive the monogenetic diseases therapy market. According to NCBI, monogenetic diseases were the second most popular indication of gene therapies after cancer, accounted for 10% of the total gene therapy trials conducted between 1989 and 2015. Other driving factors for the growth of monogenetic disease therapy market include rising awareness towards genetic diseases, coupled with the presence of key healthcare market players such as Sanofi, Bayer AG, Pfizer Inc., and others who are determined to develop innovative therapies, advanced bio-instruments, and vaccines to treat monogenetic diseases. However, high therapy cost and lower commercial success rates of treatments like gene therapy can restrain the growth of global monogenetic disease therapy market. By Inheritance Pattern On the basis of inheritance pattern, autosomal monogenetic diseases therapy market is expected to grow at a faster pace, due to rise in a number of autosomal monogenetic diseases like Cystic fibrosis (a most common type of monogenetic disease), Tay Sachs disease and Sickle cell anemia. According to the European Lung White Book, one in 25 people of European descent carries one mutant allele of CFTR, and one person among 2000-3000 people is affected by cystic fibrosis. Chromosomal disease therapy is also expected to grow during the forecast period, owing to the rising incidence of hemophilia, hunter disease, and others. Haemophilia is estimated to be afflicted in 1 in 10,000 people globally. By Therapy Type Pharmacological and surgical therapies for treating monogenetic diseases are predicted to dominate the global market, due to their higher preference among patients. For instance, in February 2018, the U.S. FDA approved a combination of ivacaftor and tezacaftor, developed by Vertex Pharmaceutical, to treat cystic fibrosis. Gene therapy is another therapy segment which is expected to witness a rapid growth over the forecast period, owing to its efficiency in the treatment of monogenetic diseases like CF and SCID. For instance, Glybera, which is the first clinically approved gene therapy in the European Union, uses an adeno-associated virus (AAV) vector drug for correction of lipoprotein lipase deficiency and to correct cystic fibrosis, it uses repeated nebulization of liposomes encoding the cystic fibrosis transmembrane conductance regulator (CFTR). Competitive Landscape Key market players operating in global monogenetic disease therapy market include Abbott Laboratories, BAG Healthcare Gmbh, Novartis, Pfizer Inc., Vertex Pharmaceuticals, Bristol-Myers Squibb, F. Hoffmann-La Roche Limited, Bayer AG, Grifols S.A, Bluebird Bio, UniQure, and Sanofi S.A. Market players, clinical research organizations and hospitals are focusing on collaborations to develop and commercialize therapies for the treatment of monogenetic diseases. For instance, in August 2018, Mustang Bio, a U.S. based pharmaceutical firm entered into an exclusive license agreement with St. Jude Children’s Research Hospital to develop the ex-vivo lentiviral gene therapy for X-linked SCID (commonly known as Bubble Boy Disease) treatment. Through this merger, Mustang Bio aims to expand their pipeline into gene therapy for patients with X-SCID. 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