Market for repeat expansion disorders treatment is expected to grow at a CAGR of 13.5% during the forecast period of 2023 to 2030. Repeat expansion disorders are a group of inherited conditions that are triggered by expansions in the DNA repeats of affected individuals. The length of the DNA repeats can range from a single nucleotide all the way up to a dodecamer or even longer. When recurrent expansions create symptoms varies by illness. These DNA expansions cause over 40 diseases. The mutations that cause many of these illnesses have been identified through research being done over the past 30 years. CAG, GCG, CTG, CGG, and CAAA repeat expansion in coding and non-coding sections of distinct genes causes a variety of diseases involving protein levels, toxicity, RNA, or both. Both coding and non-coding sequences contain repetitions.
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The complexity of recurrent disease genetics and pathobiology has revealed previously undiscovered similarities and molecular pathways, such as RNA toxicity. Post-translational modification is a critical stage in the pathogenic cascade, and the autophagy pathway degrades misfolded proteins. These are two neurodegenerative themes. Recurrent illness research sparks new research avenues. These new areas of investigation should illuminate illness molecular pathways and identify therapeutic prospects for incurable disorders. Despite these advances, determining how recurrent expansion mutations cause inherited illnesses remains difficult. Recent discoveries have highlighted the complexity of the molecular processes underlying repeat expansion-related illnesses. Extensive study on CAG-polyglutamine illnesses has demonstrated that post-translational modification of disease proteins is a crucial stage in disease progression, and data suggests that the autophagy system plays a significant role in the degradation of misfolded proteins.
Since a novel and obscure mutation was found to produce fragile X syndrome and SBMA, much progress has been achieved. The research in this field has discovered an unforeseen complexity, including protein, RNA, and genome-level toxicities. Research on recurrent disorders is vital for determining how defective RNA metabolism and autophagy cause human illness. Why treatments haven't kept pace with scientific advances in understanding mechanisms and pathways is a recurring question in recurrent disease. This disconnect applies to most neurological conditions; nonetheless, repeat diseases hold a great opportunity for developing appropriate therapeutics because conclusive diagnosis is based on repeat allele expansion. These discoveries have led to promising therapeutic breakthroughs, such as the use of oligonucleotide and morpholino anti-sense repeats against the DM1 CTG expansion.
Difficulties in designing and completing clinical trials for developing a treatment for rapid expansion diseases are expected to hamper market growth over the forecast period. It's difficult to design clinical trials for rare neurodegenerative disorders like Friedreich's ataxia since there aren't enough patients because the condition progresses slowly and variably. These variables make it hard to find reliable outcome metrics to assess disorder progression. Friedreich's ataxia is a hereditary, neurological mobility illness with a typical onset age of 10 to 15 years, according to a 2020 article in the Orphanet Journal of Rare Diseases. The average age of Friedreich's ataxia patients determined this range. From disease onset to accurate diagnosis takes a long time.
Huntington disease therapy is predicted to grow at a 20% CAGR from 2023 to 2030. The planned label expansion of Ingrezza for Huntington's chorea, the high prevalence of HD in western countries, and a solid pipeline of disease-modifying medicines are expected to be major drivers during the projected period. HD affects 30,000 people in North America, or 5.7 per 100,000. Children only account for 5–10% of all HD cases. It affects 10 in 100,000 Europeans.Geographic location may affect case ascertainment and diagnostic criteria, causing a tenfold difference in prevalence. Asia has a lower incidence than Europe, North America, and Australia. Immunomodulatory drugs are being developed to target HD's overactive immune system.
North America held 60% of the rapid expansion disorders treatment market in 2021. This expansion is attributable to the disease's high burden, higher healthcare costs, technology improvements, proactive government actions, and increased patient awareness about treatment options. Disease burden increases healthcare costs. Due to increased product penetration, unexplored potential, and the growing frequency of the illness in emerging nations, Asia Pacific is predicted to have the greatest CAGR from 2023 to 2030. Governments' efforts to improve the population's health are expected to boost the region's market.
Market is fragmented due to the scope of market that is characterized by different pharma companies leading different therapeutic segment across countries. Therefore, the competition is significantly high and is expected further intensify during the forecast period of 2023 to 2030. The key strategy adopted by Tier 1 companies is focus on R&D to develop novel therapeutic options. Some of the prominent companies dominating the market includes Pfizer, Inc., CRISPR Therapeutics, Acorda Therapeutics, Capsida Biotherapeutics Inc., Larimar Therapeutics, Inc., Healx, Intellia Therapeutics, Inc., Editas Medicine, bluebird bio, Inc., Sanofi, Merck & Co., Inc., H. Lundbeck A/S, Cellectis SA, Bio-Techne, GlaxoSmithKline plc, Allergan Aurobindo Pharma Ltd., Sun Pharmaceutical Industries Ltd., Torrent Pharmaceuticals Ltd., Intas Pharmaceuticals Ltd.Cipla Ltd and others.
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