As various governments are enhancing the focus on treating rare diseases, the demand for hunter syndrome treatment is rising. The commercialisation of innovative hunter syndrome treatments and therapies is also propelling the market growth. With the initiation of clinical trials to determine the effectiveness of new treatments for hunter syndrome, the market is poised to witness robust growth. In addition, stem cell-based gene therapy is increasingly investigated to treat children with hunter syndrome, which is likely to fuel the market’s expansion. The increasing research activities to develop new therapies that are designed to deliver proteins to peripheral tissues and the brain for treating somatic symptoms as well as neuronopathic features of hunter syndrome are expected to augment the market growth in the forecast period.
REGENXBIO Inc. (NASDAQ: RGNX), one of the leading companies in the market, announced in February 2022 the additional positive interim data from Cohort 1-3 of the ongoing Phase I/II trial of RGX-121. RGX-121 is an investigational one-time gene therapy that is designed to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme using the AAV9 vector. It is being investigated for the treatment of patients up to 5 years old that are diagnosed with Hunter Syndrome, also known as Mucopolysaccharidosis Type II (MPS II). The company announced that the interim data demonstrates neurodevelopmental trajectories in patients from Cohorts 1 and 2 and dose-dependent reductions in CSF biomarkers across cohorts. Moreover, the data support the early positive signals that are witnessed in the emerging clinical profile of RGX-121 for hunter syndrome treatment.
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The major players in the global hunter syndrome treatment market are Denali Therapeutics Inc., ArmaGen, Inc., Sangamo Therapeutics Inc., CANbridge Life Sciences Ltd. and REGENXBIO Inc., among others. The comprehensive report by EMR looks into the market share, capacity, and latest developments like mergers and acquisitions, plant turnarounds, and capacity expansions of the major players.
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