Golodirsen is a Antisense Oligonucleotide owned by Sarepta Therapeutics, and is involved in 6 clinical trials, of which 1 was completed, and 5 are ongoing. Golodirsen is is based on the exon skipping technology which skips exon 53 of the dystrophin gene, allowing for restoration of the reading frame in the mRNA sequence. By skipping this exon, a truncated, yet potentially functional form of the dystrophin protein is produced, which could ameliorate the disease process. The phosphorodiamidate morpholino oligomers (PMOs) are a distinct class of oligonucleotide analogs. They bind to RNA and efficiently interfere with gene expression in a sequence-specific manner.
The revenue for Golodirsen is expected to reach a total of $3.5bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Golodirsen NPV Report.
Golodirsen is originated and owned by Sarepta Therapeutics.
Golodiseren (Vyondys 53) is an analogue of oligonucleotide. It s formulated as solution for intravenous route of administration. Golodiseren is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
Sarepta Therapeutics Overview
Sarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. The company’s platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Its commercial products include Exondys 51, Vyondys 53 and Amondys 45 indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene. Its pipeline product includes SRP-5051, SRP-9001, SRP-9003 and SRP-5045 indicated for the treatment of DMD, limb-girdle muscular dystrophies (LGDMs) and other neuromuscular and central nervous system disorders. Sarepta is headquartered in Cambridge, Massachusetts, the US.
The company reported revenues of (US Dollars) US$701.9 million for the fiscal year ended December 2021 (FY2021), an increase of 30% over FY2020. The operating loss of the company was US$362.2 million in FY2021, compared to an operating loss of US$456.1 million in FY2020. The net loss of the company was US$418.8 million in FY2021, compared to a net loss of US$554.1 million in FY2020. The company reported revenues of US$230.3 million for the third quarter ended September 2022, a decrease of 1.4% over the previous quarter.
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