Muscles are the strength of the body, as large as walking and running, as small as the eyelid beating are inseparable from muscles, muscle diseases can seriously affect the daily life of patients. Founded inyearsdyne therapeuticsis a biopharmaceutical company developing targeted drugs** for patients with severe muscle diseasesAs a company with a team of experienced staff in muscle diseases, rare diseases, drug development, new manufacturing, and commercialization, its proprietary Force platform can overcome the limitations of tissue delivery and advance modern oligonucleotide** drug candidates. On January 11, 2024, Dyne Therapeutics announced gross earnings on its public offering of common shares of 3$4.5 billion. In September 2020, Dyne Therapeutics was listed on the NASDAQ through an IPO, and a month before the listing, the company raised 1$1.5 billion, and the last few financings have been more than 100 million dollars.
Yaorong Cloud Data wwwpharnexcloud.com: basic information of the company.
Financing can be realizeddyne therapeuticsby those who have extensive work experienceromesh subramanianFounded by DrDr. Romesh Subramanian left the company in 2021 and subsequently served as President and Chief Executive Officer of Ascidian Therapeutics, and in November 2023, Dr. Romesh Subramanian again announced his departure from Ascidian Therapeutics. Prior to founding Dyne Therapeutics, Dr. Romesh Subramanian held positions at Thrasos Therapeutics and Pfizer before co-founding Rana Therapeutics (renamed Translate Bio in 2017 and acquired by Sanofi in 2021) and as Entrepreneur-in-Residence at Atlas Ventures, a biotech venture capital firm.
Dr. Romesh Subramanian (Source: Endpoint News).
Dyne Therapeutics' proprietary antigen-binding fragments bind to receptors that are highly expressed on muscle cells for targeted delivery to skeletal, cardiac, and smooth muscles. The linker is then selected based on its demonstrated safety and efficacy in an approved product, as well as its stability and ability to release ** drug within muscle cells. Finally, the FAB and linker are attached to the payload such as antisense oligonucleotides (ASOs), phosphodiamide morpholino-oligomers (PMOs), siRNAs, or small molecules. The Force platform offers several advantages, including the ability to target delivery to muscle tissue, extend dosing intervals, redosing, and target the genetic basis of the disease to halt or reverse its progression. (Fab has significant muscle delivery advantages over monoclonal antibodies (mAbs), including enhanced tissue permeability, improved tolerability, and reduced risk of immune system activation).
Seek to expand the product pipeline on the existing basisDyne Therapeutics' pipeline is focused on myotonic muscular dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). It also intends to expand the company's product portfolio using the Force platform and continue to develop programs for other indications, including other rare bone, central nervous system, cardiac and metabolic muscle diseases.
Myotonic dystrophy type 1 (DM1).dm1It is a monogenic autosomal dominant disorder caused by an increase in CTG repeats in the DNA sequence of the DMPK (myotonic dystrophin kinase) gene, and patients often develop myotonia and progressive weakness of major muscle groups, which affects mobility, breathing, heart function, speech, digestion and vision, and cognition. There are currently no approved ones on the market**. DYNE-101 is an investigational** drug targeting DM1 consisting of an antisense oligonucleotide (ASO) conjugated to a fragment antibody (FAB) that binds to transferrin receptor 1 (TFR1), which is highly expressed on muscle. Designed to release the splicing protein by decreasing the level of mutant DMPK RNA in the nucleus, allowing normal mRNA processing and translation of normal proteins, and potentially halting or reversing disease progression, the drug has been granted orphan drug designation by the EMA and FDA for DM1,YesachieveTested fordm1Patients are evaluated and are currently in clinical trialsperiod, expected inAvailable in the second half of the yearachieveThe next clinical data update for the trial. The Achieve trial, which consists of a 24-week multiple ascending dose-randomized placebo-controlled period, a 24-week open-label extension period, and a 96-week long-term extension period, is enrolling 18 49-year-old adult patients with DM1, with primary endpoints being safety and tolerability, and secondary endpoints being pharmacokinetics and pharmacodynamics, including change from baseline in splicing, and measures of muscle strength and function.
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yearsAt the beginning of the month,dyne therapeuticsAnnouncedachievePreliminary clinical data from the trialdyne-101Exhibits dose-dependent splicing correction, muscle delivery, anddmpkKnockdown (knockdown in skeletal muscledmpkcan effectively alleviate symptoms), while also showing functional improvement in muscle rigidity.
Duchenne muscular dystrophy (DMD).DMD occurs mainly in men and is caused by mutations in the gene encoding dystrophin, a protein that is essential for the proper function of muscle cells. These mutations, most of which are deletions, lead to a deficiency of dystrophin and a gradual loss of muscle function. The DMD market is worth $3.5 billion in 2023Expectedyears will be reachedbillion billion at a CAGRdyne-251It is an investigational ** drug,deliverTrials are carried out to clinical trialsperiod, for carryingExon skipping mutationsdmdPatient. ExpectedAvailable in the second half of the yeardeliverThe next clinical data update for the trial. Phosphodiamide morpholine oligomers (PMOs) are conjugated to a fragment antibody (FAB) that binds to transferrin receptor 1 (TFR1), which is highly expressed on muscle, and is designed to achieve targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to produce truncated functional dystrophin that halts or reverses disease progression. dyne-251has beenfdaFast Track, Orphan Drug, and Rare Pediatric Disease Designation granted. Exceptdyne-251Outside,dyneis building a global scaledmdfranchises and has preclinical programs for other exons, including:NoNo. andExons. On January 4, 2024, Dyne Therapeutics presented preliminary efficacy evaluations of the Deliver trial based on 6 months of data from six male patients with DMD who were randomly assigned to receive either Dyne-251 or placebo every four weeks. When dyne-251 was administered every 4 weeks, the levels of dystrophin expression, exon skipping levels, and the percentage of dystrophin-positive fibers exceeded the levels reported in the currently approved clinical trial of exon 51** (Exondys 51:Eteplirsen of Sarepta Corporation) at 6 months, resulting in a 24-fold reduction in total PMO dose.
From the pharmaceutical finance cloud data wwwpharnexcloud.com has learned that there are currently four companies that have been targeteddmdof drugs have been approved, among otherssareptacompanyexondys 51It is the first approved** exomeGene skipping mutationsdmdTargeting**.
In China, there are no approved drugs for DMD, and the drug with the fastest research and development progress has progressed to phase 3 clinical trials, and the latest information is the progress of vitorasen injection released by Japan New Pharmaceutical Co., Ltd. and Hangzhou Tigermed Technology Co., Ltd. in October 2023.
Facicoscapulohumeral muscular dystrophy (FSHD).FSHD is a rare disease caused by abnormal expression of the DUX4 gene in muscle tissue that causes muscle death and replacement by fat, characterized by weakness of the facial muscles and shoulder girdles of the upper limbs, and there is no approved FSHD on the market**. Dyne Therapeutics aims to address the genetic basis of the disease by targeting toxic DUX4 expression, and drug candidate DYNE-301 is conjugated to ASO by the Company's proprietary FAB with an adapter to address the genetic basis of FSHD by decreasing the expression of DUX4 in muscle tissue.
Don't be afraid of losses and increase investmentFrom the pharmaceutical finance cloud data wwwpharnexcloud.com learned that Dyne Therapeutics' investment can be said to be increasing year by year, and the R&D expenses in 2022 will be 1US$4.3 billion, general and administrative expenses of US$28.2 million, net loss for the year of US$1$68.1 billion.
Pharmaceutical Finance Cloud Database: Annual Report of the Company.
For the third quarter ended September 30, 2023, the financial results for the third quarter of 2023 were $55.3 million in R&D expenses, $7.0 million in general and administrative expenses, and a net loss of $60.2 million, or 0.0 per essentially diluted share$99. Financial results for the first three quarters of 2023 R&D expenses were 1$5.2 billion, general and administrative expenses of $22.56 million, net loss of $1$6.9 billion, or $2.9 billion per diluted share$86.
As ofyearsmonthday, cash, cash equivalents and valuable** arebillion dollars, which is expected to beyears of operation to provide funding
As of press time, the company's market capitalization is about 32$5.6 billion.
Reference: nmpa cde;
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