WALTHAM, Mass., June 24, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (NASDAQ:DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced new preclinical data in a Pompe disease model demonstrating the potential of the FORCE™ platform to deliver enzyme replacement therapy to cardiac and skeletal muscle and the central nervous system (CNS). The data were presented at the New Directions in ...

Core Findings - FSHD is a severe muscle disorder caused by aberrant expression of the DUX4 gene, leading to progressive muscle wasting and skeletal muscle loss [1] - DYNE-302, developed using the FORCE platform, consists of a fragment antibody (Fab) that binds to transferrin receptor 1 (TfR1) and is conjugated to an siRNA designed to reduce DUX4 expression [1] - DYNE-302 demonstrated robust and durable DUX4 suppression and functional benefit in FSHD preclinical models, with effects lasting up to three months [4][7] FORCE™ Platform - The FORCE platform is designed to develop targeted oligonucleotide therapeutics for serious muscle diseases, leveraging TfR1's role in muscle biology [5] - The platform overcomes current limitations in delivering therapeutics to muscle tissue, aiming to stop or reverse disease progression [5] - Dyne links therapeutic payloads to its TfR1-binding Fab to create targeted treatments for muscle diseases [5] DYNE-302 Preclinical Data - In hTfR1/iFLExD mouse models, a single intravenous dose of DYNE-302 resulted in dose-dependent and robust reduction of the DUX4 transcriptome (D4T) [4] - DYNE-302 also showed high in vitro potency in FSHD patient-derived myotubes [4] - The data highlight the modularity of the FORCE platform to conjugate different oligonucleotides and target underlying disease mechanisms [4] FSHD Overview - FSHD is a rare, progressive genetic disease caused by a mutation in the DUX4 gene, leading to muscle weakness, wasting, and limited mobility [11] - An estimated 16,000-38,000 individuals in the US and approximately 35,000 in Europe are affected by FSHD, with no currently approved therapies [11] Company Overview - Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative therapeutics for genetically driven diseases [6] - The company has a broad pipeline, including clinical programs for DM1 and DMD, and a preclinical program for FSHD [6]

- Leveraging the FORCE™ Platform, DYNE-302 Achieved Robust and Durable DUX4 Suppression and Functional Benefit in FSHD Preclinical Models - "These encouraging data demonstrate that DYNE-302 exhibited prolonged activity in preclinical FSHD models, highlighting our innovative approach to targeting the genetic cause of this devastating and progressive muscle disease with no currently approved therapies," said Oxana Beskrovnaya, Ph.D., chief scientific officer of Dyne. "Additionally, the findings presented toda ...

About Dyne Therapeutics Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life- transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue. Dyne has a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystr ...

Given the current uncertain macroeconomic environment with high oil prices and elevated interest rates, could DYN face a similar situation as it did in 2021 and 2023 and underperform the S&P over the next 12 months — or will it see a strong jump? The $39 average of analysts' price estimate reflects a little over 10% upside from its current levels of $35. Note that Dyne doesn't have any marketable product yet, and its valuation is purely based on its pipeline potential. Moreover, smaller pharmaceutical firms ...

Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements relating to the anticipated closing date of the public offering, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," ...

WALTHAM, Mass., May 21, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced the pricing of an underwritten public offering of 10,500,000 shares of its common stock at a public offering price of $31.00 per share. The gross proceeds to Dyne from the offering, before deducting underwriting discounts and commissions and offering ex ...

Dyne Therapeutics (DYN) reported new clinical efficacy data from two separate mid to late-stage studies evaluating its two clinical-stage pipeline candidates, DYNE-101 and DYNE-251, in patients with myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, respectively. New clinical data from both studies demonstrated a meaningful impact on key biomarkers and functional improvement in multiple clinical endpoints that matter to patients. We remind the investo ...

Contacts: Investors About Dyne Therapeutics Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life- transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue. Dyne has a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duc ...

About Dyne Therapeutics Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life- transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue. Dyne has a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystr ...