Core Insights - Dyne Therapeutics has received Fast Track designation from the FDA for DYNE-101, aimed at treating myotonic dystrophy type 1 (DM1) [1][2] - The company plans to submit for U.S. Accelerated Approval in the first half of 2026 [1] Company Overview - Dyne Therapeutics is focused on developing therapeutics for genetically driven neuromuscular diseases, utilizing its FORCE™ platform [5] - The company is currently conducting a Phase 1/2 global clinical trial for DYNE-101 [3] Product Details - DYNE-101 is an investigational therapeutic designed to target muscle tissue and reduce toxic DMPK RNA, potentially halting or reversing disease progression [3] - The drug has received orphan drug designation from both the FDA and the European Medicines Agency for DM1 treatment [3] Disease Context - Myotonic dystrophy type 1 (DM1) is a rare genetic disease affecting over 40,000 people in the U.S. and 74,000 in Europe, with no approved disease-modifying therapies available [4]

Core Insights - Dyne Therapeutics, Inc. has revealed new data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 for myotonic dystrophy type 1 (DM1) and plans to initiate a global Registrational Expansion Cohort to support U.S. Accelerated Approval submission in H1 2026 [1][4][5] Group 1: Trial Data - Efficacy data from the 6.8 mg/kg Q8W cohort (n=8) showed significant splicing correction at 3 months, linked to improvements in multiple functional endpoints at both 3 and 6 months [2][3] - Analysis of muscle biopsy data indicated a substantial knockdown of DMPK RNA levels, with robust splicing correction at 3 months supporting CASI-22 as a surrogate endpoint for potential U.S. Accelerated Approval [7] Group 2: Safety and Tolerability - Safety data from 56 patients indicated that most treatment-emergent adverse events were mild or moderate, with no serious treatment-emergent adverse events identified [4] - Early and sustained improvement in myotonia was observed, particularly in the 6.8 mg/kg Q8W cohort, with functional measures showing clinical benefits [7] Group 3: Future Plans - Dyne anticipates completing enrollment in the Registrational Expansion Cohort by mid-2025 and aims to submit for U.S. Accelerated Approval in H1 2026 [5]

Company Overview - Dyne Therapeutics is a clinical-stage company focused on developing innovative therapeutics for genetically driven neuromuscular diseases [3] - The company leverages its FORCE™ platform to design targeted therapeutics that address delivery challenges to muscle tissue and the central nervous system [3] - Dyne has a broad pipeline including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), as well as a preclinical program for facioscapulohumeral muscular dystrophy (FSHD) [3] Upcoming Event - Dyne Therapeutics' president and CEO John Cox is scheduled to present at the 43rd Annual J P Morgan Healthcare Conference on January 15, 2025 at 10:30 a m PT (1:30 p m ET) [1] - A live webcast of the presentation will be available on the company's website, with a replay accessible for 30 days following the event [2]

WALTHAM, Mass., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage neuromuscular disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced it plans to report new clinical data from the Phase 1/2 ACHIEVE clinical trial on January 10, 2025, and to host a webcast at 8:00 a.m. ET. The company intends to issue a press release prior to the start of the event. Investor Conference Call and ...

Exhibit 99.1 Dyne Therapeutics Reports Third Quarter 2024 Financial Results and Provides Corporate Update - IND Application for DYNE-101 for DM1 Cleared by FDA - - New Clinical Data from DYNE-101 ACHIEVE Trial Expected in Early January 2025 - - Enrolling Registrational Cohort of DYNE-251 DELIVER Trial in DMD - WALTHAM, Mass., November 12, 2024 – Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ________________ to ________________ Commission File Number: 001-39509 Dyne Therapeutics, Inc. (Exact Name of Registrant as Specified in its Charter) Delawa ...

WALTHAM, Mass., Nov. 07, 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 that management is scheduled to present at the following upcoming investor conferences: Guggenheim Inaugural Healthcare Innovation Conference, fireside chat on Wednesday, November 13, 2024 at 4:00 p.m. ET in Boston Stifel 2024 Healthcare Conference, fi ...

- Presentations Will Feature Recent Clinical Data From DYNE-251 and DYNE-101 as Well as Preclinical Data in FSHD and Pompe Disease - WALTHAM, Mass., Oct. 09, 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 that previously reported clinical and preclinical data across its pipeline will be featured in poster presentations at ...

NEW YORK, Sept. 10, 2024 (GLOBE NEWSWIRE) -- Bragar Eagel & Squire, P.C., a nationally recognized stockholder rights law firm, is investigating potential claims against Dyne Therapeutics, Inc. ("Dyne" or the "Company") (NASDAQ: DYN) on behalf of Dyne stockholders. Our investigation concerns whether Dyne has violated the federal securities laws and/or engaged in other unlawful business practices. Click here to participate in the action. The investigation focuses on whether the Company issued false and/or mis ...

NEW YORK, Sept. 9, 2024 /PRNewswire/ -- Pomerantz LLP is investigating claims on behalf of investors of Dyne Therapeutics, Inc. ("Dyne" or the "Company") (NASDAQ: DYN). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980. The investigation concerns whether Dyne and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices. [Click here for information about joining the class action] On September 3, 2024, Dy ...