Dyne Therapeutics Receives Orphan Drug Designation for z-basivarsen, ACHIEVE Trial Shows Sustained Improvement
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Jan 20 2026
0mins
Source: Globenewswire
- Clinical Trial Progress: Z-basivarsen demonstrates sustained functional improvement in the ACHIEVE trial, particularly in muscle strength and function, with enrollment for the registrational expansion cohort expected to complete in early Q2 2026, further advancing treatment options.
- Orphan Drug Designation: The MHLW in Japan has granted z-basivarsen orphan drug status for myotonic dystrophy type 1 (DM1), highlighting the urgent need for new therapies and potentially providing up to 10 years of market exclusivity if approved.
- Multiple Designations: Z-basivarsen has received Breakthrough Therapy, Fast Track, and Orphan Drug designations from the FDA, along with Orphan Drug designation from the EMA, indicating its development potential and market recognition on a global scale.
- Treatment Need: DM1 affects approximately 40,000 people in the U.S. and 55,000 in the EU, and the development of z-basivarsen aims to address the significant unmet medical need, carrying important social and economic implications.
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Analyst Views on DYN
Wall Street analysts forecast DYN stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for DYN is 38.40 USD with a low forecast of 17.00 USD and a high forecast of 60.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
10 Analyst Rating
8 Buy
2 Hold
0 Sell
Strong Buy
Current: 17.700
Low
17.00
Averages
38.40
High
60.00
Current: 17.700
Low
17.00
Averages
38.40
High
60.00
About DYN
Dyne Therapeutics, Inc. is a clinical-stage neuromuscular disease company. It is discovering and advancing therapeutics for people living with genetically driven neuromuscular diseases. Leveraging the modularity of its FORCE platform, it is developing targeted therapeutics that deliver to muscle and the central nervous system (CNS). It has a broad pipeline for neuromuscular diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease. Its product candidate, DYNE-101, is being evaluated in ACHIEVE, an ongoing Phase I/II global clinical trial in patients with DM1. Its product candidate, DYNE-251, is being evaluated in DELIVER, an ongoing Phase I/II global clinical trial in patients with DMD who have mutations amenable to exon 51 skipping. Its other product candidates are DYNE-302 and DYNE-401. DYNE-302 addresses the genetic basis of FSHD.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Dyne Therapeutics Receives Orphan Drug Designation for z-basivarsen, ACHIEVE Trial Shows Sustained Improvement
- Clinical Trial Progress: Z-basivarsen demonstrates sustained functional improvement in the ACHIEVE trial, particularly in muscle strength and function, with enrollment for the registrational expansion cohort expected to complete in early Q2 2026, further advancing treatment options.
- Orphan Drug Designation: The MHLW in Japan has granted z-basivarsen orphan drug status for myotonic dystrophy type 1 (DM1), highlighting the urgent need for new therapies and potentially providing up to 10 years of market exclusivity if approved.
- Multiple Designations: Z-basivarsen has received Breakthrough Therapy, Fast Track, and Orphan Drug designations from the FDA, along with Orphan Drug designation from the EMA, indicating its development potential and market recognition on a global scale.
- Treatment Need: DM1 affects approximately 40,000 people in the U.S. and 55,000 in the EU, and the development of z-basivarsen aims to address the significant unmet medical need, carrying important social and economic implications.
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