Dyne Therapeutics R&D Day: Updates for DMD and DM1

Two and a half years after announcing its launch, Dyne Therapeutics held its very first Research & Development Day. Throughout the day, there were presentations focusing on the development of treatments for rare muscle diseases, all of which can be found here. One of the most exciting points of the day was the update on the progress of DYNE-251 and DYNE-101.

Dyne’s R&D Day

Throughout the day, there were a number of presentations given in regard to rare muscle diseases, research, and drug development. Experts in the field were present, such as the Professor of Neurology and Pediatrics and Director of the Neuromuscular Division at Stanford, Dr. Day. Information presented throughout the day includes:

  • Plans to submit Investigational New Drug (IND) applications for DYNE-251 and DYNE-101, treatments for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1), respectively.
    • The current plan is to submit an IND for DYNE-251 during the final quarter of this year, and an IND for DYNE-101 in the first quarter of next year.
  • Plans to initiate global clinical trials following IND submission.
    • A placebo-controlled, multiple-ascending dose (MAD) trial assessing DYNE-251 will enroll patients from across the world
    • Adult DM1 patients will be enrolled in a global, MAD, placebo-controlled trial as well.
  • Presentations from Dr. Sansone, the Clinical and Scientific Director of the Clinical Center NeMO and Associate Professor at the University of Milan, where she teaches neurology.
  • Presentations from Dr. Day.
  • Presentations on data from the 2021 Muscle Study Group Annual Scientific Meeting and the World Muscle Society 2021 Virtual Congress, both of which are planned to support the IND submissions.
  • Plans to submit an IND for DYNE-301, a treatment for facioscapulohumeral muscular dystrophy, in the latter half of 2022.

All of the data and information presented is very exciting for medical professionals and patients alike, as it points to new treatment options for populations facing an unmet medical need.

About DMD

Duchenne muscular dystrophy (DMD), is one of nine forms of muscular dystrophy. A mutation passed down in an X-linked recessive pattern stops patients from producing dystrophin, ultimately resulting in progressive muscle weakness and degeneration. Other symptoms include difficulty with motor skills, fatigue, learning disabilities, frequent falls, issues with moving positions, and difficulty walking. Complications may include heart disease and respiratory failure, making treatment integral. The main form of treatment is steroids, but there is ongoing research looking into new options for DMD. Other current options include amino acids, asthma treatment, physical and occupational therapy, heart medications, assisted breathing, carnitine, creatine, and other supplements.

About DM1

Myotonic dystrophy is a form of muscular dystrophy that is characterized by the progressive weakening and atrophy of the muscles. There are two forms of this condition: type 1 and type 2, with the latter often resulting in less severe symptoms and affecting different muscles. Dyne’s investigational therapy is intended to treat the first type, which is the result of a mutated DMPK gene, while type 2 occurs due to a mutated ZNF9 gene. Common symptoms of both forms include an abnormal heart rhythm, weakness of the cardiac muscles, problems with breathing, weakness in the voluntary muscles, cataracts, learning disabilities, an inability to relax the muscles, issues swallowing, gallstones, constipation, abnormal uterus muscles, and issues with speech, vision, and hearing. There is currently no cure or treatment specific to myotonic dystrophy; treatment is symptomatic.

Find the source article here.

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