Dyne Therapeutics

Dyne Therapeutics received FDA IND clearance for DYNE-101 for DM1, anticipates new ACHIEVE trial data in early 2025, and is enrolling a DELIVER trial cohort for DYNE-251 in DMD. The company reported a Q3 2024 net loss of $97.1M, with $723.7M in cash to fund operations into 2026.

RNA interference (RNAi) research, despite initial challenges, has seen advancements with six approved medicines and many in clinical testing. New startups believe RNAi is ready for broader use beyond liver diseases, with innovations in delivery techniques to other tissues. Key players include City Therapeutics, Judo Bio, Switch Therapeutics, and Aro Biotherapeutics, backed by significant funding. The potential of RNAi, first discovered in the 1990s, is now seen as a new frontier in genetic medicine.

Global exosome market to grow at ~20% CAGR by 2030, driven by chronic disease incidence, diagnostic improvements, and personalized medicine interest. North America leads, with key players like Danaher and Thermo Fisher Scientific. Notable developments include Aruna Bio's US patent for neural exosomes and EXO Biologics' EUR 16 million Series A funding.

Six clinical-stage biotech companies are advancing Duchenne muscular dystrophy treatments: Wave Life Sciences, Sarepta Therapeutics, Capricor Therapeutics, Edgewise Therapeutics, Italfarmaco, and Avidity Biosciences. These companies focus on various therapeutic approaches, including RNA medicines, gene therapies, myosin inhibitors, and HDAC inhibitors. The global Duchenne treatment market is expected to grow significantly, driven by regulatory approvals and ongoing research.

Athira Pharma's lead candidate, fosgonimeton, failed Phase II/III LIFT-AD trial for Alzheimer's, causing a 81% stock drop. Despite missing primary and secondary endpoints, Athira remains committed to fosgonimeton's development, citing potential benefits in specific subgroups. Full data will be presented at the CTAD Conference in Madrid.

Dyne Therapeutics' DYNE-251, an investigational antisense oligonucleotide, showed significant dystrophin expression in Duchenne muscular dystrophy patients amenable to exon 51 skipping in the DELIVER trial. DYNE-251 achieved a mean absolute dystrophin expression of 3.71% of normal, surpassing the standard of care. The therapy demonstrated functional improvements and a favorable safety profile, prompting the initiation of registrational cohorts with plans for an update on the path to registration by year-end 2024.

Treatment with DYNE-251, Dyne Therapeutics' exon 51-skipping therapy, improved motor function in boys with Duchenne muscular dystrophy (DMD) in a Phase 1/2 trial. DYNE-251 increased dystrophin levels, with 20 mg/kg doses reaching 3.71% of normal, significantly higher than Exondys 51. Functional benefits were observed across various assessments, meeting European Medicines Agency thresholds for clinical significance. Dyne plans to initiate registrational cohorts and pursue expedited approval pathways, with an update expected by year's end.

Dyne Therapeutics announced positive Phase 1/2 DELIVER trial data for DYNE-251 in Duchenne patients, showing 3.71% mean absolute dystrophin expression at 20 mg/kg Q4W, with improvements in functional measures. The company plans to initiate registrational cohorts and pursue expedited approval pathways.

Dyne Therapeutics announces new clinical data from its Phase 1/2 DELIVER trial of DYNE-251 in Duchenne muscular dystrophy, showing unprecedented dystrophin expression and functional improvement. The company plans to initiate registrational cohorts and provide an update on its path to registration by the end of 2024.

Dyne Therapeutics announced significant clinical data from its Phase 1/2 DELIVER trial of DYNE-251 for Duchenne muscular dystrophy, showing unprecedented dystrophin expression and functional improvements. The company plans to initiate registrational cohorts and provide a registration update by end-2024. A virtual investor event was held to discuss these findings.