NIPPON SHINYAKU CO

Atsena Therapeutics initiates Part B of Phase I/II LIGHTHOUSE study for ATSN-201 gene therapy targeting X-linked retinoschisis (XLRS), enrolling adults and children. ATSN-201 uses AAV.SPR capsid for safe gene expression in retina. Part A showed safety and efficacy, leading to Part B with optimal dose. XLRS, affecting ~30,000 males in US/EU, has no approved treatments.

Capricor Therapeutics submitted a US marketing application for deramiocel, aiming to treat Duchenne muscular dystrophy-related cardiomyopathy. The FDA filing triggers a $10M payment from Nippon Shinyaku. Deramiocel, a cell therapy, showed cardiac function improvements in studies. If approved, it could receive a priority review voucher, potentially speeding up regulatory review.

Nippon Shinyaku’s DMD therapy Viltepso faces uncertainty after failing a confirmatory trial, missing its primary objective and not distinguishing from placebo in patient mobility tests. Despite prior confidence in its efficacy, continued approval hinges on further clinical benefit verification. Meanwhile, Sarepta’s gene therapies, including Elevidys, are emerging as potential successors in DMD treatment.

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Capricor Therapeutics submitted a Biologics License Application to the FDA for deramiocel, a cell therapy for Duchenne muscular dystrophy cardiomyopathy, marking a significant step towards potential approval. Supported by clinical trial data, the company seeks a priority review, which could expedite the process. Deramiocel has shown promise in attenuating DMD's cardiac effects, with Capricor eligible for a $10 million milestone payment and a Priority Review Voucher upon approval.

Capricor Therapeutics' shares rose 8.4% after submitting a BLA to the FDA for deramiocel, a treatment for DMD cardiomyopathy. A decision is expected in late 2024. The submission triggered a $10M payment from Nippon Shinyaku. Deramiocel showed positive results in slowing DMD progression and improving quality of life.

Capricor Therapeutics submitted a Biologics License Application to the FDA for deramiocel, a cell therapy for Duchenne muscular dystrophy cardiomyopathy, supported by clinical trial data. The company seeks priority review and is eligible for a $10M milestone payment. Deramiocel has received Orphan Drug and RMAT designations, targeting DMD's cardiac implications with potential for transformative treatment.

Capricor Therapeutics submitted a Biologics License Application to the FDA for deramiocel, targeting Duchenne muscular dystrophy cardiomyopathy, triggering a $10M milestone payment. The submission includes Phase 2 trial data, with hopes for a priority review to shorten the approval timeline.

Capricor Therapeutics submitted a BLA to the FDA for deramiocel, targeting DMD cardiomyopathy, triggering a $10M milestone. Positive Phase 2 trial data supports its potential to slow cardiac damage, with priority review requested. Approval could mark a significant advancement in DMD treatment, offering hope for improved quality of life and lifespan extension.