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The Netflix documentary 'The Remarkable Life of Ibelin' deeply moved a DMD survivor, showcasing Mats Steen's life with Duchenne muscular dystrophy. Meanwhile, Capricor Therapeutics submitted a BLA for deramiocel, a cell therapy for DMD-related heart disease, marking a significant advancement.

The cell and gene therapy sectors are rapidly advancing, with significant developments like Capricor's BLA submission for DMD cardiomyopathy therapy, Arbor Biotechnologies' FDA clearance for a CRISPR-based therapy trial, Ultragenyx's BLA for MPSIII gene therapy, RESTEM's fast-tracked ULSC therapy for IIM, ViGeneron's rare pediatric disease designation for a retinitis pigmentosa gene therapy, and uniQure's public offering pricing shares at $17.

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.

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 BLA for deramiocel, a cell therapy for Duchenne muscular dystrophy cardiomyopathy. Deramiocel, using cardiosphere-derived cells, aims to treat heart muscle deterioration. Supported by phase 2 trial data, it seeks priority review for faster approval. Capricor collaborates with Nippon Shinyaku for U.S. distribution.

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.