Caprock Mining Corp

GeneDx, rebranded from Sema4 in 2023, has seen a 3,600% stock surge due to a successful pivot to whole exome and genome sequencing, focusing on rare disease diagnosis in children. The company projects $255-265 million in revenue for 2024 and aims for profitability in 2025, supported by a $3 billion market for rare disease testing expected to grow rapidly.

Capricor Therapeutics plans to file a rolling application with the FDA in October for deramiocel, its cell therapy for cardiomyopathy in Duchenne muscular dystrophy (DMD) patients, with a full submission expected by year-end. The company aims to expand the label for skeletal muscle myopathy post-approval. Deramiocel, previously known as CAP-1002, contains cardiosphere-derived cells with regenerative properties, and has shown significant improvements in heart function in clinical trials.

Wave Life Sciences' stock surged over 50% after Phase II trial data showed substantial dystrophin expression and safety of WVE-N531 in DMD patients, prompting plans for accelerated FDA approval by Q1 2025.

Capricor Therapeutics plans to file a Biologics License Application (BLA) in October 2024 for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy, based on existing cardiac and natural history data. The initial label will include all DMD cardiomyopathy patients, with potential label expansion for skeletal muscle myopathy post-approval.

Capricor Therapeutics plans to file a Biologics License Application for deramiocel, a treatment for Duchenne muscular dystrophy cardiomyopathy, starting October 2024. The company aims for full approval and is considering expanding the treatment to DMD skeletal muscle myopathy, combining trial cohorts for a stronger study.

Capricor Therapeutics plans to file a Biologics License Application (BLA) for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy, based on existing cardiac and natural history data. The BLA submission is expected by end of 2024, with a rolling submission starting October 2024. Deramiocel has shown potential in clinical trials to attenuate DMD's cardiac implications.

Capricor Therapeutics announced an investor webcast to discuss FDA interactions and updates on deramiocel (CAP-1002), a Phase 3 candidate for Duchenne muscular dystrophy. The company also explores exosome-based therapies via its StealthX™ platform. Capricor has a commercialization agreement with Nippon Shinyaku for deramiocel in the U.S. and Japan, pending approval.

The Global Exosome Market: Size, Forecast, Trials, and Trends, 2024 report details the growing exosome market, highlighting advancements in diagnostics, therapeutics, and cosmetics. Exosomes, extracellular vesicles, are valuable for disease detection, diagnosis, and treatment. The market is expanding with 388 clinical trials underway, and first exosome therapeutics expected by 2029. Key players and technologies are profiled, along with market forecasts through 2030.