NIPPON SHINYAKU CO

The FDA approved obe-cel for r/r B-cell precursor acute lymphoblastic leukemia, while clinical holds were removed for CARsgen's zevor-cel, satri-cel, and CT071 trials. A patient in Beam Therapeutics' BEACON trial died due to busulfan-conditioning, not BEAM-101. Neurogene's NGN-401 showed positive results in Rett syndrome but had a treatment-related SAE. Abeona's pz-cel BLA for recessive dystrophic epidermolysis bullosa was accepted. A study found improved mRNA delivery with ZIP-LNPs. Atamyo's ATA-200 received FDA clearance for LGMD2C/R5. Nippon Shinyaku acquired U.S. and Japanese rights for Atsena's ATSN-101 for LCA1.

Atsena Therapeutics and Nippon Shinyaku collaborate on exclusive commercialization of ATSN-101, a gene therapy for Leber congenital amaurosis (LCA1), in the U.S. and Japan. Atsena retains global rights except in these regions, receiving upfront payments, milestones, and royalties. The partnership aims to advance ATSN-101 through a pivotal trial and potential approval.

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.

DelveInsight's 'Duchenne Muscular Dystrophy (DMD) Pipeline Insight, 2024' analyzes over 75 pipeline drugs from 75+ companies, focusing on recent FDA, EMA, and PMDA approvals, clinical trials, emerging therapies, and key players like Roche, Santhera, and Sarepta. The report covers various stages of development, routes of administration, and mechanisms of action, offering insights into the evolving DMD therapeutics landscape.

Capricor Therapeutics closed a public offering of 5,073,800 shares, grossing $86.3 million, to fund product development, manufacturing, and general corporate purposes. Piper Sandler & Co. and Oppenheimer & Co. Inc. managed the offering, which was under a shelf registration statement filed with the SEC.

Capricor Therapeutics closed a public offering of 5,073,800 shares, raising $86.3 million. Proceeds will fund product development, manufacturing, and general corporate purposes. Piper Sandler & Co. and Oppenheimer & Co. Inc. managed the offering.

NS Pharma's Galactic53 study of VILTEPSO® (viltolarsen) for Duchenne muscular dystrophy published in Scientific Reports. Study shows improved pulmonary function in both ambulatory and nonambulatory participants, with higher percent predicted forced vital capacity (FVC%p) and peak cough flow (PCF) at Week 49 compared to controls. Viltolarsen treated participants also showed stabilized arm strength and mobility.

Capricor Therapeutics presents positive three-year safety and efficacy data from Phase II HOPE-2 trial for DMD cell therapy, deramiocel, and initiates BLA submission to FDA. Deramiocel shows sustained cardiac and skeletal muscle benefits, with potential for long-term efficacy.

Capricor Therapeutics announced positive three-year results from its HOPE-2 study for deramiocel, a treatment for Duchenne muscular dystrophy (DMD), showing sustained efficacy and safety, particularly in stabilizing cardiac and skeletal muscle function. The company plans to submit a Biologics License Application to the FDA by the end of 2024, aiming for fast-track approval. Deramiocel has received Orphan Drug Designation and is supported by the Regenerative Medicine Advanced Therapy Designation.

Nippon Shinyaku announces publication of Phase II Galactic53 trial data on VILTEPSO® (viltolarsen) injection for Duchenne muscular dystrophy in Scientific Reports. The study, evaluating safety and efficacy, showed mild to moderate treatment-emergent adverse events and suggested improvements in pulmonary function.