The appointment May 6 of Vinay Prasad as the head of the U.S. FDA’s Center for Biologics Evaluation and Research (CBER) “bodes poorly” for Sarepta Therapeutics Inc.’s development-stage pipeline, said Wainwright analyst Mitchell Kapoor – and Wall Street reflected as much, as the stock (NASDAQ:SRPT) ended that day down 26.6% vs. an XBI drop of 6.6% – this ahead of the after-hours earnings disclosure that pushed the Cambridge, Mass.-based firm down even farther by more than another 20%, with the XBI unchanged.
U.S. FDA Commissioner Marty Makary is starting to fill the vacancies at the agency that’s seen its senior leadership ravaged by retirements and terminations. Makary’s first pick is Vinay Prasad as the new head of the FDA’s Center for Biologics Evaluation & Research (CBER), the center that oversees vaccines, blood products, allergenics and cellular, tissue and gene therapies.
Onward Therapeutics SA’s subsidiary Emercell SAS has received investigational medicinal product dossier (IMPD) approval from the EMA to initiate a phase I trial of OT-C001, an allogeneic natural killer (NK) cell therapy, in combination with an anti-CD20 monoclonal antibody, rituximab.
Mesenchymal stem cells (MSCs) have long been recognized for their potential in cancer therapy. However, the effectiveness of MSCs in cancer treatment has been hampered by their limited tumor-homing ability and the heterogeneity of tissue-derived MSCs. To address these challenges, researchers have focused on induced pluripotent stem cell (iPSC)-derived MSCs, which offer improved homogeneity and expansion potential.
Stem cell implantation is a step closer to becoming the next strategy against Parkinson's disease. Two clinical trials, one in phase I and the other in phase I/II, have demonstrated their safety and potential to restore dopamine production in the brains of patients with this currently incurable neurodegenerative condition. The number of participants in the study is still small, and further research is needed to demonstrate the clinical benefits of these cell therapies.
Xellsmart Biopharmaceutical (Suzhou) Co. Ltd. has announced IND clearances by the FDA for its XS-411 and XS-228 cell therapies, for Parkinson’s disease and amyotrophic lateral sclerosis (ALS), respectively.
Sumitomo Pharma Co. Ltd. announced that it will sell off two more of its subsidiaries, Sumitomo Pharma (China) Co. Ltd. and Sumitomo Pharma Asia Pacific Pte. Ltd. (and their subsidiaries), to Marubeni Global Pharma Corp. April 1, as the Japanese pharma continues restructuring efforts from last year.
Cartesian Therapeutics Inc. followed up December’s phase IIb data with more good news regarding Descartes-08, offering 12-month efficacy and safety results that whetted Wall Street’s appetite for the phase III Aurora effort ahead. Milos Miljkovic, chief medical officer, told BioWorld that minimum symptom expression – among the areas where Descartes-08 shone, providing relief for “months and months after the last dose of treatment” – is especially important to patients.
Neurona Therapeutics Inc. has completed an upsized, oversubscribed $102 million financing round, the third company to do so in a month that’s only four days old. The privately held company’s lead asset is NRTX-1001, a regenerative cell therapy derived from human pluripotent stem cells.
Sumitomo Pharma Co. Ltd. announced that it will sell off two more of its subsidiaries, Sumitomo Pharma (China) Co. Ltd. and Sumitomo Pharma Asia Pacific Pte. Ltd. (and their subsidiaries), to Marubeni Global Pharma Corp. April 1, as the Japanese pharma continues restructuring efforts from last year.
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