Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.

The promise of mesenchymal stem cells (MSCs) to heal heart disease could be around the corner thanks to a new delivery method tested by regenerative medicine company Cynata Therapeutics Ltd.

The promise of mesenchymal stem cells (MSCs) to heal heart disease could be around the corner thanks to a new delivery method tested by regenerative medicine company Cynata Therapeutics Ltd.

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.

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.