The FDA has awarded orphan drug designation to Papillon Therapeutics Inc.’s PPL-002, an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, for the treatment of Danon disease.

Papillon Therapeutics Inc.’s PPL-001 has been awarded orphan drug designation by the FDA for Friedreich’s ataxia. PPL-001 is an experimental gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy.

Iron regulates the metabolism of hematopoietic stem cells (HSCs) and acts as a genetic control of their fate, preserving their identity and regenerative capacity during tissue maintenance and repair. A group of scientists at Albert Einstein College of Medicine has described the key components of a molecular pathway that iron regulates. “What we are proposing here with this mechanism is that iron serves like a switchboard and a sensor,” senior author Britta Will told BioWorld. Will is at the Department of Oncology, the Cell Biology Department, and the Ruth and David Gottesman Institute for Stem Cell Research and Regenerative Medicine at Albert Einstein College of Medicine.

A study from Weill Cornell Medicine and The Jackson Laboratory has described the epigenetic mark SARS-CoV-2 left on immune system stem cells in the most severe cases of COVID-19 early in the pandemic, before the development of vaccines. In their work published in Cell on Aug. 18, 2023, the researchers presented a new methodology to analyze the epigenetic changes in monocytes and circulating hematopoietic stem and progenitor cells (HSPCs) that give rise to monocytes. That allowed corresponding author Steven Josefowicz and his colleagues to see if there were already changes induced by COVID-19 before HSPCs differentiated into monocytes.

A study led by scientists at Osaka University Graduate School of Medicine has identified the previously unknown molecular mechanism underlying bone marrow regeneration after chemotherapy, which damages hematopoietic stem and progenitor cells.