Stem-cell based therapies to strengthen the heart muscle and treat other diseases are beginning to show promise in human clinical trials. However, other than observing clinical outcomes, lack of a repeatable, time-sensitive and noninvasive tool to assess the effectiveness of the transplanted cells within the target organ has slowed progress in the stem cell field.
Johns Hopkins researchers have developed a way to map retinal cell development and potentially advance regenerative medicine Johns Hopkins Medicine researchers report they have created a method of mapping how the central nervous system develops by tracking the genes expressed in cells. The technique, demonstrated in mouse retinas for this study, follows the activity of
Source Future Medicine Mesenchymal stem cells (MSCs) are in development for many clinical indications, based both on ‘stem’ properties (tissue repair or regeneration) and on signaling repertoire (immunomodulatory and antiinflammatory effects). Potential conflation of MSC properties with those of tissue-derived stromal cells presents difficulties in comparing study outcomes and represents a source of confusion in
An international commission has been convened by the U.S. National Academy of Medicine (NAM), the U.S. National Academy of Sciences (NAS), and the Royal Society of the U.K., with the participation of science and medical academies around the world, to develop a framework for scientists, clinicians, and regulatory authorities to consider when assessing potential clinical
Source RAPS Addressing manufacturing controls for the cell and gene therapy industry, this article discusses criticality of establishing Chemistry Manufacturing Controls (CMC) Readiness, Critical Quality Attributes (CQAs) and Critical Process Parameters (CPPs) for cell and gene therapy products. The author suggests manufacturers need sound drug development and manufacturing facility plans covering a product’s lifecycle and
Source Nature From antibiotics and organoids to CRISPR, improved biomedical methods and apparatus are enabling new therapies. Organoids Mini organs stand in for test models The term ‘organoid’ didn’t exist a decade ago. Now it’s a buzzword in biomedical science for a three-dimensional multi-cellular structure or ‘mini organ’. Thanks to advances with pluripotent stem cells
Source The Economist Western countries, as well as poor ones, are demanding transparency in the cost of drugs THESE DAYS it is hard to find a government that is not struggling with the high price of medicines. In England, the government is fighting Vertex, a drug company, over the cost of a drug for cystic
In Nov. 2018, The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) published technology roadmaps addressing needs and gaps in three key product areas: gene therapy, antibody-dug conjugates, and vaccines. The roadmaps were developed with the collaborative input of industry, academic, and government experts. This piece highlights technology opportunities for gene therapy manufacturing. The NIIMBL
Machine learning algorithms predict the repairs made to DNA after Cas9 cuts. The papersM.W. Shen et al., “Predictable and precise template-free CRISPR editing of pathogenic variants,” Nature, 563:646–51, 2018. F. Allen et al., “Predicting the mutations generated by repair of Cas9-induced double-strand breaks,” Nat Biotechnol, 37:64–72, 2019. During gene editing with CRISPR technology, the Cas9 scissors that
Since our 2018 year-end post on the busy FDA regulatory agenda, we are nearing the halfway mark of the “grace period” the Agency has extended for certain regenerative medicine product developers to come into compliance by obtaining investigational new drug applications (INDs) and working towards premarket approval of their products. Recent public statements by FDA officials reiterating
