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
Athersys, Inc. (Nasdaq: ATHX) announced today that its clinical program evaluating MultiStem® cell therapy for the treatment of Acute Respiratory Distress Syndrome (“ARDS”) has received Fast Track designation from the United States Food and Drug Administration (“FDA”). This important designation is given to qualified investigational therapies that show promise in providing benefit to patients in
Source: Reuters Novartis AG is offering price discounts in negotiations with U.S. health insurers on its gene therapy for spinal muscular atrophy (SMA), a treatment that could cost more than a million dollars, but the gesture comes with strings attached. The Swiss drugmaker wants insurers to commit to coverage for patients identified with the rare
A new ‘biological glue’ developed by Chinese researchers could one day stop hearts and arteries from uncontrollable bleeding. Placed on an open wound and activated through UV light, this life-saving sealant rapidly morphs into a non-toxic hydrogel, capable of withstanding both slippery surfaces and the pulsing of heart tissue. The adhesive sealant hasn’t been tested
While therapies that harness the power of the immune system against cancer have made remarkable progress against certain types of tumours, they still remain ineffective in most cancer patients. A new study has described a method of reprogramming the regulatory T cells that usually suppress immune responses into inflammatory cells that not only permit but
Investigators tested the preclinical safety of the innovative implant ARTiCAR for the treatment of osteoarticular lesions. Thanks to the combination of two advanced therapy medicinal products, a polymeric nanofibrous bone wound dressing and bone marrow-derived mesenchymal stem cells, the ARTiCAR promoted both subchondral bone and cartilage regeneration. [Nat Commun] The extended life expectancy and the
Researchers of the Stanford University College of Medicine have just published an important paper on FAP mesenchymal stromal cells in muscle regeneration. The necessity of mesenchymal stromal cells, called fibroadipogenic progenitors (FAPs), in skeletal muscle regeneration and maintenance remains unestablished. We report the generation of a PDGFRαCreER knockin mouse model that provides a specific means of
