News

The genes in your body’s cells play a key role in your health. Indeed, a defective gene or genes can make you sick. Recognizing this, scientists have worked for decades on ways to modify genes or replace faulty genes with healthy ones to treat, cure, or prevent a disease or medical condition. [...]

Effort to address challenges to the manufacturing scale-up of cell-based therapies follows release of Project A-Gene for gene therapy in 2021 The Alliance for Regenerative Medicine (ARM) and the National Institute for Innovation in Manufacturing BioPharmaceuticals (NIIMBL) today released Project A-Cell, a multistakeholder collaboration to incorporate Quality by Design (QbD) principles into a manufacturing case [...]

Therapeutics based on messenger RNA, or mRNA, can potentially treat a wide range of maladies, including cancer, genetic diseases, and as the world has learned in recent years, deadly viruses. To work, these drugs must be delivered directly to target cells in nanoscale bubbles of fat called lipid nanoparticles, or LNPs — mRNA isn’t [...]

Scientists at St. Jude Children’s Research Hospital have developed a method that may be able to improve CAR T-cell therapies by identifying the early cells that become effective at killing cancer. St. Jude Children’s Research Hospital scientists have demonstrated that the long-term performance of cancer-killing chimeric antigen receptor (CAR) T cells can, in some cases, be predicted [...]

The University of Rochester Medical Center (URMC) and Sana Biotechnology have entered into an agreement which will allow Sana to expand its manufacturing footprint and create new job opportunities in Rochester. Sana, a company focused on creating and delivering engineered cells as medicines, also operates in Seattle, Cambridge, and South San Francisco. The company has programs [...]

A paper from the Horae Gene Therapy Center at UMass Chan Medical School may settle a longstanding debate about how to best manufacture adeno-associated virus (AAV) vectors for gene therapy. The paper, published online in the journal Human Gene Therapy, describes using AAV genome population sequencing (AAV-GPseq) to explore the genomes of vectors produced by two [...]