Researchers report on the safety of a gene therapy to treat the common autosomal recessive hereditary disorder alpha 1-antitrypsin (AAT) deficiency in a new article in the peer-reviewed journal Human Gene Therapy. In ATT deficiency, neutrophil proteases destroy the lung parenchyma, the portion of the lungs...
In the study, published in Nature Biomedical Engineering, researchers from MD Anderson Cancer Center use EV-encapsulated mRNA to initiate and sustain collagen production for several months in the cells of photoaged skin in laboratory models. It is the first therapy to demonstrate this ability and represents a proof-of-concept for deploying the EV mRNA therapy...
Company files its first Investigational New Drug application for its lead program KYV-101, a novel fully human CD19 CAR T-cell therapy, for the treatment of lupus nephritis. Kyverna’s therapeutic platform combines advanced T-cell engineering and synthetic biology technologies to suppress and eliminate...
Although immunotherapy has revolutionized the treatment of many blood cancers, the field has encountered challenges developing similar treatments for solid tumors. When it comes to pediatric brain tumors, which are the leading cause of cancer death in children, challenges include...
A recent study has shown remarkable results from compassionate use of CAR-T cell therapy to treat severe lupus in five patients in whom multiple other treatments had previously failed. CAR-T cell therapy involves genetically engineering T cells, a type of immune cell, so that they respond to a prespecified molecular marker, or antigen. This means they mount an immune response against cells with the chosen antigen...
Twice a year, the Alliance for Regenerative Medicine (ARM) surveys the cell and gene therapy landscape and ARM’s initiatives to advance the sector and provide an update to their members, their stakeholders, and the public at large. While external conditions ebb and flow, the sector’s progress — in clinical milestones, regulatory developments, and patient impact — steadily advances...
Century Therapeutics, an innovative biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies in immuno-oncology, announced today that the company has been notified by the U.S. Food and Drug Administration (FDA) that the Company’s ELiPSE-1 clinical study may proceed to assess CNTY-101 in patients with relapsed or refractory CD19 positive B-cell malignancies. CNTY-101 is [...]
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 [...]
