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...
With several years of experience supporting a world-renowned mRNA COVID-19 vaccine company with raw material and drug substance release, Avance Biosciences™ is well-positioned to provide industry-leading assay design, validation, and sample testing services that advance our clients’ mRNA therapeutic...
The goal of the hypoimmune platform is to overcome the immunologic rejection of allogeneic cells, which if true for SC291 may result in longer CAR T cell persistence and a higher rate of durable complete responses for patients with B-cell lymphomas or leukemias. The hypoimmune platform includes disruption of major...
ASGCT held its fifth annual liaison meeting with FDA CBER’s Office of Tissues and Advanced Therapies (OTAT) on Nov. 14, 2022. A group of Society leaders and members gave two presentations to FDA on significant topics in the field, followed by a presentation from FDA. Dr. Keith Wonnacott, ASGCT’s Regulatory Affairs Committee Chair, chaired the meeting and moderated discussion...
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...
Avance offers a broad range of assay development services, including assay design, assay validation, sample testing, and technology transfer under GLP compliance to support IND-enabling and clinical studies. With decades of experience helping gene and cell therapy companies, Avance Biosciences™ has the...
Quantitative analysis of gene editing is now an essential step in getting regulatory approval of gene editing-based therapies. But designing and validating assays for on/off-target editing analysis and building an internal team to perform them can be costly and inefficient...
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...
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...
