Gene Therapy News
Gene therapy is rapidly advancing, offering new hope for treating rare diseases, cancers, and genetic disorders. This page provides a curated collection of the most important news in gene therapy drug development, from clinical trial milestones to regulatory updates worldwide. Our goal is simple: to keep you informed on the latest breakthroughs and developments shaping the future of gene-based medicine.
The guidance provides recommendations regarding information that should be provided in an investigational new drug (IND) application to assess the safety and quality of the investigational GE product, including information on product design, product manufacturing and testing, nonclinical safety...
In a landmark move, the FDA has initiated the Collaboration on Gene Therapies Global Pilot (CoGenT), signaling a significant leap toward worldwide collaboration in the field of gene therapy. This pilot aims to enable concurrent collaborative reviews of new gene therapy applications with global regulators…
In this draft guidance, the FDA emphasizes that potency assays and their corresponding acceptance criteria should be designed to make meaningful contributions to potency assurance by reducing risks to product potency. They provide illustrative examples of approaches to potency assay development that are...
In a groundbreaking development, the U.S. Food and Drug Administration (FDA) has approved two revolutionary treatments, Casgevy and Lyfgenia, marking a significant milestone in the field of gene therapy. These therapies represent the first-ever cell-based gene treatments for sickle cell disease (SCD) in patients...
We are excited to announce that Avance Biosciences™ will be attending the highly anticipated Gene Therapy Analytical Development Summit and the mRNA Assay Development Summit, and we cordially invite you to join us! As a leader in development and validation of assays for advanced therapies...
LEXEO Therapeutics will use the net proceeds from this offering to advance the clinical development of cardiac and neurological disease gene therapies. LEXEO Therapeutics, in its SEC filing, said it intends to use the net proceeds from this offering, together with their existing cash, to advance the clinical development of LX2006, LX2020 and LX1001...
We are thrilled to announce that Avance Biosciences™ will be attending the highly anticipated Cell & Gene Therapy Manufacturing Conference, and we cordially invite you to join us! As a leader in advanced therapies, we understand the importance of staying at the forefront of cell and gene therapy innovation...
We are thrilled to announce that Avance Biosciences™ will be attending the highly anticipated Gene Therapy Potency Assay Summit, and we cordially invite you to join us! As a leader in advanced therapies, we understand the importance of staying at the forefront of gene therapy innovation...
Specifically, the guidance recommends conducting analytical comparability studies to provide scientific evidence of the impact that manufacturing changes may have on the safety, potency, and purity of human cellular and gene therapy products...
A form of gene therapy currently used to treat Parkinson’s disease may dramatically reduce alcohol use among chronic heavy drinkers, researchers at Oregon Health & Science University and institutions across the country have found...