News
Your source for the latest updates, breakthroughs, and insights in drug development and manufacturing. Explore our featured stories, press releases, and event announcements to stay informed about our innovations and contributions to drug development and manufacturing advancements.
FDA approves the first gene therapy for people with beta-thalassemia who require regular red blood cell transfusions
bluebird bio has announced the the U.S. Food and Drug Administration (FDA) has approved ZYNTEGLO® (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who [...]
How gene therapy can cure or treat diseases
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 [...]
ARM and NIIMBL Release Project A-Cell to Bring Quality by Design Principles to Cell-Based Therapy Manufacturing
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 [...]
New next-gen sequencing technique could accelerate and improve mRNA therapies by quantifying how LNPs deliver mRNA into cells
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 [...]
New method combines a T-cell “barcode” with single-cell RNA sequencing to enhance CAR T–cell therapy
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 [...]
Sana Biotechnology to expand its manufacturing, advance cell-based therapies in neurological disorders
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 [...]