Genome Editing

Synthetic Organisms Self-Replicate in a New Way

A team of biologists and computer scientists show clusters of cells when freed from their original context in a developing organism, can find and combine loose cells into clusters that look and move like the original ‘parent’ ones. This new method of self-replication does not require specific evolution or genetic engineering. The scientists use artificial intelligence to design clusters with complex shapes that replicate better than the original spheroidal ones. These living machines can perform useful work as they self-replicate.
Genetic engineering, conceptual illustration

CRISPR Engineers a New Path for Antibiotic Production

New methods to develop novel antibiotics are desperately needed. Now, researchers have discovered a way to manipulate key assembly line enzymes in bacteria using CRISPR-Cas9 gene editing. They show that this method can re-engineer biosynthetic assembly lines such as nonribosomal peptide synthetases (NRPS) and related megasynthase enzymes. The technique could be used to develop future medicines to combat emerging drug-resistant pathogens—one of today’s biggest threats.

3D Bioreactor Shows Promise for Donor Cell Therapies

Rigenerand, which is working on cGMP gene and cell therapies, developed a 3D bioreactor design for its own use. However, company officials soon began to sell it as a standalone product. The concept is to use it as an accelerator for validating the cell and gene therapy approach, specifically to validate CAR-T products against primary cells or cell lines during research and development.
Gene therapy concept. Medical technology. Medtech.

New Facility Offers Quality Control for Gene and Cell Therapies

A Swedish bioanalytical services company is poised to open a Good Laboratory Practice-compliant laboratory for gene and cell therapies. TATAA Biocenter says the new facility will be among the first in the world to specialize in quality control (QC) analytics for nucleic acid-based therapies. To meet the sensitivities required for molecular analysis, the new facility has custom-built laboratory spaces.
WAVE bioreactor for Laboratory Reactor

Advanced Therapies Need 4.0

Industry 1.0--the industrial revolution--was characterized by the replacement of humans by machines for manual work. Industry 2.0 involved processes and automation, followed by the current paradigm, 4.0, which is about capturing and managing information for decision making, extreme automation, and real-time monitoring. On the basis of that evolution, Biopharma 4.0 and advanced therapies are a natural fit.
Illustration of human blood cells

Hemophilia Gene Therapy Trial Achieves Sustained Clotting Factor Levels

Results from a multicenter international phase 1/2 clinical trial are the first to show improved production of coagulation factor VIII over prolonged period, leading to reduction or complete elimination of bleeding events. The trial infuses SPK-8011, a new recombinant AAV vector engineered to express factor VIII in human liver cells, in 18 men with hemophilia A and demonstrates a 91.5% decrease in bleeding episodes.
Scientist in a clean room

An Evolving CDMO Landscape

Drug innovators with in-house production capability must consider how their existing capacity and experience will influence their use of CDMOs. The reason an internal assessment is important is that behavioral differences arising from outsourcing drivers can affect which outsourcing approach-tactical, partnership, whatever—to take. Moreover these drivers differ based on product type.
Pilot fermenter system

Australia Needs to Invest to Tap Demand for Cell and Gene Therapy Production

Australia can become a regenerative medicines hub if manufacturers are willing to invest in innovative technologies and capacity says an industry group, Ausbiotech. Sustained industry interest is a significant opportunity for Australian because it has been estimated that the country could generate at least $6 billion in annual revenue and create 6,000 new jobs by 2035.
DNA Strands 3D Illustration

Decentralized Advanced Therapy Processing Rolls Out

A company pioneering a decentralized business model for processing cell and gene therapies claims to have quintupled their capacity in the last year. Orgenesis’ business model involves working in partnership with hospital research centers and small biotech companies to develop and commercialize advanced therapies. These can be licensed back to other organizations in the Orgenesis network.
Duchenne muscular dystrophy (DMD)

The Problem with Clinical Trials in Duchenne—and What We Can Do About It

Duchenne Muscular Dystrophy (DMD) is a rare and fatal inherited disorder caused by mutations in the massive gene for dystrophin, a protein crucial to normal muscle cell function. The disease is X-linked, so patients are mostly boys. A wealth of innovation over the past decade has brought more than 20 therapeutic programs into clinical trials for DMD. Sadly, most disease-modifying clinical candidates for DMD so far have either been discontinued or have disappointed.