Lifeboat Foundation

2023 was the year that CRISPR gene-editing sliced its way out of the lab and into the public consciousness—and American medical system. The Food and Drug Administration recently approved the first gene-editing CRISPR therapy, Casgevy (or exa-cel), a treatment from CRISPR Therapeutics and partner Vertex for patients with sickle cell disease. This comes on the heels of a similar green light by U.K. regulators in a historic moment for a gene-editing technology whose foundations were laid back in the 1980s, eventually resulting in a 2020 Nobel Prize in Chemistry for pioneering CRISPR scientists Jennifer Doudna and Emmanuelle Charpentier.

That decades-long gap between initial scientific spark, widespread academic recognition, and now the market entry of a potential cure for blood disorders like sickle cell disease that afflict hundreds of thousands of people around the world is telling. If past is prologue, even newer CRISPR gene-editing approaches being studied today have the potential to treat diseases ranging from cancer and muscular dystrophy to heart disease, birth more resilient livestock and plants that can grapple with climate change and new strains of deadly viruses, and even upend the energy industry by tweaking bacterial DNA to create more efficient biofuels in future decades. And novel uses of CRISPR, with assists from other technologies like artificial intelligence, might fuel even more precise, targeted gene-editing—in turn accelerating future discovery with implications for just about any industry that relies on biological material, from medicine to agriculture to energy.

With new CRISPR discoveries guided by AI, specifically, we can expand the toolbox available for gene editing, which is crucial for therapeutic, diagnostic, and research applications… but also a great way to better understand the vast diversity of microbial defense mechanisms, said Feng Zhang, another CRISPR pioneer, molecular biologist, and core member at the Broad Institute of MIT and Harvard in an emailed statement to Fast Company.

For instance, the pegRNA molecules used in prime editing are difficult and expensive to chemically synthesise or laborious to clone, which hampers the crucial optimisation of prime-editing efficiency. Additionally, the reverse transcriptase (RT) enzymes used in prime editing are relatively error-prone and have low processivity, which may limit the precision and size of edits that can be introduced. Furthermore, RTs have a low affinity for dNTPs, which can impact prime-editing efficiency in non-dividing and differentiated cells.

To address these issues, two research groups led by Dr. Ben Kleinstiver at Mass General Hospital (MGH) & Harvard Medical School, and Dr. Erik Sontheimer at the RNA Therapeutics Institute (UMass Chan Medical School) have independently developed new approaches that build upon prime editing by replacing RT with another type of enzyme, namely a DNA-dependent DNA polymerase. This change permits the use of DNA instead of RNA as a template for editing, potentially addressing some of the main limitations of prime editing by allowing higher efficiency and adaptability.

UCLA breaks new ground in alloy research, presenting the first 3D mapping of medium and high-entropy alloys, potentially revolutionizing the field with enhanced toughness and flexibility in these materials.

Alloys, which are materials such as steel that are made by combining two or more metallic elements, are among the underpinnings of contemporary life. They are essential for buildings, transportation, appliances and tools — including, very likely, the device you are using to read this story. In applying alloys, engineers have faced an age-old trade-off common in most materials: Alloys that are hard tend to be brittle and break under strain, while those that are flexible under strain tend to dent easily.

Advancements in Alloy Research.

USC Dornsife’s CReATiNG technique revolutionizes synthetic biology by facilitating the cost-effective construction of synthetic chromosomes, promising significant advancements in various scientific and medical fields.

A groundbreaking new technique invented by researchers at the USC Dornsife College of Letters, Arts and Science may revolutionize the field of synthetic biology. Known as CReATiNG (Cloning Reprogramming and Assembling Tiled Natural Genomic DNA), the method offers a simpler and more cost-effective approach to constructing synthetic chromosomes. It could significantly advance genetic engineering and enable a wide range of advances in medicine, biotechnology, biofuel production, and even space exploration.

Simplifying Chromosome Construction

Our research at Vertex is built upon two strong pillars: biology and therapeutic innovation. We continue to fill our drug discovery toolbox with cutting-edge tools and technologies. One of these tools is CRISPR/Cas9 gene editing. Watch this video to learn about how CRISPR/Cas9 gene editing works and how it can be used in therapeutic development.\

For company updates and to learn more about Vertex Pharmaceuticals, follow us on Twitter (/ vertexpharma, YouTube (/ @vertexpharmaceuticalsglobal) and LinkedIn (/ vertex-pharmaceuticals, or visit our website at www.vrtx.com.

Europe’s health regulator followed the US and UK in backing the first gene-editing therapy to use Crispr technology, a Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG treatment for sickle cell disease.

The European Medicines Agency’s expert panel recommended on Friday authorizing the Vertex and Crispr drug, Casgevy, for people with severe sickle cell disease and another serious hereditary blood disorder, beta-thalassemia, which is traditionally treated with repeated transfusions. Vertex said before the ruling that it had yet to establish a European list price for the one-time therapy, which costs $2.2 million in the US.

The treatment makes precisely targeted changes in patients’ DNA, a months-long process that requires removing bone marrow and a stem cell transplant. In Europe, Vertex said its initial focus will be on countries with the highest numbers of patients, including France, Italy, the UK and Germany.

All living systems perpetuate themselves via growth in or on the body, followed by splitting, budding, or birth. We find that synthetic multicellular assemblies can also replicate kinematically by moving and compressing dissociated cells in their environment into functional self-copies. This form of perpetuation, previously unseen in any organism, arises spontaneously over days rather than evolving over millennia. We also show how artificial intelligence methods can design assemblies that postpone loss of replicative ability and perform useful work as a side effect of replication. This suggests other unique and useful phenotypes can be rapidly reached from wild-type organisms without selection or genetic engineering, thereby broadening our understanding of the conditions under which replication arises, phenotypic plasticity, and how useful replicative machines may be realized.

Newly approved CRISPR treatment can change a person’s “blueprint.”

The model can help evolve “better methods for growing cells for blood transfusions, novel cell therapies, and hematopoietic stem cell transplants.”

Remarkably, heX-Embryoid models developed structures akin to blood islands, the initial sites supporting the generation of blood cells in developing embryos. The study identified progenitors for red blood cells, platelets, and various white blood cell types—a pivotal advancement in the field, according to the team.

Researchers claim the model successfully replicated a process closely resembling the initial stages of blood production in humans. “This is exciting because there are extensive possibilities to apply this model to better understand how blood is formed and develop better methods for growing cells for blood transfusions, novel cell therapies, and hematopoietic stem cell transplants,” said Mo Ebrahimkhani, senior author and an associate professor at the Pittsburgh Liver Institute and the Department of Bioengineering at Pitt, in a statement.

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