Lifeboat Foundation

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.

Continue reading “Scientists craft embryo model mimicking early human blood development” »

Armed with gene technologies and CAR-Ts, scientists are attempting to eliminate viruses that escape immune detection and lurk in tissues for years.

Biotech stocks could be at a major turning point after FDA approval of the first gene editing drug using CRISPR technology to treat sickle cell disease. Gove…

A team of scientists used gene editing to create what they thought would be a calmer rodent. Instead, the gene-edited rodents were angrier.

A new Seattle biotech organization will be funded to the tune of $75 million to research “DNA typewriters,” self-monitoring cells that could upend our understanding of biology. The collaboration between the University of Washington, the Chan-Zuckerberg Initiative and the Allen Institute is already underway.

Called the Seattle Hub for Synthetic Biology, the joint initiative will combine the expertise of the two well-funded research outfits with that of UW Medicine, working in what UW’s Jay Shendure, scientific lead for the project, called “a new model of collaboration.”

The Hub (not to be confused with the HUB, or Husky Union Building, on UW’s campus) aims to strike a balance between a disinterested intellectual academic approach and a development-focused commercial approach. The $75 million will fund the organization for five years, with the option to renew then.

These three gene therapy stocks should get huge boosts over the long term from the use of gene editing techniques to treat diseases.