Beyond Baby KJ: manufacturing lessons for the next CRISPR cures

The coordinated effort that last spring saved the life of little KJ Muldoon earned widespread and enthusiastic media coverage. But between the invention of the treatment and its delivery to the patient lay a lesser-told story: an unprecedented manufacturing sprint. Genetic Engineering & Biotechnology News organized an online roundtable led by its deputy editor in chief, Julianna LeMieux, to discuss how therapeutic components were produced quickly, cost-effectively, and to clinical-grade standards.

Continue reading

CRISPR-GPT: a copilot for editing

Nature Biomedical Engineering has introduced a chatbot specifically designed to help beginners with their first experiments and to support experienced researchers in their work.

Since it was first described in Science in 2012, in the landmark paper by Emmanuelle Charpentier and Jennifer Doudna, the success of the CRISPR technique has been summed up with a handful of adjectives: cheap, precise, easy to use. But since everything is relative, it’s worth asking: how easy, and compared to what? When measured against previous genetic editing platforms, CRISPR is far simpler to apply. Whereas only a few highly specialized centers could once perform these experiments, with CRISPR a standard lab, the basic skills of an ordinary biologist, and solid familiarity with bioinformatics may be enough. Still, novices need guidance, and even seasoned researchers can run into problems.

Continue reading

A Cambrian explosion for CAR-T

Credit Mesa Shumacher/Santa Fe Institute

Around 500 million years ago, life on Earth underwent a phase of rapid diversification that led to the formation of complex biological structures and the appearance of new groups of organisms. This crucial event for evolution captured the imagination so strongly that it became a metaphor. ‘CARs in 2025: the Cambrian explosion continues’, in fact, is the title chosen by Michel Sadelain for the lecture hosted by GEN (Genetic Engineering & Biotechnology News) on 29 January. The most exciting branch of immunotherapy, using engineered T lymphocytes to efficiently and selectively attack cancer cells, is experiencing a boom in new approaches and applications.

Continue reading

A multiple sclerosis trial and more CRISPR news

Anyone interested in advanced therapies is familiar with the acronym CAR-T. These are T lymphocytes modified (also with the help of CRISPR) to better recognize and attack cancer cells, and they have already proven to be a successful strategy for blood tumors. Now hopes are high that a similar approach may also prove useful for multiple sclerosis, which is an autoimmune disease. The idea is to use CAR-Ts to prevent B lymphocytes from attacking nerve cells, including in the brain. The first clinical trial is recruiting patients in the U.S. Read more in Nature.

Let’s come to the use of New Genomic Techniques in crops. The European Commission’s regulatory proposal (approved by the EU Parliament on Feb. 7) excludes the use of edited plants in organic farming, but among organic producers not everyone is against NGTs and this may bode well for a possible peaceful coexistence between the different types of production in the years to come.

Finally, we point out the latest advance in animal editing: porcine virus-resistant pigs. The paper came out in the CRISPR Journal, but you can also read about it in GEN.