Issue 208
Though Vertex and CRISPR Therapeutics’ recent approval for sickle cell disease demonstrates the power of gene editing, our ability to add whole genes into the human genome is lacking.
Offering new promise, a technique developed by UC Berkeley researchers, called PRINT (short for “precise RNA-mediated insertion of transgenes”), directs transgene synthesis directly into the genome at a “safe-harbor” locus. “We’re not knocking out a gene function. We’re not fixing an endogenous gene mutation. We’re taking a complementary approach, which is to put into the genome an autonomously expressed gene that makes an active protein —to add back a functional gene as a deficit bypass,” said Kathleen Collins, study author and professor in the Department of Molecular and Cell Biology at the University of California, Berkeley, in a press release. “It’s transgene supplementation instead of mutation reversal. To fix loss-of-function diseases that arise from a panoply of individual mutations of the same gene, this is great.”
Do you think PRINT will stand the test of time – or will better options supersede its development? Open to opinions: [email protected].
Until next week,
Jamie Irvine | Associate Editor
Essential Reading
Acquisitional Intent
AstraZeneca has completed its acquisition of Gracell Biotechnologies – a biopharma company developing cell therapies for cancer and autoimmune diseases. AstraZeneca will gain access to GC012F, a novel dual-targeting CAR-T therapy against multiple myeloma, as well as other hematological malignancies and autoimmune diseases, including systemic lupus erythematosus. Gracell will operate as a wholly owned subsidiary of AstraZeneca, with operations in China and the US.
RNA Revelation
CAR T therapy has successfully treated blood cancers, including lymphomas and multiple myeloma, but the engineered immune cells haven’t stacked up well against solid cancers, such as pancreatic and lung cancers. Now, researchers from Stanford University have developed an RNA-targeting CRISPR platform solution that could tune immune cell metabolism without permanent genetic changes. The study, published in Cell, suggests the platform – named MEGA (short for multiplexed effector guide arrays) – can modify the RNA of cells, allowing the team to regulate immune cell metabolism in a way that boosts the cells’ ability to target tumors. “Our finding is that it performs 10 times better, in terms of reducing the tumor growth and in terms of sustaining long term T cell proliferation,” said senior author Stanley Qi, associate professor of bioengineering at Stanford and institute scholar at Sarafan ChEM-H.
Worth Your Time...
Research
Canadian-based researchers highlight foundational studies that examine relevant metabolic pathways required for effective CAR T cell cytotoxicity and persistence in human tumor microenvironment.
Researchers explore complexity of NK cell biology in humans and highlight role of these cells in cancer immunity and cell therapies.
Study performs genetic disruption in human CAR T cells and provides evidence that SUV39H1 inactivation elicits potent and durable functional persistence upon multiple tumor rechallenges.
Business
Genezen enters license agreement with CSL for Cytegrity – an inducible producer technology designed for production of lentiviral vectors.
Therapeutic Solutions International files patent on “Facilitating effects” of JadiCells on gene therapy mediated cell differentiation and mesenchymal stem cell therapy.
Janssen receives positive opinion from EMA for Carvykti – a CAR-T therapy against relapsed and refractory multiple myeloma.
