IT'S BEEN A HOT WEEK FOR GENETIC CURES! 🥵

🌁 Bridge recombinases for humans (lnkd.in/g33kE49N): this new kind of genome editing tool was discovered as jumping genes that can insert, excise, and invert many thousands of bp, without releasing cut DNA fragments. When they excise themselves from a genome, the non-coding DNA ends join into a bridge RNA (bRNA) that folds into two loops: one that binds the insertion sequence (IS) itself, another that binds the target DNA, making bRNA a bispecific guide molecule for the target and the donor sequence — Arc Institute just achieved 20% insertion efficiency, genome-wide specificity as high as 82%, inversion of up to 0.93 megabases, and excision of up to 0.13 Mb in human genomes via point mutations and scaffold modification of IS622’s bRNA.

✒️ Stylus Medicine has raised $85M (lnkd.in/g33kE49N): spun off Arc’s bridge recombinase, their initial focus is to create in vivo CAR-T therapies through lipid nanoparticle delivery. They’ve demonstrated rapid and sustained tumor regression of a mouse tumor model. Khosla Ventures, Eli Lilly and Johnson and Johnson are among their investors.

🥊 CRIPSR patent fight ain’t over yet (lnkd.in/g33kE49N): Doudna and Charpentier published work in test tubes and discussed eukaryotic use in emails months before the Broad team published theirs editing eukaryotic cells. The appeals court now says that what matters is not whether they were certain it would work but whether they had a “definite and permanent idea”, so they asked the patent board to take another look at the case.

👶 Baby cured by base editing (lnkd.in/g33kE49N): the 7-month old was born with deficiency of CPS1, a mitochondrial enzyme that catalyzes the first and rate-limiting step of the urea cycle without which ammonia accumulates toxically in the bloodstream and can lead to death. In a matter of months, the cure was tested in a custom-made human hepatocyte cell line, then in transgenic mouse models and nonhuman primates. Since the gene is large, lipid and not AAV delivery was used. One-shot therapy, accelerated. Life saved.

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