Gene Therapy Priced at $2M, Age Differs Across the Body

Spinal muscular atrophy is the most deadly genetic condition in children under 2 years. A gene therapy called Zolgensma could “approach a cure” if given before symptoms develop.

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Good morning. Spinal muscular atrophy is the most deadly genetic condition in children under 2 years. It affects about 1 in 80,000 infants, causing a slow decay of muscles and motor neurons. A gene therapy called Zolgensma, made by Novartis and first licensed in the United States in 2019, could “approach a cure” if given before symptoms develop, according to an article in The Guardian. The therapy is administered as a “single, hour-long intravenous infusion;” a veritable miracle.

There’s just one problem: It costs $2.1 million, which means many families can’t afford it.

Read more at The Guardian.

Uneven Longevity

A new study, published this very moment, finds that various organs within a single person can have vastly different biological ages. The findings are based on 4,066 people, aged between 20 and 45 years, who live in Shenzhen, China and donated blood and stool samples, as well as facial images, for the study. Genomic sequencing was performed at BGI-Shenzhen, the world’s largest genome research organization.

For each person, a deluge of data was collected; genome sequences, metabolomic profiles, microbiome tests from the stool samples, blood cell counts and immune profiles. The researchers built an “aging-rate index,” and classified each volunteer based on whether they were aging faster or slower than their true age.

The paper is a bit nebulous in scope, to be honest, but one key finding stood out:

having a more diverse gut microbiota indicated a younger gut while at the same time having a negative impact on the aging of the kidneys, possibly because the diversity of species causes the kidneys to do more work.


Read more at Cell Reports.


As Cas9 scans the genome, searching for the proper place to make a cut, it often entangles with the wrong sequence; a mismatch. For a study in Nature last week, researchers used cryo-electron microscopy to ‘watch’ a Cas9 protein throughout this process. They found that specific parts of the Cas9 protein were involved in mismatches, and might facilitate off-target effects.

When they mutated amino acids in these ‘mismatch regions’ of Cas9, they ended up creating a Cas9 protein that cuts DNA just as well as the wildtype version, but is 4,000 times less likely to cut off-target sites. They call the new protein SuperFi-Cas9 and, yeah, you should start using it.

This paper was included in Cell Crunch Index that I sent out yesterday. The lead author, Jack Bravo, also tweeted a great thread about the paper:

Read more from the University of Texas at Austin.

ResearchGate Ruling

Elsevier and the American Chemical Society filed a lawsuit against ResearchGate in Munich in 2017. Their allegation: The website “made copyrighted material freely available,” according to Nature news, because researchers can upload PDFs of their published papers and freely share them with others.

ResearchGate argued that they shouldn’t be held responsible for what authors upload — a defense commonly used in high-profile social media cases — but to no avail. The Munich court ruled against ResearchGate, ruling that they are prohibited from hosting copyrighted papers and holding them responsible for copyright infringement on the website.

Both sides plan to appeal the verdict.

Read more at Nature.

Briefly Noted

Some strains of E. coli produce and release a chemical, called colibactin, that can diffuse to other bacteria and activate dormant viruses within them. Science News

Foresite Capital, a firm that has invested in Sestina Bio and Interline Therapeutics, raised $173M to further fund biotech startups formed in their in-house incubator, Foresite Labs. Genetic Engineering & Biotechnology News

The Innovative Genomics Institute at UC Berkeley and the University of California, San Francisco, launched a genomics incubator for women entrepreneurs. Four entrepreneurs will each be given $150,000 to start their ideas. Up to two of them will then be awarded a $1M startup package. Biospace

Fierce Biotech has released their 10th annual Fierce 15 list, which celebrates innovators in the medical technology space. The first company: Benchling. Fierce Biotech

More soon,

— Niko