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Posts by Ryan Leenay

One could argue that it already has. One drug is enough, one patient is enough.

We’re just getting started.

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This edit will always live rent free in my head

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Duck duck grey duck
(IYKYK)

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In a great collaboration with @hummerlab.bsky.social and the Kräusslich lab: HIV capsid doesn't break at the NPC; instead, it cracks open the NPC itself! Details in Cell: authors.elsevier.com/sd/article/S... @mpibp.bsky.social @uniheidelberg.bsky.social A thread below:

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Spurred by AI’s possibilities, Eli Lilly and Andreessen Horowitz partner on $500M venture fund Eli Lilly is partnering with the fabled venture capital firm Andreessen Horowitz on a new fund backed with $500 million of the drug giant’s money.

In an apparently unprecedented move, Eli Lilly is partnering with the fabled venture capital firm Andreessen Horowitz on a new fund backed with $500 million of the drug giant’s money. www.statnews.com/2025/01/10/e...

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A Mole Infiltrated the Highest Ranks of American Militias. This Is What He Found. Outraged by the Jan. 6 Capitol riot, a wilderness survival trainer spent years undercover climbing the ranks of right-wing militias. He didn’t tell police or the FBI. He didn’t tell family or friends....

Read. This.

www.propublica.org/article/ap3-...

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Not something I can answer directly. I assume it’s a time thing, easier to funnel time/energy to the larger mega-round companies we’re seeing more of in biotech?

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1 is slow, 2 would be quick.

1 - Make this an expectation in time. Seed/accelerators (eg Nucleate) start the push. The best firms start to do this, and it becomes something to expect.

2 - Create leverage. Companies provide specific metrics or other essential info in exchange for deep feedback

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Yeah completely it would be.

To your original point, it comes down to how this gets incentivized. One thing I’ve seen is a founder aggressively coming back and asking for feedback and/or reasoning for a negative decision. So one aspect of this can be to empower founders to do this.

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I would worry that a lot of the passes are because they don’t understand, or believe in the differentiation. That’s a tough message to pass on without significant context?

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Some of the best advice I got early in my career was that the best scientists “never really turned their brain off”.

This is a perfect description of that. It wasn’t a call to action on working stupid hours, it’s pushing into the creative problem solving needed for complex science.

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Closure of northern Minnesota camp is ‘the greatest story.’ Here’s why. Willow River, Minn., camp One Heartland is for sale after serving kids there for nearly three decades.

After nearly three decades, a Minnesota summer camp for kids with HIV/AIDS is closing and up for sale... because retroviral drugs are so effective that there aren't enough campers.

Science works, y'all.

www.startribune.com/closure-of-n...

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Arbor Biotechnologies Announces FDA Acceptance of IND Application for ABO-101 for the Treatment of Primary Hyperoxaluria Type 1 - Arbor Biotechnologies® CAMBRIDGE, MA – December 19, 2024 – Arbor Biotechnologies™, a biotechnology company discovering and developing the next generation of genetic medicines, today announced that the U.S. Food and Drug Adm...

Huge congratulations to Arbor on their IND clearance!

arbor.bio/arbor-biotec...

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a door with the words `` you come at the king , you best not miss '' written on it ALT: a door with the words `` you come at the king , you best not miss '' written on it
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Lentiviral vectors containing mouse Csf1r control elements direct macrophage-restricted expression in multiple species of birds and mammals The development of macrophages requires signaling through the lineage-restricted receptor Csf1r. Macrophage-restricted expression of transgenic reporters based upon Csf1r requires the highly conserved...

Macrophages have a lot of innate pathways to shut down expression from exogenous places. Anecdotally, I’ve seen the same issue of a transduction with no expression on RAW264 cells, but there are ways to design around it: pmc.ncbi.nlm.nih.gov/articles/PMC...

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Caribou Biosciences - Jan 2011
Editas Medicine - November 2013
Intellia Therapeutics - November 2014

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Genome-based newborn screening for severe childhood genetic diseases has high positive predictive value and sensitivity in a NICU pilot trial Among NICU neonates not considered appropriate for genetic testing, a clinical trial found 30% to have genetic diseases by genome sequencing, 3.6% positives and 27% positive predictive value (PPV) by newborn screening, and 4.2% positives and 100% PPV by genome sequencing-based newborn screening. Genetic diseases are unexpectedly common in NICUs.

The American Journal of Human Genetics' latest article reports that nearly one-third of NICU neonates initially not considered appropriate for rapid genetic testing have a genetic disease, highlighting the promise of broad implementation of genome sequencing-based newborn screening.
#ASHG #Genetics

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1.0x too, you’re a monster

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New starter pack for all people interested in Gene Editing & Gene Therapy 🧬✂️ including Tools & Delivery|CRISPR-Cas|Zinc finger nucleases|Viral vectors|Non-viral delivery methods

go.bsky.app/8R4dVez

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Awesome news Aron, congratulations!!

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Great thread by Harmit. Industry success (individual and company) also extremely luck dependent… what matters is your *return* on said luck. Graphic and concept from “Great by Choice”.

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The Human Cell Atlas: towards a first draft atlas In a collection of research articles and related content, the Human Cell Atlas consortium presents tools, data and ideas towards the generation of their first draft atlas of cells in the human body.

www.nature.com/immersive/d4...

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CNN claiming it may have been targeted

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Completely agree on method-dependence here too, especially in HSC populations where DNA repair kinetics will vary greatly throughout the process.

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Beautiful paper by Jacob Corn’s lab on the influence of DNA repair on editing outcomes:

www.nature.com/articles/s41...

Although not included, would really be curious to understand the magnitude of the effect in non-cycling cells.

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CRISPR provides acquired resistance against viruses in prokaryotes - PubMed Clustered regularly interspaced short palindromic repeats (CRISPR) are a distinctive feature of the genomes of most Bacteria and Archaea and are thought to be involved in resistance to bacteriophages....

It’s even more basic than this!

Original discovery of the function was in 07’ (pubmed.ncbi.nlm.nih.gov/17379808/) and the e coli locus was in 1987! (pubmed.ncbi.nlm.nih.gov/3316184/)

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Whole-brain spatial transcriptional analysis at cellular resolution Recent advances in RNA analysis have deepened our understanding of cellular states in biological tissues. However, a substantial gap remains in integrating RNA expression data with spatial context acr...

REALLY cool spatial transcriptomics work by @peruhlen.bsky.social.

www.science.org/doi/10.1126/...

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🔥🔥🔥

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Mouse serum can bind (and sometimes inactivate) LVV in vivo. Do you think some of this macrophage effect is exacerbated by this? I don’t think AAV8 and APOE LNPs suffer from the same effect in mice

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Crisp-Skinned Spatchcocked (Butterflied) Roast Turkey With Gravy Recipe Spatchcocking turkey gives you stunningly crisp skin and perfectly cooked white and dark meat. Bonus: this recipe makes a flavorful gravy from the pan drippings, too.

A dry turkey is user error.

www.seriouseats.com/butterfiled-...

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