Maxime Boneza

PhD Candidate| UMN Twin-Cities | BMBB | The Smanski Lab |Streptomyces | Natural Products | Disease suppressive soils | microbial engineering | Genetic circuits.

[Yair Einhorn @yaireinhorn]

Although most of my friends on 𝕏 are Americans & probably don’t know what I’m talking about but we’re just hours from the biggest sport event in the world - the Champions League final: Paris Saint-Germain 🆚 Arsenal. As a die-hard Arsenal fan I can’t wait! #COYG! #UCLfinal ❤️

[Arsenal @Arsenal]

Standing on the brink of history.

[NBC Sports Soccer @NBCSportsSoccer]

Arsenal title celebrations 22 years apart!

It has been 22 long years for Arsenal fans, but the Premier League title has finally returned to North London!

[Liz Ann Sonders @LizAnnSonders]

College grads expect to make $80,000 one year after graduation but actual average starting salary is $56,153, about 30% lower than expectations and a difference of nearly $24,000…ten years after graduation, students anticipate earning $144,889 on average but that’s well above the $95,521 reality @ListWithClever

[Dona Sarkar @donasarkar]

Nope. Not the tool’s problem.

There is a HUMAN who set this whole thing up, gave too many permissions and did not validate the impact.

They were a bad AgentBoss.

Anyone who has managed a new vendor, intern or new hire knows the drill: YOU are responsible not the people/tech you delegated to.

[Angelica Peebles @angelicapeebles]

Regeneron's newly approved gene therapy for hearing loss will be available for free in the U.S. The company hasn't decided what it will charge overseas https://t.co/F6QIzw4HZi

[Michelle Lee @michellearning]

Welcome to the scientific revolution. 100s of robots. Zero coffee breaks. America’s largest autonomous lab, open today.

[Twist Bioscience @TwistBioscience]

Inducible, split base editors for in vivo cancer functional genomics

Nature Biotechnology https://t.co/6Oqe5ksW5p

[Niko McCarty. @NikoMcCarty]

Huge news. A gene therapy for deafness, targeting a mutation in the OTOF gene, is approved.

Restored hearing in 9 of 12 children, well enough that they could stop using cochlear implants. The treatment will be free in the United States.

[Veera Rajagopal  @doctorveera]

A monumental moment in medical history: the first gene therapy for genetic hearing loss is now FDA approved. As a former Regeneron scientist, I feel very proud. I had the opportunity to hear about this programme while it was still in development. It’s one of the few programmes that, every time you came across it, you felt the medical breakthrough in your bones and privileged just to be there while it was happening.

At this moment, it’s important that we look 30 years back when researchers mapped a locus on chromosome 2 to congenital deafness in a Lebanese family (https://t.co/o8ltjHyLNy). They named it DFNB6 (later DFNB9) with no clue about the responsible gene. Three years later, the causal gene came to light: OTOF, encoding a protein called otoferlin (https://t.co/tBGlFPzaQd). Seven years after that, in 2006, pioneering work by Christine Petit revealed that otoferlin is a calcium sensor in the inner hair cell membrane, acting as a molecular trigger that converts sound into electric signals that the brain can read (https://t.co/oxXtifPrTZ). Twenty years fast forward, we now have a successful treatment. Thirty years from discovery to medicine.

OTOF-related deafness is congenital, caused by complete deficiency of otoferlin. In these children, the cochlea is structurally intact, hair cells are there, the mechanics of sound transmission work. It’s just that final step, where hair cells hand off the signal to the auditory nerve through neurotransmitter release, that doesn’t happen. Sound arrives and dies at the synapse. It’s deafness due to a defect in the synapse caused by the absence of a single protein, which is what made this a beautiful, clean target for gene therapy.

The treatment itself is a feat of molecular engineering. OTOF is too large to fit in a single AAV capsid. The team solved this elegantly by delivering the gene in two halves separately, which then get spliced to produce the full functional protein. A single surgical injection into the cochlea, a molecular miracle unfolds. Results from the CHORD trial were striking: of 20 evaluable patients, including children as young as 10 months, 80% showed meaningful hearing improvement, and by 48 weeks, 42% had achieved normal hearing including the ability to hear whispers. Otarmeni is not only the first gene therapy for deafness, it’s also the first dual-AAV therapy to be approved by the FDA.

There are very few things in medicine that come close to giving back a sense like vision, hearing, or touch that a human never had from birth. It’s almost God’s work. A parent witnessing their child who was born deaf hearing their voice for the first time, it’s a joy that no words can describe. Multiply that by the fact that it came from a single injection, a repaired gene, and 30 years of science. We are truly in the golden era of medicine.

Regeneron press release:
https://t.co/6zvdsT2uzI

Below video is from the NEJM publication of CHORD trial (Valayannopoulos et al. NEJM 2025)
https://t.co/YvIqwQ0SDu

[Mikael Elias @elias_mikael]

⚠️All of the below images were FABRICATED by ChatGPT Images 2.0, each with a single prompt❗️ ⚠️

[Sajith Wickramasekara @sajithw]

In the AI era, the traditional biopharma industry is the underdog. Big tech and AI labs are building wet labs. China has overtaken Europe in molecules produced. But the tools available to the industry discuss science, not do it.

The hard problem in AI for science is at the interface between the physical and digital worlds.

We built an AI Scientist at that seam. It wires together the digital and physical worlds of R&D. Predictive models, data infrastructure, wet lab execution feed into a single loop that reasons, acts, and improves with every experiment. Our ambition: get molecules to the clinic twice as fast.

Last fall I wrote about why biotech needs to be rebuilt for the AI era. Today I'm sharing the next chapter: what the AI Scientist is, a blueprint for how it works, and why even Richard Feynman couldn't hack it in a wet lab.

[David Sinclair @davidasinclair]

Molecular biology is

[Brad Loncar @bradloncar]

A couple of weeks ago when I visited Solid Bio, they showed me some videos of Duchenne kids pre and post microdystrophin treatment that I was amazed by - but they weren't sharable yet. Bo just presented them at Needham this week. So cool.

[23andMe @23andMe]

Published in @Nature today, new research from the 23andMe Research Institute identifies genetic variants that may help explain why GLP-1 medications work differently based on genetics.

Read the announcement: https://t.co/xy0IuwnoEq

#GLP1 #Genetics #PrecisionMedicine #Research

[Maxime Boneza @maxanct]

This is so true! It's clever to use a pie chart on 3.14 day.

[PHD Comics @PHDcomics]

Happy π day. Here's a pie chart

[Maxime Boneza @maxanct]

What an interesting work!

[Michael Andregg @michaelandregg]

We've uploaded a fruit fly. We took the @FlyWireNews connectome of the fruit fly brain, applied a simple neuron model (@Philip_Shiu Nature 2024) and used it to control a MuJoCo physics-simulated body, closing the loop from neural activation to action.

A few things I want to say about what this means and where we're going at @eonsys. 🧵

[David R. Liu @davidrliu]

Below is the story of the first patient treated with a prime-edited therapeutic, developed by @PrimeMedicine in a trial led by Dr. Élie Haddad and his team at CHU Sainte-Justine. This teenager suffered from chronic granulomatous disease (CGD), an immunodeficiency, and now—10 months after treatment—the patient is healthy, stable, and living with a functioning immune system. Tracy Attebury, whose story was previously told by @ginakolata @nytimes, was the second patient treated with a prime-edited therapeutic.

https://t.co/ZiVq1SmYsn

[BrooklynDad_Defiant!☮️ @mmpadellan]

Good morning and Happy Monday to everyone who won’t forget that trump removed MLK Jr. Day and Juneteenth from National Park free-entry days, then added his own birthday.

The disrespect is deliberate.

[Arsenal @Arsenal]

This Chanukah we send love, strength and light to our Jewish communities around the world.

Our thoughts are with everyone affected by the tragic events in Sydney.