insitro

Making medicines differently to bring better drugs faster to the patients who benefit most with ML and data at scale.

[insitro @insitro]

Pipeline update: insitro is in Boston this week at the 5th Annual ALS Drug Development Summit, where we will share in vivo data from our fully owned and controlled ALS oligonucleotide program for the first time. insitro’s Shababa Masoud will present in vivo gene knockdown and drug exposure data from our ALS-1 ASO program, which targets a key protein implicated in mRNA modulation.

insitro’s ALS program is powered by the Virtual Human™ – our AI model of causal biology that mapped disease drivers linked to TDP-43 dysfunction and mislocalization, a pathology hallmark in ~97% of ALS patients, including aberrant cryptic exon inclusion in critical neuronal transcripts like UNC13A, STMN2, and multiple others. This platform is the foundation for our therapeutic portfolio being advanced with Bristol Myers Squibb.

If you’re attending this intimate gathering of world-leading ALS drug developers, don’t miss insitro senior scientists:

Shababa Masoud delivering the talk "Machine Learning Enabled ALS-Target Discovery and Development of an Oligonucleotide Therapeutic" (Thursday)

Cynthia Hao presenting the poster "Paired Optical and Transcriptional Screening for ALS Targets in a TDP-43 Depleted iPSC-Derived Motor Neuron Model” (Wednesday)

insitro is proud to participate in the 5th Annual ALS Drug Development Summit and share our latest research with drug developers working tirelessly toward a cure for ALS. See you in Boston!

#ALS #DrugDiscovery #AI

[insitro @insitro]

2/2: The science:
ICLR (Rio de Janeiro) – PETRI + ContrastiveBiVI for cell biology and perturbation dynamics
ARVO (Denver) – 2.5D OCT foundation model + highest‑resolution AMD GWAS (w/ @MoorfieldsBRC)
Target ALS (Boston) – first‑in‑class TDP‑43 modulators with @bmsnews
Keystone Symposium (London) – ~100× virtual screening capacity with TherML

[insitro @insitro]

1/2: Four conferences. Three continents. Two weeks.
insitrocytes took our Physical AI platforms on the road, demonstrating the breadth of our pipeline and the depth of the Virtual Human™.

[Tom M Mitchell @tommmitchell]

@DaphneKoller describes her pioneering research on machine learning for probabilistic models as a @Stanford Professor, and her move to industry, where she is currently CEO of @Insitro applying ML to human biology. https://t.co/3jnbUAg7ai

[Ethan Weinberger @efweinberger]

Very excited to be at #ICLR in Rio 🇧🇷!

I'll be around all week and am happy to chat about AI for Biology / Drug Discovery.

Also, my team at @insitro is hiring (https://t.co/c7G2O7zet0)!!! DMs are open, so feel free to reach out 😁.

[Startup Or Scaleup @StartupNScaleup]

Learn how @insitro’s unique team structure brings different disciplines together for more effective communication - helping it identify the most interesting problems to solve.

@insitro founder @DaphneKoller shares what to co ..
rt @wef

[insitro @insitro]

insitro founder and CEO @DaphneKoller joins industry R&D leaders today at the rEVOLUTION Symposium in Washington, D.C. – organized by @5amVentures, @BioCentury, @VersantVentures and @wilsonsonsini – for “AI in Drug Discovery: Separating Signal from Science Fiction.”

Daphne is featured alongside executive and R&D leadership from @TakedaPharma, @IsomorphicLabs, and @Xaira_Thera, with @FionaHMarshall (Novartis) moderating. This timely discussion will explore what AI is enabling in practice today, as well as the technical and scientific constraints that still need to be addressed to drive impact across R&D.

That is exactly what insitro was built to do. By integrating machine learning with large-scale human data and experimental systems, we capture the true complexity of disease biology to support reliable translation into therapeutic programs.

We look forward to Daphne sharing how this approach is rebuilding drug discovery from an unpredictable journey into an industrialized, repeatable process to bring better drugs faster to the patients who can benefit most.

→ Learn more at https://t.co/8KhM983uSL

[insitro @insitro]

"We had to build this at scale. We had to build this across biologically relevant, human-relevant systems. We had to do it at incredible quality so that the AI doesn't get trapped in things that are artifacts, as opposed to signal."

insitro founder and CEO @DaphneKoller spoke with @ben_guggenheim of @washingtonpost this week on AI in drug discovery, discussing the drive to build insitro’s Virtual Human™ — described by Daphne as "a first-of-its-kind platform for making at-scale interventions in cellular systems" — to reveal how disease begins, progresses, and can be resolved.

On when AI in biology became possible:
"Look, all of the human feature engineering in the world is going to get totally eaten up if you have enough data for the machine to learn on its own. And that's the right path."

Cellular data caught up first:
"The application of truly unbounded potential in AI as applied to biology began when we started to get to the scale on the cellular side."

On the role of human genetics:
"If you look at variation in a population scale, you can start to see associations and say, 'Oh, changes in this gene correspond to changes in body mass, or to Alzheimer's risk or to an inflammatory disease.' And because the genetics comes first, it's by definition causal."

Check out the link below to read more of Daphne's conversation with The Washington Post and learn how insitro is advancing a pipeline of validated first-in-class drug candidates for multiple diseases toward the clinic.
https://t.co/nrRxyQp5JG

[insitro @insitro]

insitro is excited to return to the Milken Institute Global Conference. On May 3, CFO/CBO Mary Rozenman joins Alex Azar, Jake Becraft, Robert Nelsen & Sung Hee Choe for a discussion on sustaining global leadership in biomedical innovation. We’re excited to connect with leaders in science, health, policy, finance, and philanthropy at #MIGlobal again this year. https://t.co/V2ASHNBJiU

[insitro @insitro]

Tomorrow @insitro presents at @AllofUsResearch Science Day: new work bridging @uk_biobank and All of Us through AI-based phenotype imputation, powered by our Virtual Human™. The research strengthens the genetic evidence for IRS1, a master regulator of liver fat and fibrosis – and the basis for CTRO-1013, our liver-targeted GaINAc siRNA therapy entering the clinic this year.

Register: https://t.co/XHc043lOuX

[insitro @insitro]

This expansion reflects the momentum of our joint work and strengthens our ability to advance a broader portfolio of disease-modifying programs, grounded in causal biology and built to move with speed and precision for patients living with ALS.

Learn more by viewing the slideshow.

[insitro @insitro]

insitro is proud to deepen our work in ALS with the expansion of our strategic collaboration with @bmsnews and the nomination of two additional therapeutic targets discovered through insitro’s AI-driven Virtual Human™ platform.

Read our press release: https://t.co/BoXfk7SUKZ

[insitro @insitro]

Powered by insitro’s Virtual Human™ causal discovery platform, this work integrates massive-scale, human-derived cell data with machine learning to map disease drivers with unprecedented resolution, including processes linked to TDP-43 mislocalization, a hallmark of pathology in nearly 97% of ALS patients.

Take a deep dive in our latest Perspectives blog post: https://t.co/x16MhLGWp4

[BiotechTV @BiotechTV]

𝐒𝐅 𝐇𝐞𝐚𝐥𝐭𝐡𝐜𝐚𝐫𝐞 𝐖𝐞𝐞𝐤: @insitro CEO @DaphneKoller on how AI is hitting “escape velocity” for finding causal targets for disease and speeding up drug development.

Full video: https://t.co/Kh2lPPLnez

[insitro @insitro]

Today we announced Joe Hand as insitro’s Chief People Officer.

Joe brings a wealth of experience in scaling life sciences organizations, including serving as CHRO and Executive Committee member at Celgene, where he led global talent strategy through significant growth and major transactions.

As we advance our AI-driven discovery platform and move programs toward the clinic, Joe will lead our global people strategy to support insitro’s next phase of growth. Welcome to the team, Joe!

Read more: https://t.co/t9im8jtfD2

[insitro @insitro]

insitro’s David Lloyd reported at the @KeystoneSymp the first-ever genome-wide association study (GWAS) of brown adipose tissue – and a differentiated target with anti-obesity effects, the modulation of which reduced body weight 15% and fat mass 25% in preclinical models while preserving lean tissue.

Brown fat was genetically inaccessible for decades – not for lack of interest, but because the tissue resists measurement at scale. ML-derived phenotypes from 70K MRIs resolved that.

The weight loss comes from burning more, not merely eating less.

This work suggests a path to differentiated obesity treatments and illustrates a broader shift in human genetics. We've shown that phenotype quality can transcend data limitations, and AI is helping us make that leap.

Learn more: https://t.co/iYPLmUCXfc

[insitro @insitro]

“We want to build an engine that brings transformative medicines to patients,” says insitro founder and CEO @DaphneKoller in conversation with @BiotechTV during #JPM26.

Daphne sat down with Ryan Flinn for SF Health Week to discuss how AI is accelerating drug discovery at insitro and how models designed to work in partnership with scientists are helping turn complex data into meaningful therapeutic insights.

[BiotechTV @BiotechTV]

𝐒𝐅 𝐇𝐞𝐚𝐥𝐭𝐡𝐜𝐚𝐫𝐞 𝐖𝐞𝐞𝐤: @insitro CEO @DaphneKoller on how AI is hitting “escape velocity” for finding causal targets for disease and speeding up drug development.

Full video: https://t.co/Kh2lPPLnez

[insitro @insitro]

Today marks a major milestone for insitro with the acquisition of CombinAbleAI and the launch of TherML™, our unified, modality-agnostic therapeutic design platform.

TherML expands our AI-enabled system across small molecules, oligonucleotides, and complex biologics, integrating physics-informed design so potency and developability are optimized together from the start.

We’re excited to welcome the CombinAbleAI team in Israel as insitro’s newest R&D center and take this next step toward more predictable, scalable, and accelerated drug discovery.

🔗 https://t.co/mDgWO1bg9P
Dive into TherML: https://t.co/rRNfkSIz9U
#Biotech #DrugDiscovery #AI

[insitro @insitro]

insitro Founder and CEO @DaphneKoller presents at the 44th Annual J.P. Morgan Healthcare Conference on Monday, Jan. 12 at 4 p.m. PT – providing updates on our AI-enabled discovery platform and therapeutic programs.

insitro leaders will also participate in key Healthcare Week discussions:
→ Sunday: Daphne joins the @LongwoodLeaders SF CEO panel “Identifying Next-Generation Therapeutics.”
→ Wednesday: CFO/CBO @mmrozenman joins the “Transaction to Transformation” panel at the @KPMG + @goodwinlaw Symposium.

We look forward to engaging with the investment community, partners, and industry leaders throughout the week. https://t.co/4YLVD4mPKl

#JPM2026 #Biotech #DrugDiscovery #AI