StemRIM.AI
A working session for the R&D organization

Rearranging
the Factory

Redesigning StemRIM's R&D around AI — not bolting it on top. How the bench, the Dry team, and discovery itself change.

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Executive summary · the answer first

Redesign the R&D factory around a closed AI–bench loop to generate more, better-validated assets per yen burned

Governing thought

AI's payoff comes not from adding tools to today's bench-first process, but from rebuilding the workflow so computation proposes and the bench validates.

Three mutually-reinforcing moves deliver it — and one precondition makes all three possible. We ask for three decisions to start this quarter.

Move 1 · the engine

A self-driving discovery loop

Generative AI proposes peptides; a model ranks them before synthesis; the bench validates only the winners; every result trains the next round.

Move 2 · the org

Fuse Wet and Dry

One Discovery-Loop pod measured in validated candidates per bench-week — not experiments run. The bench proves ideas; it no longer originates them all.

Move 3 · the unlock

A responder-signature model

Turn the ~400-patient rare-disease ceiling into an enriched, approvable population — a path to approval the old playbook can't reach.

Precondition: a proprietary data lake that captures every experiment by default. Moves 1, 2 and 3 all sit on it — so we build it first, this quarter.

StemRIM.AI · Rearranging the Factory
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Situation · why this isn't just "use more AI"

Electricity took 40 years to pay off — the gain came from rebuilding the factory, not from the new power

Paul David's lesson (1890s–1920s): factories that swapped a steam engine for an electric one and changed nothing got almost no gain. The payoff came only when each machine got its own motor — and the floor was rebuilt around the flow of work.

▲ The trap — 1890s

One motor on the old drive shaft

New power, old layout. Every machine chained to one central shaft. Output barely moved.

● The leap — 1920s

A motor on every machine

Machines freed from the shaft. The factory rearranged around the work. Then productivity jumped.

Adding AI on top of our bench-first process is a faster steam engine. The payoff comes when we redesign the work itself.

3
Situation · the test we apply to every AI idea

One question sorts every idea: does it speed up a step, or change the workflow?

▲ Faster steam engine

It speeds up a step that already exists.

Useful, cheap, worth doing — but it is not the transformation. Budget ~20% of effort here.

● Rearranged factory

It changes the workflow, who decides, or what flows into the data.

This is where the gains live. Budget ~80% of effort here.

Every slide that follows is labeled with this test — that's the horizontal logic of this deck.

4
Situation · our own reality

A research org on a cash clock must maximize well-validated shots on goal before the cash runs

5
indications for redasemtide (TRIM2) in Ph II — EB, stroke, ischemic cardiomyopathy, knee OA, chronic liver disease
4
earlier-stage codes — TRIM3 / TRIM4 / TRIM5 peptides + SR-GT1 gene therapy
~¥7B
cash, against ~¥1.4B annual R&D burn
69
people — small enough to actually redesign

How many high-quality, well-validated shots on goal can we generate before the cash runs? That is the question every move below answers.

Our out-licensing model (upfront + milestones + royalties; redasemtide→Shionogi) does not change. AI multiplies the assets that feed it.

5
Complication · the diagnosis

Today computation is a reporting function bolted to the end — our central drive shaft

We have a Dry (bioinformatics) team beside the Wet (experimental) team — but Dry is a service downstream of Wet, analyzing data and building apps after the bench decides what to make. That is the electric motor wired onto the old shaft.

Human hypothesis

scientist proposes

→
Wet screening

synthesize & assay

→
Animal efficacy

disease models

→
Dry analysis

sequencing, after the fact

→
Clinical / license

out-license

Serial and human-paced. Each run informs that run, then sits in folders. Faster instruments make each box quicker — the shape never changes.

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Complication · end to end

The same hidden shaft runs through all six stages of our R&D

STAGE 1

Discovery & mechanism

Ideas are born at the bench, from one hypothesis.
STAGE 2

Preclinical design

Dry is a service desk downstream of Wet.
STAGE 3

Translational / biomarker

Who responds is observed, not predicted.
STAGE 4

Clinical & PMDA

Small-n is a ceiling, not a design problem.
STAGE 5

Data & knowledge

20 yrs of data is effectively write-only.
STAGE 6

Org & day-to-day

Decisions flow Wet → Dry.

In one sentence: the wet lab is the origin of every idea, and computation is tacked on at the end to report.

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Complication → resolution · sorting the ideas

"Use more AI" hides two very different bets — we spend ~20% on speed, ~80% on redesign

▲ Faster steam engine — ~20%
  • Chatbot licenses for everyone
  • More computing power so Dry reports sooner
  • AI drafts trial documents faster
  • A better search box over the share
● Rearranged factory — ~80%
  • AI proposes peptides; a model scores them before synthesis
  • Wet & Dry fuse into one closed loop
  • A responder model turns n≈400 into approvable evidence
  • One data lake captures every experiment by default

The steam-engine column isn't wrong — we ship it in Phase 0 to free up your hours. We just don't call it transformation.

8
Resolution · move 1 — the engine

A self-driving loop where computation proposes and the bench validates every round

DESIGNgenerative AI PREDICTactivity model TESTwet-lab oracle LEARNactive learning DATA LAKE

Computation proposes. The bench validates. Every result trains the next proposal.

  • DESIGN — generative models propose peptides by the thousand, conditioned on our MSC-mobilization mechanism
  • PREDICT — an in-house model ranks them before synthesis
  • TEST — the wet lab runs only the winners, as a precision oracle
  • LEARN — results retrain the models; active learning picks the next batch

Why we can do this: TRIM3/4/5 are designed peptides with a measurable activity readout — an ideal design–assay loop. Most biotechs would kill for a target this clean.

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Resolution · move 2 — the org

Fuse Wet and Dry into one pod — the bench stops being where ideas are born and becomes where they're proven

Today

A line with a handoff

Bench decides what to make → runs it → Dry analyzes and reports. Two departments, one direction.

Your day: run my experiment, then analyze it.

● Redesigned

One Discovery-Loop pod

Wet + Dry fuse into a single team with shared OKRs, measured in validated candidates per bench-week — not experiments run.

Your day: review the model's ranked proposals, design the experiment that validates them, feed results back.

The bench stops being where ideas are born and becomes the high-value place they are proven.

10
Resolution · the concern, addressed

The loop amplifies the founder's intuition — it does not replace it

The fear is real: "the machine replaces the founder's intuition." It does the opposite. The loop's objective function is Tamai-sensei's biology — the mechanism defines what "good" means. The model searches a space no human team can; the CSO adjudicates a ranked queue instead of originating every idea by hand.

Before

Every hypothesis starts from one scientist's insight, one at a time.

After

The mechanism guides AI to propose thousands; the CSO judges the best.

Cheap test to earn trust

Blind test: the model proposes peptides; the CSO ranks AI vs human designs without knowing which is which.

An amplifier for founder intuition — not a replacement.

11
Resolution · move 3 — the unlock

A responder-signature model turns the ~400-patient ceiling into an approvable population

The wall

Dystrophic EB has only ~400 patients nationwide. A large Ph III is impossible. Small-n is treated as a statistical ceiling.

We already see it: in EB, 7 of 9 improved, 4 markedly — but we don't model who responds and why.

The unlock

A multi-omics + clinical responder-signature model predicts who responds — turning n≈400 into an enriched, approvable population.

Org change: a small translational data squad owns the biomarker as a first-class, milestone-bearing deliverable — itself licensable.

Precision medicine as the path to approval where the old playbook has none.

12
Resolution · the precondition

None of it works without the data backbone — so we build the data lake first

● The proprietary data lake

Capture everything, by default

Every assay, sequencing run, animal study and key decision flows into one structured, versioned lake — not a sprawl of shared folders. "Log it to the lake" becomes part of every protocol, enforced like GLP.

The knowledge brain

Make 20 years searchable

Index the shared drives, patents and R&D-meeting recordings into a system you can ask questions of — plus always-on IP / freedom-to-operate scans on the peptide platform.

Our 20 years of screening data is the moat a generic model can't have — but only once it's unified. This is what we start this quarter.

13
Resolution · the cross-check

Three dependencies could break this — and we sequence the plan around each

● The hard dependency

Moves 1, 2 and 3 all sit on the data lake. Build it first, or everything stalls. This is the single most important line in the plan.

Cold start

Thin labeled data hobbles generative design → start with active learning, graduate to generation as the lake fills.

Org gravity

Without real workflow authority, the loop slides back to Dry-as-service → the org change must lead the tooling.

External levers

AI-designed trials depend on PMDA & academic PIs → apply Stage-4 redesign to assets we still own (TRIM3/4/5), not Shionogi's.

14
Resolution · the plan, ~24 months

Each phase funds credibility for the next — irreversible foundation first, exciting parts second

Phase 0 · Q1–Q2
Foundation
Stand up the data lake (one assay end-to-end). Index the knowledge brain. Appoint the CDAO. Ship 2–3 steam-engine quick wins.
Phase 1 · Q2–Q4
First Loop
Run the full loop on one TRIM3/4 program (active learning first). Fuse Wet+Dry. Begin the EB responder model.
Phase 2 · Yr 2 H1
Scale
Extend the loop to TRIM5 & indication selection. Informal PMDA pre-consult on one enriched design.
Phase 3 · Yr 2 H2
AI-Native
The loop is the operating model, not a project. Every new idea runs through the test by default.

Irreversible foundation first; the exciting parts second. A 69-person factory can be redesigned in a year.

15
Resolution · de-risking

Every risk has a cheap test we run before we commit

RiskCheap test — run it first
Cold start — not enough labeled data~2 weeks. Backtest: can a model trained on past data re-rank known hits to the top?
Org resistance to the inversionOne quarter. Run one program as a fused pod; measure candidates/bench-week vs a control.
"AI can't capture the founder's intuition"A blind test. CSO ranks AI vs human peptide designs, unlabeled.
Data lake becomes an IT boondoggle30-day mandate. One assay captured end-to-end — no 2-year platform.
PMDA / PIs reject AI-designed trialsOne meeting. Informal PMDA pre-consult before any build.
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Conclusion · what we're asking for

Three decisions to start this quarter

1

Fund Phase 0

The data lake + knowledge brain. The precondition for everything.

2

Charter one pod

Fuse Wet+Dry on a single TRIM3/4 program. Run the blind test.

3

Name a CDAO

Workflow authority, reporting to the CEO, peer to the CSO.

We keep exactly what StemRIM is. We change how the factory is laid out — for more, better-validated assets per yen burned.

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