CASE STUDY

ACCELERATING TARGET DISCOVERY FOR MASH

HOW KINETIC AI BIO USED EMBYR'S PLATFORM TO IDENTIFY HIGH-CONFIDENCE TARGETS AND PRIORITIZE THREE LEAD CANDIDATES IN 14 WEEKS

THE CHALLENGE

Kinetic AI Bio had identified several advanced leads through a phenotypic screen for treating metabolic dysfunction-associated steatotic liver disease (MASH). Under intense pressure from a pharmaceutical partner, the company needed to rapidly pinpoint drug targets and fully understand the mechanistic basis of efficacy.

THE OBJECTIVE

Identify the pharmacological targets of the lead compounds and generate a high-resolution, pathway-level reconstruction of their mechanisms of action (MoA).

THE EMBYR SOLUTION

PHASE 1: TARGET DISCOVERY & MOA RECONSTRUCTION (WEEKS 1–6)

Embyr generated multi-modal single-cell omics data capturing compound responses across multiple cell types relevant to MASH. Using Embyr's AI-powered Target Identification Framework, these data were integrated with Kinetic AI Bio's preclinical results and public datasets to produce high-confidence target hypotheses.

This analysis delivered:

•       On-target drivers of therapeutic efficacy

•       Potential off-target liabilities linked to safety risks or toxicity

•       Bayesian network-based MoA reconstruction, mapping all biological pathways perturbed by each compound

By combining pathway perturbation profiles with nominated targets, Embyr identified which mechanisms were most consistent with the observed pharmacology—and which pathways likely contributed to off-target effects.

PHASE 2: HIGH-THROUGHPUT PERTURBATION & VALIDATION (WEEKS 6–14)

For the top five compounds, Embyr used its proprietary CRISPR "Swiss Army Knife" perturbation platform to experimentally test and validate hundreds to thousands of predicted targets and mechanisms in a single experiment.

The Results

Key Deliverables

  • High-confidence on- and off-targets for all five compounds

  • Detailed mechanistic insights confirming pathways and drivers of efficacy

  • Data enabling prioritization of three lead candidates for the MASH program

  • Development candidate nomination projected in 8–10 months

5
Compounds Analyzed

3
Leads prioritized

14
Weeks to results

"I've evaluated a lot of discovery platforms, and Embyr stands out for its scientific rigor. Their multi-modal single-cell profiling, integrated with our own preclinical data, produced target hypotheses of exceptional quality. Their Bayesian MoA reconstruction mapped pathway-level biology with a precision we've never achieved internally. Embyr provided true mechanistic clarity and gave us the confidence to prioritize the right leads for our MASH program."

— Chief Scientific Officer, Kinetic AI Bio