Biotx
Founded 2017 · 15 employees on LinkedIn · 2 known investors
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biotx.ai maps how genes causally shape human biology by systematically testing every human gene across tissues using large-scale Mendelian randomization, colocalization, and causal AI. This generates high-confidence causal links between genes, diseases, and biomarkers for drug target discovery.
Also known as Biotx AI · biotx.ai · biotx.ai GmbH
Founders & leadership
Biotx was founded in 2017 by Joern Klinger.
Investors · 2
Company profile
researched Aug 2026biotx.ai GmbH develops causal inference and machine-learning methods that use human genetic data to identify and validate drug targets and to inform clinical trial design. The company frames human genetics as a naturally occurring randomized experiment: because genetic variants are allocated randomly at conception, they can be used as instrumental variables in Mendelian randomization (MR) to estimate causal effects between an exposure and a disease outcome, avoiding much of the confounding present in observational data.
To apply MR systematically, biotx.ai assembled a catalog of thousands of genome-wide association studies covering more than 25 million cases across more than 3,000 diseases, and ran MR and colocalization analyses on all approximately 23,000 human genes plus more than one million omics markers across 60 tissues for each disease. The resulting billions of causal data points, and AI models trained on them, are presented as a map of how genes causally shape biology, intended to support novel target discovery and trial design across therapeutic areas. Earlier company descriptions positioned biotx.ai around predictive biomarkers for decision support in precision medicine using machine learning applied to biomedical data.
The company publishes research on its methods and applications, including a 2026 retrospective evaluation of human genetic evidence for clinical trial success using Mendelian randomization and machine learning, a 2025 study validating the L-type calcium channel blocker amlodipine as a candidate ADHD treatment via cross-species analysis, drug-target MR and medical-record evidence, and a 2023 paper on targeting the longevity gene SLC13A5 in age-related bone fragility and osteoporosis.
Founding story
Founded in 2017 by Jörn Klinger and Marco Schmidt.
Business model
biotx.ai works with pharmaceutical and biotech clients, applying its causal AI models and human genetic database to their compounds and programs — identifying the best indication for a drug, generating human evidence for efficacy, assessing likely side-effect profiles versus competing compounds, and informing trial protocols and endpoints. It also partners with service providers such as contract research organizations, which offer its predictive analyses to their own clients.
Partner and client engagements with drug developers and CROs for causal analyses supporting indication selection, efficacy and safety evidence, and trial design; specific pricing terms are not disclosed.
Traction
The company states its causal AI accurately predicted the outcomes of eight COVID-19 clinical trials, with results replicated across other indications, and that its analyses have been instrumental for several clients in raising funding for phase 2 trials. It has published peer-reviewed style research outputs in 2023, 2025 and 2026, and maintains a strategic CRO partnership.
Latest developments
The company highlights a study on whether Mendelian randomization can help predict clinical success, with a related publication dated February 23, 2026 on retrospective evaluation of human genetic evidence for clinical trial success using Mendelian randomization and machine learning.
▸Full profile — market position, technology, go-to-market, geography, history
Market position
Positions itself as the first to apply causal modelling in drug development at scale, addressing the low predictability of clinical efficacy — phase 2 success rates below 30% — by using genetic support, which the company notes underpinned two-thirds of FDA drug approvals in 2021.
Systematic, genome-wide and tissue-wide application of Mendelian randomization and colocalization at a scale the company describes as generating billions of causal data points, coupled with AI models trained on that causally annotated dataset, rather than case-by-case genetic association analysis.
Technology
The core technical approach combines large-scale drug-target Mendelian randomization and colocalization with machine learning ("causal AI"). Genetic variants serve as instrumental variables proxying exposures, enabling causal effect estimation from retrospective data. Analyses span all ~23,000 human genes, over one million omics markers and 60 tissues against thousands of diseases drawn from a curated GWAS catalog covering more than 25 million cases and more than 3,000 diseases. A 2023 description of the underlying human genetic resource cited genomes, full phenotypes and medical histories of 3.3 million patients spanning over 12,000 diseases. The company states its causal models simulate clinical trial outcomes, citing successful prediction of the outcomes of eight COVID-19 trials and subsequent replication across other indications and therapeutic areas.
Go-to-market
Direct partnering with drug developers (partnering@biotx.ai) alongside channel partnerships with clinical research organizations; a strategic partnership with mid-size CRO Simbec-Orion announced in October 2023 makes biotx.ai's predictive, causal-AI trial-outcome insights available to Simbec-Orion clients in oncology, rare disease and clinical pharmacology.
Biotech and pharmaceutical drug developers, particularly small and mid-sized companies advancing early-stage assets, as well as CROs serving them.
Geography
Headquartered in Potsdam, Germany, operating as biotx.ai GmbH; partnership announced with UK-headquartered Simbec-Orion, which has offices in Europe and the US.
History
Founded in 2017 and based in Potsdam, Germany, biotx.ai received investment from Hoxton Ventures in 2019 through its Hoxton II fund, with Hussein Kanji as the partner involved. Reported additional funding in 2020. In October 2023 the company announced a strategic partnership with CRO Simbec-Orion; at that time Joern Klinger was described as CEO. Internal records list Marco Schmidt as CEO and Charles Ravarani as CTO.
Compiled by commissioned research from 8 cited public sources — announcements, filings, and press listed under research sources below.
Key figures
latest reportedCompany-reported or press-reported figures, each dated to when it was claimed — not independently audited.
Timeline · 6
launches, deals, and filingsbiotx.ai and full-service mid-size CRO Simbec-Orion announced a strategic partnership to bring causal AI models and predictive trial-outcome insights to Simbec-Orion clients, informing clinical development strategy and trial protocol and endpoint design.
$2.2M source ↗
Dated company events from announcements, filings, and press; legal rows summarize public dockets and regulator releases.
▸Research sources · 8
primary sources listed
- Biotxbiotx.ai · web
8 public sources were cited for this profile; the first-party ones are listed here.
Frequently asked questions
- What does Biotx do?
- German company applying large-scale Mendelian randomization and causal AI to human genetics for drug target discovery and trial design.
- Who founded Biotx?
- Biotx was founded by Joern Klinger in 2017.
- Who are Biotx's investors?
- Biotx's investors include Hoxton Ventures, Oxonian Ventures.