Turning human genetic data into the actionable evidence that moves decisions

for pharma R&D biotech life science investors CROs

for 
pharma R&D
biotech
life science investors
CROs

The Genetic Edge

Human genetic evidence is one of the most powerful tools available to de-risk drug targets, yet its potential remains largely untapped in early-stage R&D. Unlike animal models or observational data, human genetics delivers causal evidence directly from human biology, at the stage where it changes decisions. For the first time, the data and tools now exist to leverage human genetics rigorously and at scale across therapeutic areas.

90%

of Phase II failures are driven by insufficient evidence that the target causally drives disease in humans.

150k+

human genetic datasets on clinical outcomes across therapeutic areas and molecular traits available for causal analyses

3 Weeks

from target and indication to a decision-ready evidence report

2.6x

Higher clinical success rate for targets with human genetic support

Minikel et al. 2024 >

MendelyticsTM: Rigour at Speed

Through our MendelyticsTM platform, our team delivers deep genetic intelligence in weeks, without compromising the analytical rigor your decisions depend on. We maintain scientific integrity and transparency. Every output comes with full methodological transparency, so you understand not just what we found, but why you can trust it.

Use Cases

Whether you’re a biotech building a programme, a pharma team assessing an asset, an investor deciding whether to back a programme, or a CRO strengthening the evidence behind your client work, AquilaGen delivers the evidence layer specific to your question.

De-risking before committing capital

Independent due diligence on target biology, co-creation of new programmes with genetic foundations, and ongoing genetics support for portfolio companies.

Genetic evidence at every stage of your journey

Target identification, target validation before Series B/C, indication prioritization, building the genetic evidence package for investor decks and IND narratives.

Faster, better-informed decisions on targets and assets.

Due diligence on in-licensing targets, comparative evidence across competing assets, go/no-go on pipeline programs.

Extending your scientific offering

Add genetics-driven target validation to your preclinical and clinical workflows, strengthening the scientific case you deliver to pharma clients.

Advance drug target research

Independent causal analysis for hypothesis testing, methodological support for grant applications and postgraduate research, and training courses in causal inference methods.

Your Genetics Team

AquilaGen is your dedicated genetics team. We embed analytical rigour, clinical expertise and commercial fluency into your programme, translating genetic findings into the decisions that matter most: which target to progress, which indication to prioritize, and where the human evidence is strong enough to act on. Our cross-disciplinary team brings together statistical genetics, clinical medicine, and drug development expertise to systematically integrate causal human evidence across the life sciences ecosystem.

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pharma, biotech and investor engagements
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publications in high-impact journals
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world-leading institutions.

Capabilities

From foundational target validation to upstream discovery, every analysis is designed to inform a decision, not just add data.

Tier 1

Foundational evidence

Validation

Confirm, challenge, or redirect your programme before you commit further capital or time. The essential first layer of genetic evidence.

Tier 2

Deeper intelligence

Advanced Characterization

When the headline signal is not enough. Genetic insight that directly informs trial design, patient stratification, and regulatory strategy.

Tier 3

Upstream intelligence

Discovery

No target yet? We build the genetically supported target landscape for your indication from the ground up.

News

Resources

Precision in biotech: a person inserts a DNA sample in a tube into a sequencing machine
Condimentum primis vitae sollicitudin tempus malesuada...
07
May
Chemist doctor explaining to coworker vaccine development
Condimentum primis vitae sollicitudin tempus malesuada...
07
May
Doctor in ppe suit working at pc while chemist using microscope
Condimentum primis vitae sollicitudin tempus malesuada...
07
May
team-board-writing-science-formula-group-meeting-with-brainstorming-idea-planning-with-support-cooperation-cure-glass-diversity-people-with-dna-research-collaboration-talk
Abstract Introduction Large artery stiffness (LAS)...
28
Oct

Ask us about your targets

We'll bring the evidence Whether you have a specific asset in mind or want to understand how genetic intelligence could strengthen your program or portfolio – let’s talk.