Rare diseases drug discovery

Introducing the largest open-access intelligence hub for rare disease drug discovery

At Explority AI, we are launching an open registry that brings together scattered information about drug discovery covering 5,846 rare diseases. It replaces days of manual research with a single source of value: population, burden, standard of care, and drug discovery landscapes that biotech and pharma teams can act on immediately.

At Explority AI, we are launching an open registry that brings together scattered information about drug discovery covering 5,846 rare diseases. It replaces days of manual research with a single source of value: population, burden, standard of care, and drug discovery landscapes that biotech and pharma teams can act on immediately.

It's a single directory built to answer the questions a research or business development team needs answered before evaluating a rare disease opportunity: population and burden data, standard-of-care context, the latest published research, and a live map of a drug discovery landscape.


One page. Complete disease context.

Scattered data slows down drug discovery: prevalence figures from one registry, natural history from a review article, current treatments from a clinical guideline, competitive intensity from an orphan drug database, and the newest science from hundreds of research papers. Pulling all of that together, disease by disease, can take days, and the work has to be repeated every time the landscape evolves.

Explority AI replaces that process with a single, continuously updated page for each of 5,846 rare diseases, built on the same 1M+ paper research corpus that powers Explority's probability-of-success models, so researchers, biotech companies, and business development teams can easily:

  • Spot unmet medical need

  • Navigate the latest research

  • Identify standard-of-care gaps

  • Access ready-to-use drug discovery landscapes


Inside a disease page

Every profile follows the same structure, so learning to read one means you can read all 5,846. Take the Fabry disease page as an example.

  • Overview – a clinical summary of the disease.

  • Analytics – population, burden, and therapy data in three parts: how many people are affected and who they are, the clinical and economic burden of the disease, and the current standard of care.

  • Research – the live count of drug discovery papers published on the disease, how many describe first-in-class or next-in-class therapies forecast to outperform average preclinical success rates, and the most recent publications, linked to the original articles.

  • Drug discovery landscape – a complete table of every orphan drug designation granted for the disease by the FDA and EMA, including therapy type, regulator, designation date, approval status, and sponsor, updated as new designations are granted.


Why it's open

Explority AI's mission is to bring clarity to the earliest and riskiest stages of pharmaceutical research. Opening this directory to everyone is a first step: the more researchers, R&D teams, investors, and patient advocates who can see where the unmet need and the real opportunities lie, the faster new therapies can move from paper to patient.

Explore the full directory at explority.ai/diseases, or talk to our team about getting access to Explority AI's full probability-of-success forecasts.

ready to solve rare diseases?

Get access to Explority AI's forecasts to outperform average preclinical success rates. Whether you're planning your next partnership, selecting next R&D idea or just have questions - drop us a message. Let’s explore how we can work together to solve rare diseases.

From insights to

impact.

impact.

Let's accelerate therapies for rare diseases affecting over 350 million people worldwide.

From insights to

impact.

impact.

Let's accelerate therapies for rare diseases affecting over 350 million people worldwide.

From insights to

impact.

impact.

Let's accelerate therapies for rare diseases affecting over 350 million people worldwide.
Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.