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What we do

We are accelerating access to treatments for rare disease patients in two ways: helping healthcare systems find and diagnose their undiagnosed patients, and by developing promising new drug therapies.   

Our pipeline

Our drug therapy pipeline

We breathe new life into assets that have not been fully unlockedWe acquire drug candidates that demonstrate, based on existing human safety and efficacy data, the potential for approval in rare disease indications where there's no current treatment.  

We are building a broad, late-stage portfolio that is designed to deliver regulatory approvals in a shorter time and with higher probability than more traditional early-stage biotechnology business models.

Explore our acquisition-based rare disease pipeline

Val401

Phase 2

QoL palliative care/cachexia in pancreatic cancer

Renzapride

Phase 2

Gastrointestinal symptoms in cystic fibrosis

Our MagnetID  service

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We help healthcare systems, like hospitals better identify and diagnose rare disease patients by analyzing existing electronic health records, drawing out rare disease cases like a magnet finding needles in a haystack. 

MagnetID  is powered by a validated AI platform developed over the last ten years. It identifies patients’ symptoms as described within their electronic health records and compares them to the symptoms for the target rare disease(s), with the potential to search for all 7,000 rare diseases.

 

The results can direct physicians and healthcare providers to confirmatory diagnosis. They can also provide additional clinical, cost and commercial insights into rare disease populations and data for resource prioritization. ​

 

Whether you are searching for a single rare disease, or a list of many, among your patient population, MagnetID  can help. 

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