As Rare Disease Day 2026 shines a spotlight on patients living with life-limiting conditions, Cereno Scientific is focusing on disease-modifying therapies for rare cardiovascular and pulmonary diseases, including pulmonary arterial hypertension (PAH).

Despite progress in rare disease drug development, many patients with PAH still face delayed diagnosis and limited treatment options that primarily manage symptoms rather than target the biological drivers of disease. With new clinical data emerging and late-stage development underway, the company says its ambition is to move beyond symptom control toward therapies that may slow, halt or potentially reverse disease progression.

Below, Sten Sörensen, CEO of Cereno Scientific, outlines how the company is advancing its HDAC inhibitor platform, expanding access through clinical development and engagement with the rare disease community, and preparing for the next phase of global trials as Rare Disease Day 2026 puts the focus on turning awareness into action.

How is your company helping improve awareness, diagnosis, or treatment access for rare diseases?

At Cereno Scientific, our mission is to pioneer treatments that enhance and extend life for people living with rare cardiovascular and pulmonary diseases. In conditions such as pulmonary arterial hypertension (PAH), patients often face delayed diagnosis and therapies that manage symptoms but do not fully address the underlying disease.

Cereno’s pioneering drug development is built on our epigenetic HDAC inhibitor platform, represented by CS1 and CS014. The platform is designed to target fundamental biological processes including vascular remodeling, fibrosis, inflammation, and thrombosis, which drive disease progression across many rare cardiopulmonary conditions. Our ambition is to move beyond symptom control and toward disease modification, to slow down, halt and possibly revert the disease progression.

Following encouraging Phase 2a data, CS1 is now entering a global Phase 2b development phase in the rare cardiovascular disease PAH supported by a Fast Track designation and Orphan Drug Designation (ODD) in the US and Europe. In this effort, we will engage closely with the rare disease community, including patient organizations such as PHA Europe.

Our Expanded Access Program has further allowed eligible patients to continue treatment for 12 months following the Phase 2a trial with CS1.

Rare Disease Day reminds us that behind every rare diagnosis is a person living with uncertainty and limited treatment options. For me personally, Rare Disease Day is about turning awareness into action — accelerating innovation so that new therapies that can extend and enhance life will reach patients as efficiently as possible.

What new initiatives or partnerships are you most proud of that directly support rare disease communities?

What I am most proud of is that we have moved from early clinical promise to global execution.

With CS1 now entering a global Phase 2b trial, we are working alongside a distinguished Clinical Steering Committee led by Professor Marc Humbert, a leading PAH expert, and executed in partnership with a leading global CRO. This Phase 2b trial will enrol its first patient in the second quarter of 2026 in the US, followed by Europe and Latin America. This represents an important step forward for patients living with PAH.

At the same time, our next-generation HDAC inhibitor, CS014, is progressing toward Phase 2 development in pulmonary hypertension associated with interstitial lung disease (PH-ILD), reinforcing our commitment to the promising potential of our epigenetic platform through HDAC inhibition and its ability to address the underlying drivers of disease.

Equally important is our ongoing dialogue with the rare disease community. Collaboration between clinicians, researchers, patient organizations and industry are essential if we are to change the trajectory of these serious conditions.

As Rare Disease Day approaches on February 28, it reinforces why we pursue this work: to develop pioneering therapies that have the potential to slow down, halt or reverse disease progression.