Podcast recap: Benny Sorenson on patient-centered drug development for clotting disorders

In the latest episode of The Genetics Podcast, we sat down with Dr. Benny Sorenson, CEO of Hemab Therapeutics, to explore a relatively overlooked therapeutic area: bleeding disorders beyond hemophilia. While innovation in hemophilia care has surged over the past decades (with more than 30 approved therapies today), many other serious clotting conditions have seen little progress since the 1960s. Benny and the team at Hemab are aiming to change that.

The patient-centered origin of Hemab

Benny emphasizes that the foundation of Hemab’s approach is empathy. Before building a pipeline or selecting a modality, the team begins by listening to patients. One of the company’s earliest programs, targeting Glanzmann thrombasthenia, emerged not from clinical data but from persistent conversations with patients whose symptoms were being underestimated. Despite receiving feedback from clinical peers that these patients “don’t really bleed,” Benny pushed further. He found a condition marked by chronic, often life-threatening hemorrhages that demanded a preventive solution.

The company has since developed a bispecific antibody that mimics a validated mechanism and has shown encouraging results in phase 2 trials. Hemab is also advancing programs in Von Willebrand disease and Factor VII deficiency, tailoring each to the needs of patients based on deep engagement and natural history studies.

Beyond hemophilia: Addressing the gaps

While hemophilia A and B have benefited from technological revolutions, including plasma-derived products, recombinant therapies, gene therapy, and bispecific antibodies, many other bleeding disorders have been left behind. Conditions like Glanzmann thrombasthenia and Von Willebrand disease affect tens of thousands globally, and many patients remain undiagnosed, untreated, or inadequately managed.

Hemab’s approach to this problem is to build around deep domain expertise. The company’s small R&D team has contributed to 60% of all approved products in the clotting space. Rather than center development around a single modality, Hemab tailors therapies to each unique condition.

Inclusion and gender equity in autosomal bleeding disorders

A key insight from the conversation was the shift from X-linked to autosomal disorders. Unlike hemophilia, which predominantly affects men, autosomal bleeding disorders affect men and women equally. Yet women – particularly those experiencing heavy menstrual bleeding – have been historically neglected in both diagnosis and drug development. Hemab is actively working to reverse this disparity by designing trials and treatments that fully account for the lived experiences of all patients, regardless of gender or geography.

Benny also reflected on the importance of global representation in data collection. From structured natural history studies to digital outreach, Hemab is building an inclusive research infrastructure that spans rural clinics and major research hospitals alike.

A philosophy of persistence

Throughout the discussion, Benny consistently reinforces his belief in being “unreasonable” – a mindset he credits to his time at Alnylam. For Benny, being unreasonable means refusing to stop at conventional thinking and always pushing for a deeper understanding of diseases and patient perspectives. This approach has not only shaped Hemab’s scientific direction but also its culture. As Benny puts it, the company may be small in size, but it achieves a lot by partnering with domain experts, tapping into global networks, and building with purpose.

Looking ahead

Hemab is preparing to initiate a pivotal trial for its Glanzmann thrombasthenia program, expand into new indications like Factor VII deficiency, and deepen its engagement with patients and advocacy groups. The company is also planning to launch additional programs with different modalities, based on what the biology and the patients demand.

By centering empathy and expertise, Hemab is working to bring modern, preventive, and truly transformative therapies to patients with bleeding disorders who have long been underserved.

Listen to the full episode below.