Chief Executive Officer & Executive Director, Uniqure Nv
Search every verified Matthew Kapusta interview, podcast appearance, and on-the-record quote — each transcript cross-checked by AI and human review to confirm speaker identity. Matthew Kapusta, CEO of uniQure, has discussed the company's gene therapy Hemgenix, which he described as a one-time treatment for Hemophilia B that provides constant Factor 9 expression. Kapusta stated that Hemgenix is now offered to 10,000 to 15,000 patients across Europe and the United States, and he noted that clinical data from three studies involving about 70 patients showed a greater than 90% reduction in infusions, with nearly all patients remaining off prophylactic replacement therapy. He characterized the therapy as potentially more cost-effective than existing treatments, citing annual replacement therapy costs of $500,000 to $600,000 for severe Hemophilia B. Kapusta has also highlighted uniQure's history as a gene therapy pioneer, noting the company was founded in 1998 and achieved two approved gene therapies. He emphasized the company's investment in manufacturing capabilities, stating that uniQure controls its manufacturing process and built facilities from the ground up. In earlier remarks, Kapusta discussed the economic value of gene therapies, describing the potential for a one-time administration to replace ongoing high-cost treatments, and noted that pricing would require discussions with payers.
“Uniqure is one of the first pioneers within gene therapy; the company was founded in 1998 and has achieved two approved gene therapies, including Hemgenix, the first gene therapy for the treatment of Hemophilia B, which is currently on the market in the United States, Europe, and other countries.”
“Hemgenix, partnered with CSL Behring as our global commercial partner, is now being offered to 10 to 15,000 patients with Hemophilia B across Europe and the United States, representing a transformative therapy that provides constant Factor 9 expression over time and is theoretically one and done.”
“Clinical development across three different studies involving about 70 patients shows remarkable durability with Hemgenix, including greater than 90% reduction in the amount of infusions and nearly all patients continuing to be off prophylactic replacement therapy.”
“Patients treated with Hemgenix report feeling like they have their life back again, freed from the burden of their disease, with improvements in activity levels, job retention, and overall quality of life.”
“Existing therapies for severe Hemophilia B can cost upwards of $20 million over a patient's lifetime, with annual replacement therapy costing $500,000 to $600,000, making Hemgenix not only a more convenient and durable option but also potentially more cost-effective in the long term.”
“There is increasing evidence that Hemgenix is superior in preventing bleeds and providing hemostatic control compared to existing therapies, offering significant value to multiple stakeholders throughout the healthcare continuum.”
“The purpose of gene therapy is for one-time administration that can have very durable effects. The first gene therapies in hemophilia have now followed patients for more than eight years and showed durable effects, so it's looking very good.”
“We announced some very positive results for our phase 2b study. We are currently enrolling patients in the pivotal study and will be providing guidance on the process of enrollment early next year.”
“Our first goal is to complete the enrollment of the study. We'll be looking at factor 9 activity, which is the biomarker for efficacy, about 6 months after we treat the last patient, and then bleeding data after one year of enrolling the last patient as well.”
“There's probably around 400,000 people in the world that have hemophilia, approximately 70,000 of those have hemophilia B today. There's probably more than 10 billion dollars of revenue predominantly going after factor replacement for those patients that require infusions.”
“The notion of that much revenue a year in fact replacement being replaced by a one-time administration is pretty attractive for the patients and certainly the healthcare economic community.”
“There's probably around five to seven thousand patients with hemophilia B in the United States, and probably four times that many that have hemophilia A.”
“The cost of care for severe hemophilia, the infusions that patients take, can be somewhere around six to seven hundred thousand dollars a year, and that's for the rest of that patient's life.”
“If you have a gene therapy that can potentially provide curative benefit, there's no doubt significant healthcare economic value, and obviously the exact price of that will have to require discussions with the payer community as well.”
“In total over the last 20 years we've raised about 450 million dollars so an enormous amount of money just with one company that had the mission of really bringing these transformative gene therapies to patients.”
With approvals from the United States and the European Union, gene therapy is becoming more accessible to patients with ...
Matt Kapusta, CEO of Uniqure, discusses his company soaring on hope for a new hemophilia gene therapy treatment.
Delivering Promise of Gene Therapy.
uniQure's CEO Matt Kapusta discusses gene therapy's potential to revolutionize the healthcare industry, and uniQure's mission to ...
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