Matthew Kapusta on gene therapy
From Gene Therapy for Hemophilia: ARM Studios Interview with Matthew Kapusta · · Alliance for Regenerative Medicine
“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.”
On , Matthew Kapusta, Chief Executive Officer & Executive Director at UNIQURE NV, spoke about gene therapy during Gene Therapy for Hemophilia: ARM Studios Interview with Matthew Kapusta on Alliance for Regenerative Medicine.
About Matthew Kapusta
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.