Cell therapy weekly: Pfizer reports positive results from Phase III hemophilia B clinical trial
This week: FUJUFILM Cellular Dynamics and Novo Nordisk entered into a license agreement to develop induced pluripotent stem cell therapies, Pfizer reports positive results from Phase III hemophilia B clinical trial, Capsid Biotherapeutics join forces with Prevail Therapeutics to develop gene therapies.
- FUJUFILM Cellular Dynamics and Novo Nordisk enter license agreement to develop induced pluripotent stem cell therapies
FUJIFILM Cellular Dynamics (WI, USA), a leading developer and manufacturer of human induced pluripotent stem cells have entered a license agreement with Novo Nordisk (Bagsværd, Denmark). Novo Nordisk will receive non-exclusive rights to FUJIFILM’s human induced pluripotent stem cell lines and GMP-grade cell lines to develop potential cell therapies for chronic diseases.
Tomoyuki Hasegawa, CEO, Fujifilm Cellular Dynamics stated: “Fujifilm Cellular Dynamics is a global leader in the development and manufacturing of GMP-grade iPSCs for cell therapy applications. Our goal is to drive the iPSC field forward by building strategic partnerships. Novo Nordisk is strongly positioned to be a pioneer in delivering cell-based treatments to tackle serious chronic diseases based on iPSC-derived therapeutics.”
- Pfizer reports positive results from Phase III hemophilia B clinical trial
Pfizer (NY, USA) has reported positive results from a phase III trial evaluating the efficacy and safety of fidanacogene elaparvovec, a gene therapy candidate. The drug, targeted to patients with moderately severe to severe hemophilia B, is comprised of a bio-engineered adeno-associated virus capsid and coagulation FIX gene. The results, shared as the trial met its primary endpoint, reveal an average bleeding rate reduction of 71% in participants over a one-year period.
“The BENEGENE-2 data demonstrate the promise of this gene therapy candidate as a potential one-time option for people living with hemophilia B as a means of reducing the clinical and treatment burden over the long term,” said Adam Cuker, Director of the Penn Comprehensive and Hemophilia Thrombosis Program (PA, USA).
- Capsida Biotherapeutics joins forces with Prevail Therapeutics to develop gene therapies
Capsida Biotherapeutics (CA, USA) have announced a multi-year strategic partnership with Prevail Theraputics (NY, USA). Prevail will leverage Capsida’s expertise in adeno-associated virus technology and capsids to develop intravenous gene therapies that are effective at targeting diseases of the central nervous system.
“We are thrilled to be entering into this strategic collaboration with Prevail. Prevail’s expertise in neuroscience, gene therapy R&D, and access to Lilly’s world-class commercialization capabilities complements Capsida’s fully integrated approach, including our next-generation AAV engineering platform. Prevail and Capsida are committed to developing highly effective and safe gene therapies that have the potential to be transformative for patients living with serious CNS diseases,” commented Peter Anastasiou, CEO of Capsida.
