This collaboration marks a significant advancement towards finding a treatment for this fatal autosomal recessive disorder.
The initiative is significantly supported by the Riaan Research Initiative, a nonprofit patient-advocacy organization, which has provided the largest known donation towards Cockayne syndrome research. This partnership between Andelyn and UMass Chan Medical School, bolstered by the Riaan Research Initiative’s contribution, brings clinical trials for this devastating condition closer to reality.
Advancing gene therapy
Miguel Sena-Esteves associate professor of neurology and director of the Translational Institute for Molecular Therapeutics at UMass Chan Medical School, expressed his optimism about the collaboration: “Our dream of making a difference for Cockayne patients is coming closer every day, and signing the contract with Andelyn Biosciences to make the clinical material is a major step in that direction.
“Developing a gene therapy for fatal pediatric neurological diseases is challenging in many ways, but together with Riaan Research Initiative we have an outstanding team to bring our program to a first-in-human gene therapy clinical trial for Cockayne syndrome.”
Cockayne syndrome is a rare genetic disorder characterized by developmental and neurological degeneration. Currently, there are no effective treatments, making this collaboration a beacon of hope for affected families.
Andelyn’s role and expertise
Matt Niloff, chief commercial officer of Andelyn, emphasized the significance of their involvement in this project. “We are honored to have been selected to be a key contributor in the advancement of this critical therapy for Cockayne syndrome. With more and more organizations across the industry adopting our data-driven and configurable AAV Curator™ Platform, we are excited to add UMass as an important partner leveraging the platform to drive the acceleration of the Cockayne syndrome program.”
Andelyn Biosciences is renowned for its high-quality and scalable end-to-end development and manufacturing capabilities. Their expertise will be pivotal in industrializing the production process for the AAV9-CSA vector, ensuring consistent and reliable manufacturing of this gene therapy. Their facilities in Columbus, Ohio, are equipped to meet the rigorous demands of such innovative treatments.
Looking ahead
The collaboration between Andelyn Biosciences and UMass Chan Medical School represents a significant milestone in the fight against Cockayne syndrome. By harnessing Andelyn’s advanced manufacturing capabilities and UMass Chan’s pioneering research, the partnership aims to accelerate the development of a viable gene therapy for this challenging disorder.
The successful production of clinical-grade AAV9-CSA vectors will pave the way for first-in-human clinical trials, offering hope for a treatment that could significantly improve the lives of those affected by Cockayne syndrome.
With ongoing support from organizations like the Riaan Research Initiative, the path forward for gene therapy treatments for rare diseases looks promising. This partnership exemplifies how collaborative efforts and innovative technologies can drive progress in the field of gene therapy, bringing life-changing treatments closer to those in need.