Breaking News: Regenxbio and Nippon Shinyaku Join Forces to Fight MPS Diseases with RGX-121 and RGX-111
Description:
REGENXBIO to receive $110 million upfront, potential milestone payments of up to $700 million and meaningful double-digit royalties on net sales, and lead manufacturing Nippon Shinyaku to lead commercialization of first potential gene therapies for Mucopolysaccharidosis II (MPS II) and Mucopolysaccharidosis I (MPS I) in U.S. and Asia REGENXBIO retains rights to RGX-121 Priority Review Voucher (PRV) with potential accelerated approval expected in 2025; rolling BLA submission underway ROCKVILLE, Md. , Jan. 14, 2025 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX) and Nippon Shinyaku Co., Ltd.
Breaking Barriers in Gene Therapy for MPS Diseases
In a groundbreaking collaboration, Regenxbio and Nippon Shinyaku have come together to combat MPS diseases with the development of RGX-121 and RGX-111. Mucopolysaccharidoses (MPS) are a group of inherited metabolic diseases, caused by the deficiency of enzymes needed to break down complex sugars. These diseases can lead to a range of serious health issues, affecting multiple organs and systems in the body.
With the emergence of gene therapy as a promising approach to treating genetic disorders, the partnership between Regenxbio and Nippon Shinyaku marks a significant step towards addressing the unmet medical needs of individuals suffering from MPS II and MPS I. RGX-121 and RGX-111 hold the potential to provide a targeted and long-lasting solution to these debilitating conditions.
Empowering Patients with Innovative Treatments
By combining Regenxbio’s expertise in gene therapy with Nippon Shinyaku’s commercialization capabilities, the collaboration aims to bring these groundbreaking therapies to patients in the U.S. and Asia. The upfront payment of $110 million, along with potential milestone payments and royalties, underscores the value and potential impact of this partnership in the field of rare genetic diseases.
Moreover, the retention of rights to the RGX-121 Priority Review Voucher (PRV) positions Regenxbio for potential accelerated approval, with a rolling Biologics License Application (BLA) submission already underway. This expedited regulatory pathway could pave the way for the timely availability of these life-changing therapies to individuals affected by MPS II and MPS I.
Looking Towards a Brighter Future
As the landscape of gene therapy continues to evolve, collaborations like the one between Regenxbio and Nippon Shinyaku exemplify the power of partnership in driving innovation and advancing patient care. The potential approval and commercialization of RGX-121 and RGX-111 could herald a new era in the treatment of rare genetic diseases, offering hope and improved quality of life to individuals and families impacted by MPS conditions.
Effect on You:
As a potential recipient of gene therapy for MPS diseases, this collaboration could bring new treatment options and hope for a better quality of life. The development and commercialization of RGX-121 and RGX-111 may offer a targeted and long-lasting solution to the health challenges posed by MPS II and MPS I.
Effect on the World:
The partnership between Regenxbio and Nippon Shinyaku represents a significant advancement in the field of gene therapy, with the potential to impact individuals globally affected by rare genetic disorders like MPS II and MPS I. The development and availability of novel treatments could transform the lives of patients and families worldwide, ushering in a new era of hope and progress in the fight against debilitating genetic diseases.
Conclusion:
The collaboration between Regenxbio and Nippon Shinyaku to combat MPS diseases with RGX-121 and RGX-111 marks a significant milestone in the field of gene therapy. As these innovative treatments progress towards potential approval and commercialization, the potential benefits extend to individuals affected by MPS II and MPS I, as well as the broader community of rare disease patients. This partnership symbolizes the power of collaboration and innovation in driving positive change in healthcare and advancing the treatment of genetic disorders.
