RegenXBio Secures $110M Upfront in Strategic Partnership with Nippon Shinyaku for Hunter Syndrome Gene Therapy

Strategic Partnership:
RegenXBio has entered into a strategic partnership with Nippon Shinyaku to advance novel gene therapies for mucopolysaccharidosis type II (Hunter syndrome) and type I4.

Financial Terms:
The deal includes an upfront payment of $110 million to RegenXBio, with potential milestones reaching up to $700 million. Additionally, RegenXBio will receive double-digit royalties on net sales of the gene therapies in the U.S. and Asia4.

Gene Therapies Involved: The partnership involves two gene therapies:
RGX-121 for Hunter syndrome and RGX-111 for mucopolysaccharidosis type I. RGX-121 has shown promising results in reducing cerebrospinal fluid levels of a key disease biomarker and improving neurodevelopmental skills in treated children24.

Regulatory Progress:
RGX-121 has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, which is designed to expedite drug development and review processes for promising new treatments5.

Market Impact:
The announcement led to a significant surge in RegenXBio's stock price, increasing by nearly 20% before the opening bell on Tuesday4.

Sources:

2. https://www.prnewswire.com/news-releases/regenxbio-announces-pivotal-trial-of-rgx-121-for-the-treatment-of-mps-ii-achieves-primary-endpoint-302056283.html

4. https://www.biospace.com/business/regenxbio-jumps-on-potential-800m-rare-disease-deal-with-nippon-shinyaku

5. https://www.prnewswire.com/news-releases/regenxbio-receives-fda-regenerative-medicine-advanced-therapy-rmat-designation-for-rgx-121-gene-therapy-for-hunter-syndrome-301831944.html