FDA places clinical hold on REGENXBIO's RGX-111 and RGX-121 gene therapies after neoplasm case

REGENXBIO Inc.

Single neoplasm (intraventricular CNS tumor) found in 5-year-old asymptomatic patient 4 years post-RGX-111 dose; AAV vector integration detected near PLAG1 proto-oncogene.
RGX-121 placed on hold due to product, population, and risk similarities with RGX-111.
No neoplasms reported in 9 other RGX-111 or 32 RGX-121 patients.
CEO surprised by RGX-121 hold while investigation continues; confident in benefit-risk based on positive safety and efficacy data.
Company has not yet received full clinical hold letter and awaits FDA details.

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