Regenxbio’s Gene Therapy Faces Another FDA Setback in Hunter Syndrome

Six Montgomery County companies raised over $566 million in Q3. Forbright (Nasdaq) completed an IPO, raising $142.2 million. Xometry (Nasdaq) announced a $225 million secondary offering. RegenxBio (Nasdaq) raised $90.04 million in a public offering. Georgiamune secured $92.69 million in venture funding. Arculus Solutions raised $1.95 million in Seed-1 funding. Jesse & Ben raised $15 million in Series A funding.

REGENXBIO (RGNX) faces an FDA clinical hold on its Hunter Syndrome gene therapy (RGX-121) due to asymptomatic spinal MRI findings in five participants. The company is evaluating additional data with partner NS Pharma. Other late-stage programs, including RGX-202 for Duchenne muscular dystrophy and surabgene lomparvovec for wet AMD, remain on track. The Hunter Syndrome treatment market is projected to grow to $1.8B by 2030, according to Grandview Research.

Regenxbio's stock fell 25% to $8.05 after the FDA placed a clinical hold on its gene therapy candidate RGX-121 due to spinal nodules in five trial participants. The company halted near-term BLA resubmission plans for RGX-121 but will file a BLA for its Duchenne treatment this quarter and expects wet AMD trial results in Q4. According to the company, the nodules are likely benign, but the FDA required further review.

REGENXBIO (RGNX) announced an FDA clinical hold on RGX-121, its gene therapy for MPS II, due to asymptomatic spine MRI findings in five participants. The company does not expect to resubmit the BLA soon and will focus on other candidates. Investigators consider the findings likely benign and will continue monitoring. REGENXBIO plans to evaluate additional data and FDA feedback for next steps. MPS II is a rare genetic disorder affecting approximately 2,000 patients worldwide.

Regenxbio (RGNX) stock fell 24.3% to $8.12 after the FDA placed a clinical hold on its RGX-121 gene therapy due to safety concerns. The company delayed resubmitting the therapy's Biologics License Application. Analysts had recently downgraded the stock. The broader market showed minor declines, and peers were unaffected.

REGENXBIO Inc. (NASDAQ:RGNX) shares dropped 35% after the FDA placed a clinical hold on its gene therapy RGX-121 for MPS II due to asymptomatic spine MRI findings in five participants. The company does not expect to resubmit the BLA soon but remains focused on other candidates, including Duchenne and retinal disease treatments. REGENXBIO plans to submit a BLA for Duchenne this quarter and announce wet AMD data in Q4.