With proof of concept for its NAV platform from two gene therapy products partners have brought to the market, two products slated for regulatory submission in the next two years, and a new manufacturing facility, Regenxbio Inc. is poised to take the next step to becoming a fully realized commercial biotech, CEO Kenneth Mills told Scrip in a recent interview.
Regenxbio: On The Cusp Of Becoming A Commercial Gene Therapy Player
Partnered with AbbVie on a wet AMD gene therapy, Regenxbio is also advancing wholly owned candidates for Duchenne muscular dystrophy and MPS I and MPS II.

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Bharat Biotech is the latest entrant in India’s cell and gene therapy space, joining majors like Intas Pharma, Sun Pharma, Cipla and Dr. Reddy’s. An indigenously developed CAR-T cell therapy launched last year, what is driving growing interest and success in cell and gene therapies?
Deal Snapshot: Lilly is the third company to sign a licensing deal for STAC-BBB with Sangamo, which also aims to secure a deal for its Fabry disease program in the second quarter.
The company closed a $150m series C funding round shortly after opening the second part of its Phase I/II study of ATSN-201 and partnering with a Nippon Shinyaku on ATSN-101.
The promise of innovative therapies seems to have been constrained not by efficacy or safety concerns, but because the high price of treatments is incongruous with the reimbursement of short-course therapies.
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Seeking cell therapy approaches to cure type 1 diabetes, Vertex abandons a candidate encapsulated to avoid immune system detection but hopes to file another candidate for approval in 2026.
Kyoto-based venture moves HQ to California to expand R&D and business outreach for its regulatory T-cell technology, as it raises around $46m in public and private funding.
The promise of innovative therapies seems to have been constrained not by efficacy or safety concerns, but because the high price of treatments is incongruous with the reimbursement of short-course therapies.