BioSpace News Archive

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Insights

Single Pivotal Trials Demand Stronger Data and Risk Strategies

Single-trial approvals are raising the bar on trial design and execution. The new paradigm is pushing sponsors to plan earlier, step up their data and risk‑based quality management and use modeling and AI to generate one compelling, regulator‑ready evidence package.

March 10, 2026

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3 min read

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Jennifer C. Smith-Parker

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Duchenne muscular dystrophy

Dyne Plans Post-Prasad FDA Run as Duchenne Exon Skipper Sustains Benefit in Long Term Data

Dyne Therapeutics is plotting an approval application for z-rostudirsen in the back half of 2026—a push that will only be bolstered by the departure of controversial CBER chief Vinay Prasad, according to analysts at Stifel.

March 10, 2026

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3 min read

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Tristan Manalac

NextGen

Rising Amid Flurry of CAR T Deals, Stylus Proves Cell Therapy Is Not Dead

Stylus Medicine, a member of BioSpace’s NextGen Class of 2026, launched in May 2025 to develop new, less complex genetic medicines. The company’s in vivo approach has attracted “intense” interest from Big Pharma.

March 10, 2026

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2 min read

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Annalee Armstrong

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Funding

AI-Focused Fund Breakout Raises $114M To Support Early-Stage Biotechs

Breakout Ventures’ focus on early-stage companies stands out as more and more investors elect to save their dollars for derisked assets.

March 10, 2026

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2 min read

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Tristan Manalac

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Manufacturing

Cell and Gene Therapy Manufacturing Market To Skyrocket to $146B by 2032

Analysts expect the market for manufacturing cell and gene therapies, worth less than $20 billion in 2024, to expand rapidly as approvals drive higher volumes of production.

March 10, 2026

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3 min read

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Nick Paul Taylor

BioNTech CEO Ugur Sahin and Chief MEdical Officer Özlem Türeci.

C-suite

BioNTech Founders To Leave Famed mRNA Biotech for New Venture

The move comes as BioNTech shifts to being a multiproduct commercial biotech, allowing Ugur Sahin and Özlem Türeci to transition back into research on next-generation mRNA therapeutics.

March 10, 2026

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2 min read

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Annalee Armstrong

External view of the U.S. Capitol Building

Rare diseases

Senator Ron Johnson Launches Investigation Into FDA Rare Disease Rejections

The senator, who has long advocated for expanding access to experimental therapies, reportedly called the FDA’s request for a sham surgery–controlled Phase 3 trial for uniQure’s Huntington’s disease gene therapy “bureaucratic idiocy.”

March 10, 2026

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3 min read

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Heather McKenzie

FDA signage at its headquarters in Maryland

Manufacturing

FDA Sets Scope for Attempt To Reduce Manufacturing-Related Approval Rejections

Industry and FDA representatives have reached a general agreement on planned pre-submission facility meetings but have expressed different views about the specifics.

March 10, 2026

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2 min read

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Nick Paul Taylor

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Manufacturing

Nonprofit Aims To Modernize Manufacturing by Clearing Capital Hurdle

Industry groups have identified upfront costs as a barrier to streamlining U.S. drugmaking. The nonprofit API Innovation Center has a proposed answer for how to tilt finances in favor of investments in continuous manufacturing.

March 10, 2026

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5 min read

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Nick Paul Taylor

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IgA nephropathy

Vertex’s Nephropathy Asset Delivers ‘Strong’ Late-Stage Data, Clears Path to FDA

Vertex Pharmaceuticals has a rolling biologics license application with the FDA for povetacicept in IgA nephropathy. With new data from RAINIER, the biotech expects to complete its submission by the end of March.

March 10, 2026

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2 min read

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Tristan Manalac