From Bottleneck to Backbone: Building the U.S. “Interventional-Genetics Interstate”
Pioneer of next-generation gene editing, David Liu’s challenge at last week’s FDA cell and gene therapy round-table was stark: if the United States can compress concept-to-clinic manufacturing to “well under 90 days,” at least a thousand ultra-rare patients could receive bespoke in-vivo gene-editing therapies by 2030. The underlying science may be ready, but the physical and digital scaffolding that would let those therapies flow is not. Below is a high level review of where the U.S.’s infrastructure currently stands, where the cracks lie, and how proven international models can help finish the road in time.
A patchwork that almost works
Start-ups and academics have shown glimpses of what an agile infrastructure might look like. Philadelphia’s CHOP/UPenn Clinical Vector Core has produced 181 GMP vectors for 37 INDs touching 859 patients since 2007, showing what lean, mission-driven facilities can do over time. Vector production timelines remain a bottleneck for the field - Andelyn Biosciences’ 16-suite Columbus headquarters released its first clinical AAV lot in May 2025 ten months after tech-transfer began. Other modalities are emerging to address the shortcomings of vectors - in Boston, Landmark Bio’s 44 k ft² “full-spectrum” facility producing mRNA, LNPs and traditional vector products has been running since late 2022, providing eight re-configurable cleanrooms within walking distance of the Harvard–MIT medical cluster. Federally, the Bespoke Gene Therapy Consortium (BGTC) selected eight prototype diseases in May 2023 to demonstrate a repeatable regulatory-and-manufacturing playbook, while NINDS’ URGenT Network continues to fund IND-enabling work for ultra-rare neurologic disorders under its January 2025 funding notice (PAR-25-326).
Yet capacity is brittle. National Resilience, once the poster-child for “mega-factory” gene-therapy CDMOs announced yesterday, that it will close six of its ten U.S. sites because “capacity expansion has outpaced demand”. The message is clear: square footage alone does not guarantee throughput.
The gap between ambition and reality
Even with bright spots, most sponsors still wait nine months or more for GMP vector slots, rely on site-specific QC assays, and repeat pre-clinical toxicology from scratch; miles away from Liu’s sub-90-day aspiration. The United States currently lacks:
Geographic reach. Production remains concentrated in a handful of coastal hubs; patients elsewhere often cross state lines for dosing.
Shared digital infrastructure. Batch-release data live in siloed LIMS, making cross-site comparability almost impossible.
Economic resilience. Large plants built for blockbuster demand now sit idle, while hospital vector cores cannot scale beyond a dozen lots per year.
Lessons from health systems that are already doing it
The U.K.’s Advanced Therapy Treatment Centre (ATTC) Network embeds GMP suites inside NHS hospitals and links them through a national digital backbone. In February 2025 the UK government injected another £17.9 million to extend the model, explicitly to accelerate early-phase ATMP trials across the country. Every centre runs 2–4 multipurpose rooms and shares a harmonised set of SOPs - exactly the federated approach Liu argues the U.S. needs.
Singapore’s ACTRIS hub takes the same idea to city-state scale: 14 GMP-compatible suites, four translational labs and a single QC core feed hospitals, academics and start-ups under one roof, coordinated by a government logistics task-force. Both systems show that small, interoperable nodes can beat single mega-sites on utilisation, workforce agility and patient proximity.
Investing in nimble, connected manufacturing infrastructure today paves an Interventional-Genetics Interstate that will shorten timelines, cut costs and most importantly get transformative treatments to rare-disease patients faster.
How a U.S. Interventional-Genetics Interstate could take shape
Repurpose idle space. One wing of a downsized Resilience plant could house several 500 ft² closed-system pods tailored to micro-batch gene-editing runs, while the rest converts to warehousing and analytics. That keeps sunk capital alive without overshooting local demand.
Create a national QC cloud. Require BGTC and URGenT awardees to deposit release data into a shared LIMS opening the door to “plug-in” manufacturing sites that regulators can audit remotely.
Adopt a “grants-not-guarantees” funding model. NIH C06 facility grants and state matching schemes already release construction dollars in tranches tied to occupancy or throughput milestones; the same approach could seed regional micro-factories without repeating past oversupply.
Spin a workforce fly-wheel. Short, NIIMBL-style bootcamps that rotate technicians between hospital cores, CDMOs and start-ups would create a steady talent circuit instead of one-off hiring sprees.
Institutionalise digital twins. Inline PAT sensors plus cloud process models now routine in UK Catapult pilots, can cut tech-transfer time and give the FDA live visibility, shaving weeks off each bespoke batch.
What it means for technology suppliers
For analytics and PAT vendors, interoperability will clinch procurement decisions. Equipment makers should design skids that fit tight footprints and swap quickly between AAV, LNP and mRNA workflows. Digital-platform providers can own the value chain by stitching together batch genealogy from lab bench to hospital bedside. In short: if your tools make micro-batch production faster, cheaper and easier to regulate, the Interstate will need you.
The road ahead
The United States already has all the raw materials: pioneering science, ambitious CDMOs, and a small but growing federal appetite for public-private gene-therapy ventures. What it lacks is connective tissue. The next five years will decide whether the U.S. can deliver a federated, nimble network that can crank out micro-batches for 7,000+ ultra-rare conditions. Borrowing the ATTC formula of regional hubs plus national standards, and the ACTRIS emphasis on co-located clinical-manufacturing ecosystems, could close today’s fissures faster than any single plant investment. The prize is tangible: a future in which a child diagnosed this morning could receive a customised in-vivo therapy before the end of the school term.
