That bottleneck has become one of the biggest constraints on the next decade of cell and gene therapy growth, with hundreds of late-stage programs chasing a manufacturing footprint that has not expanded fast enough to match them.
An estimated 450,000 cancer patients were eligible for CAR-T in 2019; that number is projected to hit 2 million by 2029.
A bioethicist with a PhD in molecular genetics and a seat on the Vatican's Pontifical Academy for Life sits in a rare position to bridge the two.
Behind every approval sits a medical-safety sign-off that determines whether a one-shot cell or gene therapy reaches European patients.
Most CROs were built for small-molecule oncology, not for therapies that have to be manufactured fresh per patient and shipped under tight cold-chain windows.
Arbor Biotechnologies' CRISPR-based gene editor ABO-101 is being trialled as a one-time in vivo fix, with the redePHine Phase 1/2 study enrolling up to 23 adult and paediatric patients at Mayo Clinic and additional sites in the US, UK and Europe.
Cell and gene therapy manufacturing still runs largely on paper records and disconnected spreadsheets, and unlike a recalled small molecule, a single batch failure on a personalised therapy can cost a patient their life.
Circular RNA is roughly an order of magnitude more stable than linear mRNA, extending expression of a therapeutic protein from one day to about a week and unlocking dramatically lower dosing of AAV gene therapies.
The MHRA cleared 84 clinical trial applications in its first 60-day window last year – and Lawrence Tallon wants the next 60 days to look nothing like that.
Over 400 organisations across 25 countries are racing to bring these therapies to patients with otherwise untreatable diseases.
Stuart Therapeutics is testing the first collagen mimetic peptide for dry eye disease – a novel mechanism that produced Week 1 staining gains vs. vehicle greater than any reported result for an approved therapy in their recent Phase 3.
A single CAR-T dose still takes up to 33 days to manufacture, at $170,000–$220,000 per batch.
Stuart Curbishley on how adthera bio is tackling the biggest bottleneck in cell and gene therapy: getting therapies manufactured at scale.
Venture investment in cell and gene therapy has crashed by nearly $8 billion since 2021's peak, putting the field's translation to patients at risk.
With over 2,000 cell and gene therapies in clinical trials globally, the industry’s reliance on manual, labour-intensive manufacturing is an existential bottleneck.
With zero FDA-approved personalised iPSC therapies on the market, the vast majority of patients who could benefit from regenerative treatments still can’t access them.
