AviadoBio is running three CNS gene therapy programs and recently announced a capsid partnership with Apertura to take its silencing platform from rare disease into Alzheimer's.
In cell and gene therapy, the science is rarely the bottleneck – making it at scale is. Inside the Bayer–Charité incubator betting on Berlin to fix that.
Heart disease kills more people than every cancer combined. Inside Tenaya's bet that the precision-medicine playbook oncology pioneered will work on the heart, too.
More and more cell types cannot be frozen anymore. If you think about organoids, mixed cultures, artificial tissues – all of those require warm chain logistics.
In a funding-constrained biotech market, most early-stage teams still try to reinvent the AAV and advanced-therapy CMC playbooks the industry has already solved a dozen times over.
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.
