Signals
2Sickle cell disease curative therapy uptake exposes the conditioning-regimen access gap
Approved curative therapies for sickle cell disease (lentiviral gene therapy and CRISPR-edited autologous stem-cell therapy) are facing real-world uptake constrained by the conditioning regimen requirements and the specialist-centre infrastructure for autologous cell therapy.
AAV gene-therapy manufacturing capacity remains the rate-limit on commercial uptake
The AAV gene-therapy class continues to face manufacturing-capacity constraints that limit commercial scale, particularly for high-dose indications. Investment in capacity is happening, but the lead times are long and the implications for commercial uptake are persisting longer than the field expected.
Snapshots
1Explained
2How natural history studies are being co-designed with sponsors
Natural history studies in rare disease are increasingly being designed jointly between patient organisations, academic networks, and sponsors with assets in development. The co-design model has implications for evidence quality, regulatory acceptability, and the commercial use of the resulting data.
Patient registries are now critical infrastructure, not nice-to-have evidence
Patient registries in rare disease have moved from research-grade data sources to operational infrastructure underpinning regulatory submissions, HTA decisions, post-marketing evidence, and patient-finding models. The framing has shifted; the funding model is still catching up.