Signals
4Phenylketonuria therapy options mature past low-phenylalanine diet
Pegvaliase commercial maturity, sapropterin use, and emerging gene therapy programs are restructuring phenylketonuria management.
Hereditary angioedema oral options reach maturity
Oral plasma kallikrein inhibitors and emerging factor XIIa inhibitor programs are restructuring HAE prophylaxis and on-demand therapy.
Cell therapy moves into non-malignant rare disease
Cell-therapy approaches are beginning to land in non-malignant rare-disease indications including severe lupus, scleroderma, and inherited metabolic conditions.
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
2Hereditary angioedema therapy reference (2026)
Reference snapshot of HAE prophylaxis and on-demand therapy options.
Antisense oligonucleotide platform landscape (2026 reference)
Reference snapshot of approved ASO therapies, late-stage programs, and the delivery and chemistry platforms behind them.