Section 1 of 6

Terms and abbreviations

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Abbreviation	Term
ACAAI	American College of Allergy, Asthma & Immunology
AE	Adverse event
AE-QoL	Angioedema Quality of Life Questionnaire
AHFS	American Hospital Formulary Service
ALT	Alanine aminotransferase
BLA	Biologics license application
C1-INH	C1 esterase inhibitor
C1INH	C1 esterase inhibitor
CI	Confidence interval
CRISPR	Clustered regularly interspaced short palindromic repeats
EAACI	European Academy of Allergy and Clinical Immunology
ED	Emergency department
FDA	Food and Drug Administration
FFP	Fresh frozen plasma
FXII	Coagulation factor XII
FXIIa	Activated coagulation factor XII
HADS	Hospital Anxiety and Depression Scale
HAE	Hereditary angioedema
HAE-FXII	Hereditary angioedema associated with a factor XII gene variant
HAE-nC1INH	Hereditary angioedema with normal C1 inhibitor
HAE-PLG	Hereditary angioedema associated with a plasminogen gene variant
HRQoL	Health-related quality of life
IV	Intravenous
KKS	Kallikrein-kinin system
KLKB1	Kallikrein B1 gene
LNP	Lipid nanoparticle
LP01	Ionizable lipid component of NTLA-2002
LTFU	Long-term follow-up
LTP	Long-term prophylaxis
MCID	Minimal clinically important difference
mRNA	Messenger RNA
NHP	Nonhuman primate
ODT	On-demand treatment
PD	Pharmacodynamics
pdC1INH	Plasma-derived C1 inhibitor
PDUFA	Prescription Drug User Fee Act
PK	Pharmacokinetics
PRIME	Priority Medicines
PRO	Patient-reported outcome
QALY	Quality-adjusted life-year
QoL	Quality of life
RMAT	Regenerative Medicine Advanced Therapy
RNA	Ribonucleic acid
RWE	Real-world evidence
SC	Subcutaneous
SD	Standard deviation
TEAE	Treatment-emergent adverse event
WAO	World Allergy Organization
WPAI	Work Productivity and Activity Impairment questionnaire