Hereditary Angioedema
Clinical research includes hereditary angioedema, with studies focused on hereditary angioedema due to C1 esterase inhibitor deficiency and related attack prevention. The programme also includes long-term follow-up in treated participants and evaluation of disease control in adults with recurrent swelling episodes.
- HAE attacks
- C1 esterase inhibitor deficiency
- Long-term safety
Research activity in this area is centred on reducing disease burden in patients with recurrent angioedema episodes.
Transthyretin Amyloidosis
The sponsor supports trials in transthyretin amyloidosis, including both hereditary transthyretin amyloidosis with polyneuropathy and transthyretin amyloidosis-related cardiomyopathy. These studies address neurological and cardiac manifestations of the disease, with follow-up in previously treated participants.
- ATTRv-PN
- ATTR-CM
- Cardiac muscle disease
Work in this field reflects a strong focus on inherited amyloid disorders affecting the peripheral nerves and the heart.
Alpha-1 Antitrypsin Deficiency
Clinical investigation also extends to alpha-1 antitrypsin deficiency-associated lung disease, including pulmonary emphysema. The research programme examines lung-related disease in adults with inherited deficiency states and associated respiratory impairment.
- AATD-associated lung disease
- Pulmonary emphysema
- Adult respiratory disease
This area adds a pulmonary dimension to the sponsor’s portfolio of inherited disease research.
Genetic Medicine
The overall portfolio is concentrated in genetic medicine and rare disease research, with activity across inherited disorders that affect the immune, neurological, cardiac, and respiratory systems. The sponsor’s clinical work spans multiple countries and research sites, supporting studies in severe monogenic conditions.
- Rare inherited disorders
- Neurological disease
- Cardiovascular disease
Medical affairs activity is directed toward clinical development in genetically defined patient populations.
