2.6. Hereditary angioedema: Biotech innovation in action

Hereditary angioedema (HAE), arguably, represents an ideal case of the Orphan Drug rules in action. Because of the incentives on offer, a mini-cluster of firms has, over the past fifteen years, developed a well-stocked cabinet of therapies for this rare disease. 

The condition is linked to deficiencies in C1 esterase inhibitor activity and results in dysregulation of the kinin-kallikrein pathway, which is involved in regulating inflammation, blood pressure, and coagulation. The ultimate culprit is bradykinin, a potent, pro-inflammatory vasodilator. 

People living with HAE are at risk of regular acute attacks, which are characterised by fluid release from leaky blood vessels, swelling, inflammation, and pain. Various organ and tissue systems can be affected, including the larynx, which can result in asphyxiation. 

The HAE space represents the evolution of the wider biotechnology industry in microcosm – plasma-derived C1 esterase inhibitor products were followed by recombinant equivalents (including one obtained from transgenic rabbits, which is itself an important branch of biotech history). Small molecule and antibody drugs were brought to bear on different targets within the kinin-kallikrein signal cascade. These have reduced, but not eliminated episodes. There is room for further innovation – and it is coming. 

Intellia Therapeutics recently published compelling, albeit preliminary, data for its CRISPR-Cas9-based gene editing therapy, while siRNA and antisense molecules are also in clinical development. Can these gene-based medicines supplant the incumbents? Time – and more data – will tell. 

Companies mentioned in this episode: 
ADARx Pharmaceuticals, Astria Therapeutics, BioCryst Pharmaceuticals, CSL Behring, Dicerna Pharmaceuticals, Intellia Therapeutics, Ionis Pharmaceuticals, Jerini, Lev Pharmaceuticals, Pharming, Shire, Regeneron Pharmaceuticals, Takeda, Viropharma

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