Our immediate focus with our lead asset CTx001 is the treatment of Geographic Atrophy (a form of late dry age-related macular degeneration), where no licensed treatment is currently available

Our immediate focus with our lead asset CTx001 is the treatment of Geographic Atrophy (a form of late dry age-related macular degeneration), where no licensed treatment is currently available

Age-related macular degeneration (AMD) is a chronic and progressive degenerative disease of the macula, the central part of the retina, which culminates in blindness and affects mainly the elderly population. AMD pathogenesis and pathophysiology are complex due to the structural and cellular complexity of the retina and subretinal tissues, and the variety of risk factors and molecular mechanisms that contribute to disease onset and progression.


AMD develops with age and there are 2 types, termed dry and wet. Dry AMD has 3 stages: early, intermediate, and late. It usually progresses slowly over several years. There is no treatment for late dry AMD, often called Geographic Atrophy.

Wet AMD (also called neovascular AMD) is other late-stage form of AMD and this usually causes more rapid visual loss. Wet AMD can develop at any stage of dry AMD and is the result of pathological blood vessels growing in the macula. Effective treatment options are available for wet AMD.

AMD is driven by a combination of genetic predisposition, natural ageing changes and lifestyle factors, such as smoking or nutritional intake. The mechanism by which these risk factors interact and converge towards AMD are not fully understood and therefore drug discovery is challenging particularly in the management of dry AMD, where no therapeutic attempt has been successful thus far.

Genetic and molecular studies have identified the complement system as a key driver of AMD onset and progression, and there is increasing evidence that complement inhibition can slow the progression of geographic atrophy.

AMD and the role of Complement

Age-related macular degeneration is estimated to affect 196 million people globally with approximately 35% of these individual developing geographic atrophy for which there is currently no approved treatment. 

The role of complement in the pathogenesis of AMD has been the subject of extensive research with a dysregulation of a number of specific components of the cascade implicated, including C3, C5, factor H and factor I amongst others.

Through an extensive programme of original research CTx has gained powerful new insights into the role of complement in the eye and how it is dysregulated in age-related macular degeneration. 

In particular, research conducted by the founders of CTx identified the pivotal role of factor H related proteins (FHRs) in the development of AMD providing the foundation for the therapeutic strategy being pursued by CTx with its lead asset CTx001. 

As the role of FHR proteins is better understood across complement mediated conditions, the potential utility of the company’s therapeutic assets expands to encompass a number of these diseases such as renal and neurological conditions. 

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