Genable Technologies Limited

A development stage gene therapy company, built on a technology platform that provides a unique solution for dominant disorders

Strong financial backing from leading US & European venture capital and patient groups with an experienced team comprising leading scientists, industry professionals, patient organisations and investors

A lead product with orphan and ATMP designation for an inherited form of retinitis pigmentosa an ophthalmic rare disease

Scientific provenance and development is backed by growing alliances with key companies and institutions including, Spark Therapeutics, NIH, Trinity College Dublin, Benitec

A first in class therapeutic for Rho-adRP with a novel validated mode of action

Our Technology

    This video was developed by ICON Firecrest
    Please view this video animation to learn more about our orphan drug development candidate RhoNovaTM for the treatment of rhodopsin linked autosomal dominant retinitis pigmentosa (Rho-adRP).

    Please view this video animation to learn more about the inherited retinal degenerative condition rhodopsin linked autosomal dominant retinitis pigmentosa (Rho-adRP) and the progress of our gene therapy drug development candidate RhoNovaTM.
    Genable is developing gene-based therapies for the treatment of dominantly inherited genetic disorders. This class of incurable disorders comprises hundreds of individual members including different forms of autosomal dominant retinitis pigmentosa (adRP).

    Dominant inheritance means that on average 50% of the children of an affected person will be affected by the disorder. Another common feature of these disorders is that the causative genes are often characterised by mutational heterogeneity.

    Therefore typically a large number of individual mutations in a single gene can lead to a particular disorder. For example any one of approximately 200 different mutations in the rhodopsin gene (RHO) can lead to adRP. Developing individual therapies for so many RHO mutations is practically impossible.

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    News & Events

      • News

        • We are delighted to learn that our video animation on RhoNova has won a Digital Health Award

          Our video animation on the development and mode of action of RhoNova™ an ophthalmic gene therapy being investigated for the treatment of patients with a dominant form  of retinintis pigmentosa has won an award. ICON Firecrest received a merit award from Digital Health Awards for “RhoNova™: A 3D Journey,” an animation developed and created in collaboration with Genable.   Watch it […]

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      GENABLE TECHNOLOGIES LTD team event listings for 2014

      From Amsterdam to Washington, see where the Genable team have been speaking and attending this year or contact them if you want Genable at your event

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      Our Company

      Genable Technologies Ltd. is a privately held Irish bio-pharmaceutical company developing new gene medicines to treat autosomal dominant genetic diseases based on the pioneering work of Professor Jane Farrar, Dr Paul Kenna & Professor Peter Humphries. Genable has received significant financial backing to date from Fountain Healthcare Partners, Delta Partners, Fighting Blindness Ireland and Foundation Fighting Blindness Clinical Research Institute.

      Genable’s approach is innovative and unique, it utilises well-established, clinically safe & effective AAV vectors to obtain expression of RNA interference (RNAi) molecules which suppress the expression of faulty genes and replaces this a gene that is refractory to suppression but still encodes a normal wild type protein.

      The combination of suppression and replacement overcomes the significant hurdle in dominant disease of mutation variability by eliminating the need to target specific mutations and is applicable to a wide range of disorders. Genable’s technology is protected by a broad suite of granted patents and patent applications in the USA, EU and worldwide

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