5th Aug 2005 07:00
Ark Therapeutics Group PLC05 August 2005 Ark makes breakthrough in targeted gene therapy delivery Avoids harmful consequences of therapeutic gene being inserted in wrong place 5 August 2005: Ark Therapeutics Group plc ("Ark") announces today that itsscientists in Finland have discovered a novel gene therapy delivery technologywhich selectively inserts DNA into the specific therapeutic site in the genome(targeted integration). This technology is the subject of an Ark PCT filing. The existing generation of integrating vectors in clinical trials, based mostlyon retroviral, adeno-associated virus and lentiviral technologies, are notsite-specific and carry the risk of a random gene insertion into an undesiredand potentially harmful position on the chromosomes. The risks associated withnon-specific integrating vectors became apparent in the X-linked severe combinedimmune deficiency disease (X-SCID) gene therapy trial, in 2002. The trial useda retrovirus, which was found to have inserted next to the leukaemia inducingoncogene, as a vector. Although the treatment was beneficial, three out ofeleven patients treated in the study developed a leukaemia-like disease as aresult of undesired random insertion. Ark's novel technology could herald abreakthrough in molecular medicine because it removes the potentially harmfulconsequences of a beneficial therapeutic gene being inserted into the wrongplace, and could thus greatly improve the predictability and safety of genetherapy. The technology is based on a fusion protein that catalyses the targetedintegration of the treatment gene into a benign site on the human chromosomes.The process whereby the DNA carrying vector inserts the treatment gene in thechromosome is achieved by a specific DNA-binding nuclease-integrase fusionprotein. The fusion protein breaks the DNA chain specifically in the benignlocus of 28s ribosomal RNA gene (28s rDNA) and inserts the therapeutic gene atthis site. Controlling the site where the insertion occurs has been so far verydifficult, even with hybrid vectors or designed zinc finger proteins. Ark'snovel vector technology removes the risk of such potentially harmful randominsertions by this molecular chromosome locus targeting approach. Commenting on this discovery, Professor Seppo Yla-Herttuala, ConsultantDirector of Molecular Medicine in Finland, said: "This is potentially a highlysignificant step forward in gene therapy, enabling us to deliver therapeutic DNAprecisely in a way that it will provide the medical benefit. Gene therapy hasalways offered the possibility of great therapeutic benefit but has beenhampered by the difficulty of inserting the beneficial gene in the right place.Whilst we have further work to do, the ability potentially to overcome thisproblem is a vital breakthrough, improving the overall efficiency and safety inan area of medicine that we expect to become increasingly important in the nearfuture." For further information, please contact: Ark Therapeutics Group plc 020 7388 7722Dr Nigel Parker, CEOProfessor Seppo Yla-Herttuala, Cons. Director of Molecular Medicine 00 358 17 162075 Financial Dynamics 020 7831 3113David Yates / Lucy Briggs Notes to editors Ark Therapeutics Group plc Ark is a specialist healthcare group (the "Group"), addressing high value areasof clear unmet medical need. With one marketed product, Kerraboot(R), and threefurther lead products in late stage clinical development: Vitor(TM), Cerepro(TM) and Trinam(R), the Group is transitioning from an R&D focused company to acommercial, revenue generating business. Capitalising on over ten years ofresearch in vascular biology and gene-based medicine, Ark has a broad productportfolio targeted at specific unmet clinical needs within vascular disease,wound care and cancer. These are large and growing markets, where opportunitiesexist for effective new products to generate significant revenues. Cerepro(TM) is on track to becoming one of the world's first commercially available gene-based medicines. Ark's products are sourced from related but largely non-dependent technologieswithin the Group and have been selected to enable them to be taken throughdevelopment within the Company's own means and to benefit from Orphan DrugStatus and/or Fast Track Designation, as appropriate. This strategy has allowedthe Group to retain greater value and greater control of clinical developmenttimelines, and to mitigate the risks of dependency on any one particularprogramme or development partner. Ark has secured patents or has patentapplications pending for all its lead products in principal pharmaceuticalmarkets. This announcement includes "forward-looking statements" which include allstatements other than statements of historical facts, including, withoutlimitation, those regarding the Group's financial position, business strategy,plans and objectives of management for future operations (including developmentplans and objectives relating to the Group's products and services), and anystatements preceded by, followed by or that include forward-looking terminologysuch as the words "targets", "believes", "estimates", "expects", "aims","intends", "will", "can", "may", "anticipates", "would", "should", "could" orsimilar expressions or the negative thereof. Such forward-looking statementsinvolve known and unknown risks, uncertainties and other important factorsbeyond the Group's control that could cause the actual results, performance orachievements of the Group to be materially different from future results,performance or achievements expressed or implied by such forward-lookingstatements. Such forward-looking statements are based on numerous assumptionsregarding the Group's present and future business strategies and the environmentin which the Group will operate in the future. Among the important factors thatcould cause the Group's actual results, performance or achievements to differmaterially from those in forward-looking statements include those relating toArk's funding requirements, regulatory approvals, clinical trials, reliance onthird parties, intellectual property, key personnel and other factors. Theseforward-looking statements speak only as at the date of this announcement. TheGroup expressly disclaims any obligation or undertaking to disseminate anyupdates or revisions to any forward-looking statements contained in thisannouncement to reflect any change in the Group's expectations with regardthereto or any change in events, conditions or circumstances on which any suchstatements are based. As a result of these factors, prospective investors arecautioned not to rely on any forward-looking statement. This information is provided by RNS The company news service from the London Stock ExchangeRelated Shares:
PVG.L