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News Article | July 27, 2017
Site: www.prnewswire.com

Proceeds of the Series A financing will be used to develop novel molecules and to advance PreciThera's lead candidate through IND and into clinical studies with the goal to establish clinical proof of concept and identify a path to registration. PreciThera is focusing on orphan bone diseases that cannot be treated via traditional protein replacement therapies. The company utilizes a combination of informatics-based tools to combine information from RNA sequencing and from genomic and clinical databases to validate the major pathway responsible for clinical symptoms in rare genetic disorders. Its precision medicine strategy is particularly well-suited to address genetic diseases that, until now, were classified based on their clinical manifestations and treated with non-specific agents. By leveraging both a novel understanding of the biology of these disorders and its precision medicine strategy, PreciThera aims to achieve truly disease-modifying outcomes for patients. Industry veterans and scientific experts Dr. Philippe Crine and Dr. Susan Schiavi founded Montreal, Canada-based PreciThera in 2016. Prior to PreciThera, Dr. Crine founded Enobia Pharma, which advanced its lead product, asfotase alfa, into clinical development for treatment of the orphan bone disease hypophosphatasia. Enobia Pharma was acquired for $1.1B in 2012 by Alexion, which now markets the product as Strensiq. Dr. Schiavi brings bone and metabolic disease expertise from her many years as director of Genzyme's Bone and Mineral Metabolism team in support of the company's blockbuster drug, Renvela. Dr. Philippe Crine, co-founder of PreciThera, said: "We are grateful for the support of these premier healthcare investors who believe in our vision of novel therapeutic strategies for rare diseases and the potential of precision medicine for speeding up and de-risking drug development in this rapidly expanding area of the pharmaceutical industry. Our mission is to provide highly innovative and life changing therapies to patients with heterogeneous genetic diseases affecting the skeleton who had up to now no or inadequate therapeutic options." Pierre Beauparlant, chief executive officer of PreciThera, commented: "This fundraising validates the potential that investors see in PreciThera's approach. The decision to support the company's efforts was based on strong scientific research, combined with the great market potential for the company's drug candidates." Peter McWilliams, Ph.D., managing director of Sanderling Ventures, said: "We are pleased to be part of this oversubscribed Series A financing combining a strong syndicate of Quebec based and US based investors to launch PreciThera, another important new venture enhancing the vibrant life science innovation ecosystem in Quebec." About Sanderling Ventures Sanderling Ventures is one of the oldest investment firms dedicated to building new biomedical companies. Sanderling emphasizes early-stage financing and active management of its portfolio companies. Its principals play an active role in new ventures by providing seed and early-stage funding, contributing management leadership and administrative support, developing cost-control strategies to extend available dollars, supplying technical and regulatory expertise where needed, and offering the insight and perspective of those who have "done it before." For further information, please visit www.sanderling.com About Arix Bioscience plc Arix Bioscience plc is a global healthcare and life science company supporting medical innovation. Headquartered in London and with an office in New York, Arix Bioscience sources, finances and builds world class healthcare and life science businesses addressing medical innovation at all stages of development. Operations are supported by privileged access to breakthrough academic science and strategic relationships with leading research accelerators and global pharmaceutical companies. Arix Bioscience plc is listed on the Main Market of the London Stock Exchange. For further information, please visit www.arixbioscience.com About the Fonds de solidarité FTQ Fonds de solidarité FTQ is a development capital investment fund that channels the savings of Quebecers into investments. As at May 31, 2017, the organization had $13.1 billion in net assets, and through its current portfolio of investments has helped create and protect 186,440 jobs. The Fonds is a partner in more than 2,700 companies and has 645,664 shareholder-savers. For further information, please visit http://www.fondsftq.com About CTI Life Sciences Based in Montréal, CTI Life Sciences, is a Canadian venture firm that invests in high quality emerging life sciences companies at the pre-clinical and clinical development stage in Canada and in the U.S. CTI Life Sciences has $245 million in assets under management. For further information, please visit http://www.ctisciences.com About Emerillon Capital Based in Montreal, Emerillon Capital is a venture capital fund dedicated to investing and supporting companies with strong technological expertise that are positioned in sectors with strong growth potential. It aims to accelerate commercial start-up and support their expansion. Emerillon Capital invests primarily in Canada. By leveraging the network of CIC Capital, the Canadian subsidiary of CM-CIC Investissement, its lead sponsor, Emerillon Capital offers entrepreneurs a gateway to support their development projects in Europe. For more information, visit www.emerilloncapital.com/en/ About PreciThera, Inc. PreciThera, Inc. is a biotechnology company committed to the development of therapies for rare bone diseases using the combined application of computational technology and a deep understanding of disease pathology. The company focuses on heterogeneous genetic disorders that primarily manifest in bone dysfunction. Understanding of novel biology will allow PreciThera's targeted strategies to meaningfully impact both the skeletal symptoms as well as the extraskeletal issues found in these patients. For further information, please visit www.precithera.com For more information, please contact:

Dickson G.J.,University College London | Dickson G.J.,King's College London | Bustraan S.,University College London | Hills R.K.,University of Cardiff | And 7 more authors.
British Journal of Haematology | Year: 2016

Older adult patients (≥60 years) with acute myeloid leukaemia (AML) are generally considered to be poor-risk and there is limited information available regarding risk stratification based on molecular characterization in this age group, particularly for the double-mutant CEBPA (CEBPADM) genotype. To investigate whether a molecular favourable-risk genotype can be identified, we investigated CEBPA, NPM1 and FLT3 status and prognostic impact in a cohort of 301 patients aged 60 years or more with intermediate-risk cytogenetics, all treated intensively. Overall survival (OS) at 1 year was highest in the 12 patients (4%) that were CEBPADM compared to the 76 (28%) with a mutant NPM1 and wild-type FLT3 (NPM1MUTFLT3WT) genotype or all other patients (75%, 54%, 33% respectively), with median survival 15·2, 13·6 and 6·6 months, although the benefit was short-term (OS at 3 years 17%, 29%, 12% respectively). Combination of the CEBPADM and NPM1MUTFLT3WT genotype patients defined a molecular group with favourable prognosis (P < 0·0001 in multivariate analysis), with 57% of patients alive at 1 year compared to 33% for all other patients. Knowledge of genotype in older cytogenetically intermediate-risk patients might influence therapy decisions. © 2016 John Wiley & Sons Ltd.

PubMed | Karolinska Institutet, Fred Hutchinson Cancer Research Center, University of Colorado at Denver, CTI Science and 6 more.
Type: | Journal: Nature reviews. Disease primers | Year: 2016

Acute myeloid leukaemia (AML) is a disorder characterized by a clonal proliferation derived from primitive haematopoietic stem cells or progenitor cells. Abnormal differentiation of myeloid cells results in a high level of immature malignant cells and fewer differentiated red blood cells, platelets and white blood cells. The disease occurs at all ages, but predominantly occurs in older people (>60 years of age). AML typically presents with a rapid onset of symptoms that are attributable to bone marrow failure and may be fatal within weeks or months when left untreated. The genomic landscape of AML has been determined and genetic instability is infrequent with a relatively small number of driver mutations. Mutations in genes involved in epigenetic regulation are common and are early events in leukaemogenesis. The subclassification of AML has been dependent on the morphology and cytogenetics of blood and bone marrow cells, but specific mutational analysis is now being incorporated. Improvements in treatment in younger patients over the past 35 years has largely been due to dose escalation and better supportive care. Allogeneic haematopoietic stem cell transplantation may be used to consolidate remission in those patients who are deemed to be at high risk of relapse. A plethora of new agents - including those targeted at specific biochemical pathways and immunotherapeutic approaches - are now in trial based on improved understanding of disease pathophysiology. These advances provide good grounds for optimism, although mortality remains high especially in older patients.

Clarke D.,Arkansas State University | Buchanan R.,Arkansas State University | Gupta N.,University of Kentucky | Gupta N.,CTI Science | And 2 more authors.
Toxicological and Environmental Chemistry | Year: 2012

The toxic effects of mercury are known to be complex with specific enzyme inhibitions and subsequent oxidative stress adding to the damaging effects. There are likely other factors involved, such as the development of impaired metal ion homeostasis and depletion of thiol- and selenium-based metabolites such as cysteine and selenium. Much of the toxicity of mercury occurs at the intracellular level via binding of Hg 2+ to thiol groups in specific proteins. Therefore, amelioration of mercury toxicity by the use of chelation would likely be enhanced by the use of a chelator that could cross the cell membrane and the blood brain barrier. It would be most favorable if this compound was of low toxicity, had appropriate pharmacokinetics, bound and rendered mercury cation non-toxic and had antioxidant properties. Herein we report on such a chelator, N,N′-bis(2-mercaptoethyl)isophthalamide (NBMI), and, using an animal model, show that it prevented the toxic effects associated with acute exposure induced by injected mercury chloride. © 2012 Taylor and Francis Group, LLC.

Patel R.B.,Ohio State University | Kotha S.R.,Ohio State University | Sauers L.A.,Ohio State University | Malireddy S.,Ohio State University | And 7 more authors.
Toxicology Mechanisms and Methods | Year: 2012

Lung vascular alterations and pulmonary hypertension associated with oxidative stress have been reported to be involved in idiopathic lung fibrosis (ILF). Therefore, here, we hypothesize that the widely used lung fibrosis inducer, bleomycin, would cause cytoskeletal rearrangement through thiol-redox alterations in the cultured lung vascular endothelial cell (EC) monolayers. We exposed the monolayers of primary bovine pulmonary artery ECs to bleomycin (10 μg) and studied the cytotoxicity, cytoskeletal rearrangements, and the macromolecule (fluorescein isothiocyanate-dextran, 70,000 mol. wt.) paracellular transport in the absence and presence of two thiol-redox protectants, the classic water-soluble N-acetyl-L-cysteine (NAC) and the novel hydrophobic N,N′-bis-2-mercaptoethyl isophthalamide (NBMI). Our results revealed that bleomycin induced cytotoxicity (lactate dehydrogenase leak), morphological alterations (rounding of cells and filipodia formation), and cytoskeletal rearrangement (actin stress fiber formation and alterations of tight junction proteins, ZO-1 and occludin) in a dose-dependent fashion. Furthermore, our study demonstrated the formation of reactive oxygen species, loss of thiols (glutathione, GSH), EC barrier dysfunction (decrease of transendothelial electrical resistance), and enhanced paracellular transport (leak) of macromolecules. The observed bleomycin-induced EC alterations were attenuated by both NAC and NBMI, revealing that the novel hydrophobic thiol-protectant, NBMI, was more effective at μM concentrations as compared to the water-soluble NAC that was effective at mM concentrations in offering protection against the bleomycin-induced EC alterations. Overall, the results of the current study suggested the central role of thiol-redox in vascular EC dysfunction associated with ILF. © 2012 Informa Healthcare USA, Inc.

CTI Science | Date: 2013-12-03

Pharmaceutical and veterinary preparations; sanitary preparations for medical purposes; dietetic food and substances adapted for medical or veterinary use, food for babies; dietary supplements for humans and animals; plasters, materials for dressings; material for stopping teeth, dental wax; disinfectants; preparations for destroying vermin; fungicides, herbicides.

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