San Francisco, CA, United States
San Francisco, CA, United States

ACT Biotech Inc is a San Francisco-based, privately held biopharmaceutical company focused on the development and commercialization of targeted cancer drugs.The Company's clinical stage pipeline includes :Telatinib, an oral kinase inhibitor for the first-line treatment of advanced gastric cancer. It has been granted Orphan Drug status by the U.S. Food and Drug Administration . It has reported encouraging interim results from a phase II trial.ACTB1003, an oral kinase inhibitor that targets cancer cells through multiple modes of action. It inhibits cancer cell growth by targeting the FGF receptor family, which are mutated in a number of human cancer types. ACTB1003 also directly induces apoptosis by targeting kinases downstream of the PI3K pathway, all at low nanomolar concentrations.Other pipeline products include an oral Aurora A and B kinase inhibitor at the pre-IND stage, and an ABL tyrosine kinase inhibitor targeting the T315I mutant enzyme in pre-clinical development. Wikipedia.

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Grant
Agency: European Commission | Branch: FP7 | Program: CP-IP | Phase: HEALTH-2007-1.4-4 | Award Amount: 14.73M | Year: 2009

For many disabling or fatal diseases, there is pre-clinical or clinical evidence of the potential therapeutic efficacy of gene therapy and, yet, the limitations of current gene transfer technologies have prevented success or even caused serious adverse events leading to termination of trials. PERSIST will explore the use of highly innovative gene-modifying and delivery technologies and capitalize on recent discoveries in gene expression control to develop radical solutions to the problem of precisely controlling the fate and expression of exogenous genetic information in gene therapy with applications in these and other deadly diseases. Our proposal combines 20 of Europes outstanding experts from 8 countries in the field of genetic engineering for persisting gene expression. Partners have pioneered the use of Zinc Finger Nucleases, engineered recombinases and transposases for gene targeting, synthetic promoters, epigenetic switches and micro-RNA for regulating gene expression, developed state-of-the-art gene delivery platforms based on lentiviral, AAV and gutless adenoviral vectors, conducted front-line clinical trials, and productively collaborated in previous FP projects. PERSIST includes 16 work packages of which 6 focus on vector innovations (part A: emerging technologies), 6 on applications & evaluation (part B), 1 on process development and the remaining 2 on training/dissemination and project management including IPR issues. Targeting deadly diseases, such as inherited immunodeficiencies, storage disorders and haemophilias, PERSIST is in line with the Strategic Research Agenda (SRA) of the Innovative Medicines Initiative (IMI) and will result in: faster discovery and development of better medicines; more attractive professional environment for scientists; better European expertise and know-how to attract biomedical R&D investment in Europe; and a stronger competitive advantage for SMEs, spin-offs and start-ups to enhance Europes economy.


Grant
Agency: European Commission | Branch: FP7 | Program: CP-IP | Phase: HEALTH.2013.2.1.1-1 | Award Amount: 15.78M | Year: 2013

The aim of SYBIL is to carry out extensive functional validation of the genetic determinants of rare and common skeletal diseases and the age related factors contributing to these painful conditions. To achieve this goal SYBIL will gather complementary translational and transnational scientists, systems biologists, disease modellers, leading SMEs and industrialists that will perform in-depth characterisation (complete molecular phenotyping) of pre-clinical models (cellular and animal) for a variety of common and rare skeletal diseases. SYBIL will establish a systematic pipeline of in vitro, ex vivo and in vivo models of increasing complexity and will also make use of novel technologies such as iPS cells and exclusive Virtual Patient software to identify potential therapeutic targets for further validation through simultaneous modelling of fundamental and complex physiological pathways. SYBIL will rely on i) an Omics Knowledge Factory for systematically generating new knowledge on skeletal disease pathophysiology and to generate the relevant Omics profiles necessary to detect and validate new disease determinants, biomarkers and therapeutic targets for future clinical developments, and ii) a Systems Biology Hub to integrate the high-throughput and data-dense information, to gain a global understanding of pathophysiological commonalities between different skeletal diseases and recognize predominant shared pathways and mechanisms that may represent new targeted routes to treatment. SYBIL will also identify potential modifier genes and study the epigenome that will ultimately influence the timing and efficacy of new personalised treatments. Overall SYBIL achievements will tremendously boost the efficient pre-clinical assessment and development of therapeutics against skeletal diseases and thus indirectly reduce their social and healthcare burden.


Grant
Agency: European Commission | Branch: FP7 | Program: CP-IP | Phase: HEALTH.2013.2.1.1-1 | Award Amount: 16.16M | Year: 2013

Recently intense research identified around 4,000 single nucleotide polymorphisms (SNPs) associated with human age related diseases such as metabolic disorders. Despite their highly significant association to pathology, the functional role of these genetic variants is, in most cases, yet to be elucidated. The evolutionary distance of most animal models from humans represents a major limitation for the functional validation of these SNPs. To overcome these difficulties, HUMAN will generate mouse models carrying human hepatocytes or pancreatic cells from either primary cells (hepatocytes) or induced pluripotent stem cells (iPSCs). This innovative approach offers the unique possibility of studying function of genetic risk variants associated with metabolic diseases in an integrated living system (the mouse body), but within human-derived organs, i.e. liver and pancreas. iPSCs used to generate hepatocytes and cells will derive from extreme phenotypes, i.e. patients affected by severe metabolic diseases such as type 2 diabetes (T2D) or subjects selected for exceptional healthy longevity (subjects over 105 years and offspring of nonagenarian sibships) all fully clinically and metabolically characterised and genotyped; they will be selected according to the best combination of risk and protective alleles. We will test the effect of different nutritional regimes (e.g. high fat diet, caloric restriction), to disentangle the complex molecular mechanisms and circuitry across organs (e.g. hypothalamus-liver axis) which lead to pathology. HUMAN associates a core of outstanding basic research institutions to leading European biotech SMEs, and has the capability to produce at least 500 humanised mice. HUMAN will generate iPSCs biobanks and comprehensively manage all associated information. HUMAN is uniquely situated to drive innovation towards a better knowledge of the genetic basis of human metabolic diseases, thereby contributing to healthier aging of European citizens.


Grant
Agency: European Commission | Branch: FP7 | Program: CP-FP | Phase: HEALTH.2012.2.4.4-1 | Award Amount: 7.00M | Year: 2013

Fanconi anemia (FA) is a rare inherited syndrome characterized by the early development of bone marrow failure and increasing predisposition to cancer with age. Allogeneic hematopoietic cell transplantation (alloHCT) is the only curative therapy for hematopoietic manifestations of FA, although associated with complications arising from myeloablation, graft versus host disease and increased incidence of squamous cell carcinoma. The genetic correction of autologous hematopoietic stem cells (HSC) with lentiviral vectors constitutes a recent and safe alternative for the treatment of different genetic diseases affecting mature cells from different tissues and/or committed progenitors of the hematopoietic system. One of the key features of FA that make it a unique disease for gene therapy approaches rely on the characteristic proliferation defect that is already evident in the very primitive HSCs. Thus, a marked survival advantage would be expected from corrected HSCs, potentially allowing normalization of hematopoiesis in the absence or after mild conditioning. Difficulties in the collection of sufficient numbers of HSC from FA patients and the use of sub-optimal transduction protocols with gammaretroviral vectors limited the success of FA gene therapy trials conducted 10 years ago in the USA. Our innovative approach to develop for the first time an efficient and safe gene therapy of FA is based on two recent innovations: 1) Discovery of potent HSC mobilizers, such as plerixafor, and 2) Development of a new lentiviral vector by members of this Consortium, designed as Orphan Drug by the EC in December 2010. The principal objective of this Project is, therefore, the development of a multicentric Phase I/II gene therapy trial for FA-A patients, based on the genetic correction of plerixafor\G-CSF mobilized HSCs with the novel lentiviral vector, accompanied by comprehensive and groundbreaking safety and efficacy patient monitoring studies.


Grant
Agency: European Commission | Branch: FP7 | Program: MC-ITN | Phase: FP7-PEOPLE-2011-ITN | Award Amount: 2.54M | Year: 2012

One of the most interesting questions in prehistory is that of the origins of European peoples following the introduction of settled farming life to the continent some 8,000 years ago. The interdisciplinary BEAN network will address this by combining teaching and research in three different fields: i) Anthropology and genetics, ii) computer simulation and modelling, and iii) prehistoric archaeology. Particularly, it will provide state of the art training in palaeo-genomics, mathematical modelling of prehistoric culture change and statistical demographic inference methods. The network includes one industrial and seven academic participants and five associates and will incorporate an integrated educational system that combines intensive specialized training in each subject and rotation of early stage and advanced researchers. This research has wider impact, for example within the multibillion-euro cultural heritage industry, and BEAN will include internships at three private sector partners; a biotech company, a tourist company and a publisher, as well as the German national statistics office. Thus, participants will engage with cutting edge scientific methods, will combine diverse disciplines and schools of thought and will be guaranteed contact with private companies and state organisations for further career development. Additionally, there will be a special focus on translational skills, particularly media relations and scientific writing, both of which will feed into the final BEAN-book, which will be co-authored by ESRs, ERs and PIs, and published by Springer.


Grant
Agency: European Commission | Branch: H2020 | Program: RIA | Phase: PHC-05-2014 | Award Amount: 7.99M | Year: 2015

While prevention of most female specific cancers (ovarian, breast, endometrial) has not progressed substantially in recent years, significant progress has been made with cervical cancer due to accessibility of the cell of origin (cervical smear) and availability of a test for the causal agent (human papilloma virus); together these enable identification of high risk individuals and interventions to prevent infection or halt progression to invasive cancer. Our consortium has developed an exciting opportunity to utilise clinically abundant cervical cells in tandem with a multi-omics enabled (genome, epigenome, metagenome) analysis pipeline to understand an individuals risk of developing a female specific cancer and to direct a personalised screening and prevention strategy. Cervical cells currently collected within cervical cancer screening provide an ideal window into other female specific cancers because they are (i) an excellent non-invasive source of high quality DNA, (ii) provide a readout for environmental exposure, (iii) are part of the Mllerian tract and (iv) are hormone sensitive, recording (via the epigenome) various hormonal conditions over a lifetime that trigger cancer development. The FORECEE project is aligned with the novel concept of P4 Medicine (predictive, preventive, personalized, and participatory): it aims to translate the risk prediction tools output into personalised recommendations for screening and prevention of female cancers. Our consortium comprises a multi-disciplinary team of experts in clinical oncology, risk-benefit communication, omics technologies, decision analysis, health economics and public health. We will examine the effectiveness of the proposed cervical cell omics analysis method and investigate the legal, social, ethical and behavioural issues related to implementation of the risk prediction tool, through direct interaction with stakeholder groups, to ensure its rapid translation into clinical practice across Europe.


Grant
Agency: European Commission | Branch: FP7 | Program: CP-FP | Phase: HEALTH.2012.2.1.2-1 | Award Amount: 3.93M | Year: 2012

Lung transplantation (LT) is the standard of care for selected patients with chronic respiratory failure. Chronic lung allograft dysfunction (CLAD) (i.e. Bronchiolitis obliterans syndrome (BOS) and Restrictive Allograft Syndrome (RAS)) represents a major health risk for LT recipients, requiring the use of heavy treatments and possible retransplantation. Observed in almost 50% of patients after 5 years post LT, it is currently impossible to predict the appearance of CLAD before the onset of first symptoms. This project aims to develop the SysCLAD model which will allow to predict, within the 1st year post LT, the recipients at risk of developing CLAD by 3 years post LT. Building upon available data from the cohort of lung transplantation (COLT, recruited since mid-2009), this project will integrate new LT recipients to form the European cohort of lung transplantation (ECOLT). The SysCLAD prediction tool will be based on a mathematical model developed through a system biology approach integrating both clinical and biological data collected from a total of 400 LT recipients. The model will be validated on the first 200 LT recipients (3 years follow-up at project start) and refined using the new set of 200 LT data with 3 years follow-up by 2014. The aim is to identify and validate the signature of CLAD both at the clinical and molecular levels to allow for an early recognition and specific interventions in patients at risk of CLAD. The implementation of the model is expected to significantly improve the cost-effectiveness of post-LT treatments, limit the risk of graft rejection in LT recipients and, ultimately lead to an improved quality of life and a prolonged life expectancy of patients following LT. Finally, the SysCLAD model holds further great promises in the context of other chronic bronchial inflammatory diseases of major incidence such as severe asthma and Chronic Obstructive Pulmonary Disease (COPD) to predict decline in lung function.


Grant
Agency: European Commission | Branch: FP7 | Program: CP-FP | Phase: HEALTH.2012.1.2-1 | Award Amount: 7.65M | Year: 2012

CLINICAL NEED Every year in the EU ~45,300 and 330,000 women are diagnosed with ovarian and breast cancer respectively & 28,800 and 90,000 of these two groups of women will die as a consequence of these diseases. Currently there are no tools available that allow for: a) Effective screening of ovarian and/or breast cancer of sufficient sensitivity and specificity to avoid potential over-diagnosis, or; b) Stratification of patients into optimal personalised therapy regimes in ovarian/breast cancer. EpiFemCare ADVANCES Progress in personalised cancer medicine will only be possible with the development of bioassays involving the analysis of easy accessible biomaterials that contain stable target molecules reflective of disease. We will establish and clinically validate a series of blood tests based upon DNA methylation technology that will facilitate both early detection and prediction of therapeutic outcome in breast and ovarian cancer. CONSORTIUM Our pan-European academic-industrial consortium demonstrates diverse clinical, scientific & industrial expertise. We have access to the latest state of the art technologies and, integrally, the best available cohort and clinical trial sample sets required to ensure the success of the EpiFemCare program. GATC-Biotech and Genedata are Europes leading providers of DNA sequencing and bioinformatics for biomarker development and have developed serum DNA based prenatal tests. The clinical partners have access to unique cohort and clinical samples collected from >200,000 women well in advance of disease (UK Collaborative Trial of Ovarian Cancer Screening) or before and during treatment (SUCCESS Trial). IMPACT As a result of refined and improved patient stratification, EpiFemCare will: - Reduce the late stage presentation of ovarian & breast cancer by 50% - Reduce the requirement for 50% of breast cancer patients to have adjuvant therapies - Reduce female cancer related fatalities as well as treatment-related morbidity by 20%


Grant
Agency: European Commission | Branch: FP7 | Program: CP-FP | Phase: HEALTH.2012.2.4.4-1 | Award Amount: 8.30M | Year: 2012

This project is focused on the clinical development of a new orphan drug that can rapidly become a new treatment option for patients with the X-linked form of chronic granulomatous disease (X-CGD). This rare primary immune deficiency of phagocytes is caused by mutations in the gp91phox gene. Affected patients are highly-susceptible to infections and develop inflammatory granulomas. Several members of the Net4CGD consortium have already attempted hematopoietic gene correction of X-CGD using gp91 gammaretroviral gene transfer vectors. While functional correction and clinical benefit was initially achieved, problems arose, linked to insertional mutagenesis, vector silencing and lack of long-term engraftment. Net4CGD proposes future trials to achieve (i) effective transduction of hematopoietic cells, (ii) physiological expression of the transgene and (iii) long-term engraftment of gene modified cells. A new lentiviral vector (LV) was developed to express gp91phox in myeloid cells. Encouraging results obtained in preclinical studies and through the compassionate treatment of a patient, prompt us to test the LV in a multi-center study in several European centers expert in CGD, under the sponsorship of a rare disease specialist. The tasks include i) Manufacturing clinical grade vector to support clinical studies, ii) Conducting a multi-center phase I/II trial in eligible X-CGD patients, with LV gene-modified autologous hematopoietic stem cells to evaluate the safety and efficacy of the procedure iii) Ensuring high-quality and harmonization of products and procedures to facilitate future product registration iv) Obtaining state-of-the art information on biological efficacy and safety in patients by assessing immune restoration and large-scale integrome data. If positive, this study will be used to register the orphan drug. The treatment is expected to improve patients quality of life and will reduce the economical burden of CGD. Results should benefit other RD.


Grant
Agency: European Commission | Branch: FP7 | Program: CP-IP | Phase: HEALTH.2010.1.4-1 | Award Amount: 15.80M | Year: 2010

Primary immune deficiencies (PID) are inherited disorders of the adaptive and innate immune system marked by severe infections, autoimmunity and high risk of cancer. Treatment entails hematopoietic stem cell (HSC) transplantation from allogeneic donors, however in the absence of an HLA compatible donor, HSCT outcome is limited by delayed or suboptimal reconstitution and complications. SCID-X1 and ADA-SCID have been successfully treated with autologous gene corrected HSC, however, associated with safety issues inherent to first generation retroviral vectors. This project utilizes genetically modified HSC and their descendants as immunotherapeutic cells to build a healthy immune system in PID patients, and is carried out by clinical centres, scientists and industrial partners pioneering in the field of advanced therapies and aiming at broad clinical application of safe cell-based therapeutic products. Multicentre phase I/II clinical trials for SCID-X1 and WAS are ready to start. Disease targeted technology to cure ADA-SCID, V(D)J recombination defects and CGD by gene corrected HSC and novel approaches in IPEX and HLH to gene modify already committed cells will be investigated. Based on rigorous preclinical efficacy and toxicology evaluation, flanked by basic studies aimed at improving HSC homing capacity and thymic epithelium regeneration, new clinical trials will be implemented. The consortium will establish a technology platform to implement, harmonize and run controlled, standardized multicentre preclinical studies using state-of-the-art advanced therapy. Strict observance of good practice quality guidelines and regulation of medicinal product development will be ensured. The successful completion of the project will be instrumental to accomplish and broaden clinical application of medicinal products able to rebuild and modulate the immune system with an anticipated impact that extends beyond PID to acquired immune disorders, allogeneic HSCT and cancer treatment.

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