Sygnature Discovery > Orphan Disease

Orphan Disease

Making a difference to patients is one of Sygnature Discovery’s core values. Our experienced scientists understand the challenges of working in the field of rare and orphan diseases, using our knowledge of disease mechanisms to add value to your research projects.

Our co-located  Chemistry, DMPK, Computational Chemistry and Bioscience teams work flexibly with our clients to design and implement tailored and informed strategies to advance your discovery project, enabling our clients to develop and commercialise novel therapeutics for rare, debilitating, and potentially fatal diseases.

Using our Integrated approach, we offer:

  • A collaborative work ethic
  • Intellectual input into compound design, project screening strategy and progression pathways
  • Design of complex in vitro and ex vivo Screening Cascades
  • Provision of data to support client patent applications
  • Clinical Biomarker Identification and validation
  • In vivo support via our established network of strategic partners

Sygnature Discovery has a proven track record in successfully supporting a number of challenging orphan disease projects, such as rare metabolic disorders (Corcept Case Study) and Lysosomal Storage Disease (LSD). By applying our extensive drug discovery knowledge and expertise, we have enabled the delivery of two orphan indication clinical candidates since 2011, one of which is currently in Phase II clinical development.

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Diabetes

Diabetes is a metabolic disorder that is an increasingly prevalent disease worldwide. We have established assays to support the investigation of diabetic therapies including relevant functional assays. We also have access to tissue and blood from in vivo diabetic models through strategic collaborations.

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Orphan Disease

Sygnature’s team of scientists have a strong track record of adding value to client projects in the field of rare and orphan diseases.  Sygnature’s advanced intellectual input and established relationships with providers of diseased tissue allows rapid progression of projects in this challenging therapeutic area.

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Anti-Infectives

Sygnature have a history of enabling discovery success in the field of anti-infectives. Our Medicinal Chemistry and Computational Chemistry teams have worked in close collaboration with many clients to design potent and efficacious compounds, resulting in the delivery of two preclinical candidates. Our Bioscience team can provide cytopathic effect and viability screening assays to assess compounds designed to inhibit virus attachment, entry or replication in human host cells.

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Neuroscience

Our scientists have a well-developed understanding of the challenges associated with the design and optimisation of compounds that target the central nervous system (CNS). Our experience is both broad, reaching across a number of neurodegenerative diseases (such as Alzheimer’s, Parkinson’s and Amyotrophic Lateral Sclerosis) and neuropathic pain, and deep-rooted, with members of our senior team having 10+ years in CNS drug research.

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Respiratory

We have an excellent track record of success in the field of respiratory diseases using our extensive in-house expertise, accelerating 5 compounds to the clinic and a further 4 into preclinical development for our clients. Our collaborative approach to drug discovery and our established network of KOLs in the field have been invaluable to the progression of our clients’ projects

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Inflammation and Immunology

Our scientists have a rich heritage in this therapeutic area and provide significant experience in developing in vitro assays utilising Peripheral Blood Mononuclear Cells (PBMCs), T lymphocytes (e.g. CD4+/CD8+), leukocytes, and phagocytes, amongst others, that assess various inflammation and immunological biomarkers, processes, and endpoints.

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Immuno-oncology

Modulation of the immune system presents an exciting new approach for the therapeutic perturbation of cancer cell growth. Sygnature have developed a comprehensive platform to study the processes involved in immuno-oncology, including the assessment of cytokines, intracellular signalling, T-cell proliferation assays and antibody-dependent cell cytotoxicity (ADCC) assays.

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Oncology

We have successfully delivered more than 16 pre-clinical oncology projects, utilising our Integrated Drug Discovery expertise to develop small molecule drug candidates to treat a variety of cancers such as leukaemia, breast and prostate cancer. From initial screening and profiling through to clinically relevant Biomarkers, our DMPK and bioscience group can provide full support for your discovery projects.

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