Rare & Orphan Diseases

We leverage our experience working closely with patients, advocacy groups, and key opinion leaders to successfully recruit, retain and empower patients.

In the realm of rare diseases, traditional large-scale study approaches can fall short, necessitating a blend of deep scientific expertise and innovative thinking. Our team excels in designing unique programs that maximize the impact of even the smallest patient cohorts.

How we work with rare and orphan disease drug developers:
  • Pre-IND and IND FDA Regulatory work
  • Development of Bioanalytical Assays and Identifying Biomarkers
  • FDA/EMA Orphan Drug Designation applications
  • Liaising with patient support groups all over the world
  • Identifying countries with the highest prevalence of the rare diseases
  • Liaising with orphan disease funding bodies
Common areas in which we have expertise include:
  • Huntington's Disease
  • Usher Syndrome
  • Ehlers-Danlos Syndrome (EDS)
  • Sickle Cell
  • Cystic Fibrosis
  • Duchenne Muscular Dystrophy (DMD)
  • Haemophilia
  • Gaucher Disease
  • Cutaneous T-Cell Lymphoma
  • Glioblastoma Multiforme

Understanding the complexities of clinical research in rare and orphan diseases

  • Rare and orphan disease research presents distinctive challenges. Patient recruitment is a critical aspect, and iNGENū leverages our experience working closely with patients, families, advocacy groups, and key opinion leaders to successfully recruit and retain vulnerable patient populations.
  • We understand the importance of identifying the right regulatory strategy from the outset, offering support in developing optimal strategies, applying for orphan drug designations, and facilitating engagements with the FDA and EMA.
  • Developing innovative rare disease therapies, particularly those involving cutting-edge products like cellular and gene therapies or uncommon administrative routes is often complex. Whether you are an emerging biotech or a global pharmaceutical company, iNGENū is committed to guiding you through the complexities of rare disease research, ensuring the success of your groundbreaking initiatives.

How iNGENū addresses key pain points in rare and orphan disease clinical research

  • Genetic and Molecular Understanding: iNGENū leverages advanced genomics and molecular analysis techniques to uncover the genetic and molecular mechanisms underlying rare diseases. Our team collaborates with leading researchers, employs cutting-edge technologies, and participates in international research networks to accelerate discoveries in this field.
  • Drug Development and Therapeutics: We are committed to facilitating collaboration between academia, industry, and patient advocacy groups. Through our integrated approach, we streamline the drug development process, providing regulatory expertise and leveraging incentives for orphan drug designation to expedite the development of novel therapies for rare diseases.
  • Patient Registries and Natural History Studies: iNGENū supports the establishment and maintenance of patient registries and natural history studies. Our data management and analytics capabilities enhance the quality and accessibility of these resources, contributing to a deeper understanding of rare diseases and facilitating more effective research strategies.
  • Access to Diagnosis and Treatment: We promote initiatives to raise awareness among healthcare professionals and implement educational programs for early recognition of rare diseases.
  • Patient Empowerment and Advocacy: Through partnerships with patient advocacy groups, iNGENū empowers patients and their families by providing resources, support, and guidance to navigate complex healthcare systems, ensuring their voices are integral to the research process.
Ready to discuss your trial?