Rare and 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

Our clinical team has over

120

years of combined clinical trial experience

Access up to

43.5%

Government R&D tax incentive (even for foreign companies)

Fast start-up

8-12

weeks on average, without sacrificing quality

How iNGENū CRO Addresses Key Pain Points Associated with Rare and Orphan Disease Clinical Trials 

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.

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.

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.

We promote initiatives to raise awareness among healthcare professionals and implement educational programs for early recognition of rare diseases.

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.

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.

Rare and Orphan Disease Clinical Trial Resources

iNGENū CRO - ALS-1

Amyotropic Lateral Sclerosis (ALS)

Exploring Diagnostic Criteria, Drug Development, and Clinical Trial Challenges.

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Glioblastoma

Navigating the Complexities of Glioblastoma Clinical Trials: From Diagnosis to Enhanced Protocols.

The iNGENū CRO Difference

Expertise
Expertise from the Start

At iNGENū CRO, you engage directly with experienced specialists from the outset. Our team, comprised of highly qualified doctors and PhD scientists, provides immediate, accurate answers to your complex inquiries, ensuring efficiency and clear communication.

Drug Dev Plans
Highly Customized Drug Development Plans

iNGENū CRO stands out for our ability to create highly customized drug development plans. Our experienced team leverages their extensive background in standard and non-standard pharmaceutical practices to develop innovative and unique solutions tailored to your specific drug.

Service Offering
Full Service CRO

We provide a full range of services from preclinical research through Phase 1, 2, and 3 clinical trials. Unlike other CROs that may outsource certain stages or recommend third-party vendors, iNGENū CRO handles everything in-house.

"We have been using iNGENū for a Phase 1, first-in-human trial of our investigational drug. We have used iNGENū for: Medical writing, project management, clinical data management and full end-to-end, clinical trial execution and monitoring.

We have been very happy with all aspects of the management of the clinical trial and have been particularly impressed by the out-of-the-box creative solutions to speed up and reduce overheads for our company during this clinical trial."

Giles Moss
Chief Executive Officer

iNGENu testimonial-logos-monotone-1

Ready to discuss your Rare or Orphan Disease Clinical Trial?