Rare and Orphan Diseases

Expertise in

Rare and Orphan Disease Investment

At Clendon Biotech Capital (CBC), our commitment extends beyond mere financial investment. We specialize in nurturing innovations within the rare and orphan disease sectors, driven by a deep understanding of the unique challenges and opportunities these conditions present. Our investment philosophy is built on a foundation of support not just for groundbreaking research and development, but also for the communities most intimately connected with rare diseases.

Our Support Framework Includes:

  • Not-for-Profit Organizations: We partner with not-for-profits dedicated to rare disease research, providing them with the necessary resources to advance their mission.
  • Patient Advocacy Groups: Recognizing the power of community, we back patient advocacy groups that are pioneering self-funded efforts to combat rare and orphan diseases.
  • Individual Families: We extend financial support to families undertaking the monumental task of partially self-funding clinical trials, offering hope and practical help in equal measure.
  • Private and Public Companies: Our investment portfolio includes both private and public entities committed to targeting rare and orphan studies, ensuring that promising research receives the backing it needs to thrive.

Leveraging the FDA Orphan Drug Pathway

Our strategic approach harnesses the FDA Orphan Drug Designation, which is reserved for diseases affecting fewer than 200,000 individuals in the USA. This pathway not only provides data exclusivity but also opens up significant tax credits for CBC, enhancing our investment strategy’s efficiency and impact.

Areas of Funding

We are dedicated to advancing research and development across various stages, including:

  • Clinical Trials: From early phase studies to advanced clinical evaluations, we fund trials that push the boundaries of what’s possible in treatment and care.

  • Regulatory Advisory:  Navigating the complex regulatory environment is crucial, and we provide expert guidance to ensure compliance and strategic advantage.

  • Preclinical Studies: Our funding extends to vital nonclinical studies, including toxicology and genotoxicology assessments, supporting the foundational science behind new therapies.

  • Bioanalytical Services: We invest in cutting-edge bioanalytical services, crucial for understanding the pharmacokinetics and dynamics of potential treatments.

Focus Areas

Our interest is particularly piqued by innovations targeting:

  • Rare Neurological Syndromes
  • Inborn Errors of Metabolism
  • Rare Gastrointestinal Syndromes
  • Pediatric Syndromes
  • Cardiorespiratory Syndromes

At CBC, we believe in the power of partnership and innovation to transform the landscape of rare and orphan disease treatment. Our investment strategy is designed to accelerate the development of life-changing therapies, ensuring that those affected by rare diseases have access to the hope and help they deserve.