Rare Partners
Non-profit organization focused on translational development of therapies and diagnostics for rare diseases. Activities include preclinical research support, clinical trial protocol development and coordination, regulatory strategy (including orphan drug designation), intellectual property management, partnership facilitation with academic and industry groups, and fundraising to support projects in cystic fibrosis, hemoglobinopathies and inherited retinal disorders.
Industries
Nr. of Employees
small (1-50)
Rare Partners
Tamworth, New South Wales, Australia, Oceania
Products
Sirolimus development programs (beta-thalassemia, sickle cell disease)
Repurposing and clinical development of sirolimus to induce fetal haemoglobin in beta-thalassemia and sickle cell disease; includes completed and ongoing clinical trials.
4,6,4'-trimethylangelicin (TMA) program for cystic fibrosis
Preclinical development and IP covering TMA as a CFTR modulator and anti‑inflammatory agent for cystic fibrosis; orphan designation obtained in EU.
Inhaled/nebulized teicoplanin formulation for MRSA lung infections in cystic fibrosis
Collaborative development of an inhalable teicoplanin formulation to treat MRSA lung infections in cystic fibrosis patients; received orphan drug designations in EU and US.
Usher Syndrome program (TUDCA clinical pilot, reference center and artificial retina support)
Portfolio including patient registry, pilot clinical study of TUDCA for retinal degeneration, support to creation of a reference clinical center and funding/support for artificial retina research.
Sirolimus development programs (beta-thalassemia, sickle cell disease)
Repurposing and clinical development of sirolimus to induce fetal haemoglobin in beta-thalassemia and sickle cell disease; includes completed and ongoing clinical trials.
4,6,4'-trimethylangelicin (TMA) program for cystic fibrosis
Preclinical development and IP covering TMA as a CFTR modulator and anti‑inflammatory agent for cystic fibrosis; orphan designation obtained in EU.
Inhaled/nebulized teicoplanin formulation for MRSA lung infections in cystic fibrosis
Collaborative development of an inhalable teicoplanin formulation to treat MRSA lung infections in cystic fibrosis patients; received orphan drug designations in EU and US.
Usher Syndrome program (TUDCA clinical pilot, reference center and artificial retina support)
Portfolio including patient registry, pilot clinical study of TUDCA for retinal degeneration, support to creation of a reference clinical center and funding/support for artificial retina research.
Services
Support in preparing and managing orphan drug designation requests and interactions with regulatory agencies (EMA, FDA).
Protocol writing and operational coordination for pilot and phase II studies in rare diseases.
Oversight and funding coordination for preclinical studies and mechanistic assays to support clinical translation.
Fundraising campaign organization and management of charity partnerships and events to raise funds for projects.
Support in preparing and managing orphan drug designation requests and interactions with regulatory agencies (EMA, FDA).
Protocol writing and operational coordination for pilot and phase II studies in rare diseases.
Oversight and funding coordination for preclinical studies and mechanistic assays to support clinical translation.
Fundraising campaign organization and management of charity partnerships and events to raise funds for projects.
Expertise Areas
- Translational research for rare diseases
- Clinical trial design and site coordination
- Regulatory strategy for orphan designations (EMA, FDA)
- Drug repurposing and repositioning
Key Technologies
- Gene-therapy approaches
- Light-sensitive polymer-based artificial retinas
- Patient registries and clinical data management
- Preclinical disease models and pharmacology assays
News & Updates
Award from the Wellcome Trust to fund clinical development of sirolimus in beta-thalassemia (Innovator Award following earlier Pathfinder Award).
Initiation of patient enrolment for a phase 2 clinical trial in transfusion-dependent beta-thalassemia (Sirthalaclin).
European Commission granted orphan medicinal product designation to TMA for treatment of cystic fibrosis.
United States Patent covering use of TMA as a CFTR corrector issued (US Patent No. 9283206).
European Commission and US FDA granted orphan drug designations for sirolimus in beta-thalassemia and for sirolimus in sickle cell disease.
Orphan designations obtained for inhalable teicoplanin formulation for treatment of MRSA lung infections in cystic fibrosis (EMA 2017; FDA 2018).
Award from the Wellcome Trust to fund clinical development of sirolimus in beta-thalassemia (Innovator Award following earlier Pathfinder Award).
Initiation of patient enrolment for a phase 2 clinical trial in transfusion-dependent beta-thalassemia (Sirthalaclin).
European Commission granted orphan medicinal product designation to TMA for treatment of cystic fibrosis.
United States Patent covering use of TMA as a CFTR corrector issued (US Patent No. 9283206).
European Commission and US FDA granted orphan drug designations for sirolimus in beta-thalassemia and for sirolimus in sickle cell disease.
Orphan designations obtained for inhalable teicoplanin formulation for treatment of MRSA lung infections in cystic fibrosis (EMA 2017; FDA 2018).