Novel Therapeutic Approaches of Rare Genetic and Haematological Diseases

Takeda Pharmaceutical are launching an open innovation initiative inviting researchers to submit their proposals addressing novel therapeutic approaches related to rare diseases, specifically rare genetic and rare haematological diseases. One‑year projects will be funded up to JPY 10,000,000 or equivalent amount, including indirect costs.

Projects should address the following area:

REF#5 Novel therapeutic approaches for lysosomal storage disorders, inborn errors of metabolism (liver metabolic and neurometabolic) and rare non-malignant haematological diseases, especially sickle-cell disease and haemophilia.

The following topics will be prioritised:

• Next-generation gene therapy/gene editing technologies, prioritising in vivo approaches
• Non-viral gene therapy delivery technologies that allow re-dosing, addressing current challenges such as nuclear translocation of transgenes
• Targeting technologies for CNS and liver for use in viral and non-viral gene therapies 
• Small molecules with potential for transformative treatment, as well as novel targets for small-molecule intervention in above disease areas

Jurisdictions of Interest

Only submissions from Australia, New Zealand, Singapore, South Korea and Taiwan will be eligible for this campaign.

Submission Information

To view more details on each area of interest seeking proposals for funding, download a proposal template here. For terms and conditions, click here. To submit your proposal, please visit our website at discover.in-part.com, register, and submit your proposal under the appropriate Discover campaign.