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Development of new effective therapies for rare diseases

Published by European Commission (EC)

GrantRare Disease

This topic aims at supporting activities that are enabling or contributing to one or several expected impacts of destination 3 “Tackling diseases and reducing disease burden”. To that end, proposals under this topic should aim for delivering results that are directed, tailored towards and contributing to some of the following expected outcomes:

Researchers and developers make the best use of the state-of-the-art knowledge and resources for a fast and effective development of new therapies for rare diseases.
Researchers and developers increase the development success rate of therapies for rare diseases by employing robust preclinical models, methods, technologies, validated biomarkers, reliable patient reported outcomes and/or innovative clinical trials designs.
Developers and regulators move faster towards market approval of new therapies for rare diseases (with currently no approved treatment option) due to an increased number of interventions successfully tested in late stages of clinical development.
Healthcare professionals and people living with a rare disease get access to new therapeutic interventions and/or orphan medicinal products.

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CBRC (Comprehensive Biomedical Research Centre) at UCL

Published by UCL

Accelerators or IncubatorsBasic ResearchClinical ResearchIndustrial Development or SMENational (UK)Preclinical Research

CBRC (Comprehensive Biomedical Research Centre): a maximum of £50,000 per award can be applied for using a standard UCLB application form.
Applications for CBRC funded awards require approval by the CBRC Advisory Board.
HEIF (Higher Education Innovation Fund):
applications under £25,000 per award can be applied for using a standard UCLB application form and will be approved internally by UCLB.
Applications exceeding £25,000 will be submitted to the Office of the Vice Provost for Enterprise for approval.
• J&J (Johnson & Johnson) POC: Awards of up to £75,000 are available on a call by call basis.
Preliminary abstracts can be submitted to UCLB and forwarded to J&J. If the abstract is approved, the PI will be invited to submit a full application to the UCLB/J&J committee for final selection.
• MIMIT (Manchester Integrating Medicine &
Innovative Technology): Open to applications on a
case by case call only. Applications can be made to
MIMIT using a dedicated MIMIT application form.
• Moorfields Fund: The Moorfields Fund is open to
applications of up to £50,000 for projects relating to
ophthalmology. Applications over £25,000 require
external independent approval by a dedicated
selection committee.
• UCLB Fund: A UCLB controlled fund of up to
£100,000 per annum. This award is approved and
managed internally by UCLB.

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Regione Sicilia

Published by REGIONE SICILIA

Basic ResearchClinical ResearchPreclinical ResearchPublic

The OP ERDF Sicily 2014/2020 was built on the basis of an analysis of the relevant needs, problems and opportunities that characterize the Sicilian Region with the involvement of the territory through a path of public consultation. The thematic objectives and investment priorities have been identified on the basis of EU Regulations n. 1301/2013 and n. 1303/2013 and the Partnership Agreement for Italy 2014/2020.

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BRIDGE HEAD

Published by EIT

Clinical ResearchIndustrial Development or SMEPreclinical ResearchPublic

This is an internationalisation programme for start-ups interested in being hosted by one or more top-notch Accelerators or Incubators/clusters from the EIT Health European network. Start-ups travel to accelerators of their choice to speed up access to markets, tap into local networks of partners and clients, access infrastructure and get a first-hand feeling for the local regulatory and business context.

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Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases

Published by NIH

Clinical Trial

The purpose of this funding opportunity announcement (FOA) is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions. Additionally, through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare diseases and exert a broad and positive impact on rare disease drug development.

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Pilot Projects Investigating Understudied Proteins Associated with Rare Diseases

Published by NIH

Druggable ProteinPilot

The purpose of this funding opportunity announcement (FOA) is to solicit applications for pilot projects to elucidate a role for understudied proteins associated with rare diseases. Awards will support generation of preliminary data and/or tools around eligible understudied protein(s). A list of eligible proteins is provided and are members of druggable protein families that have a known association with a rare disease. This FOA is intended to jumpstart research on understudied proteins that are associated with rare diseases and provide applicants with sufficient funding to perform basic biochemical and/or biological work to further the characterization of understudied proteins associated with rare disease.

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Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases

Published by NIH

Clinical Trial ReadinessNeuromuscular

This Funding Opportunity Announcement (FOA) invites researchers to submit applications for support of clinical studies that address critical needs for clinical trial readiness in rare neurological and neuromuscular diseases. These studies should result in clinically validated biomarkers and clinical outcome assessment measures appropriate for use in upcoming clinical trials. Through the support of trial readiness studies, NINDS expects to enhance the quality and increase the likelihood of success of clinical trials in these rare diseases.

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