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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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Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access

Published by NIH

CROContract Research OrganizationContract Medical OrganizationCMO

The purpose of this FOA is to provide investigators with a mechanism to access contract research/medical organizations (CROs/CMOs) and subject matter experts (SMEs) within the NINDS Ultra-Rare Gene-based Therapy (URGenT) Network to support planning, manufacturing, and limited nonclinical therapeutic development efforts.

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Critical Path Public Private Partnerships

Published by NIH

Research Demonstration Cooperative Agreements

The FDA seeks an application to continue to maintain, manage existing consortia groups convened and established by the Critical Path Institute. This is a renewal of the cooperative agreement #5U18FD005320 as part of the Critical Path Initiative. This is in support of Critical Path Initiative introduced in Section 566 of the Federal Food, Drug, and Cosmetic Act. This includes developing innovative, collaborative projects in research, education, and outreach for fostering drug product innovation, enabling the acceleration of development, manufacturing, and translational therapeutics, enhancing safety, efficacy, quality, and performance. This funding opportunity will provide support, depending on availability of FDA funding.

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Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders

Published by NIH

Research Project Cooperative Agreements

The Ultra-Rare Gene-Based Therapy (URGenT) network supports Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) clinical testing of gene-based or transcript-directed therapeutics, such as oligonucleotides and viral-based gene therapies, for ultra-rare neurological or neuromuscular disorders. The goal of this funding opportunity announcement (FOA) is to accelerate the development of a promising clinical candidate with robust biological rationale and demonstrated proof of concept (POC) data for the intended approach in a model system relevant to a specified patient population towards an IND filing and the initiation of a clinical trial.

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Limited Competition: Small Grant Program for the NCATS Clinical and Translational Science Award

Published by NIH

Small Grant Program

The purpose of this NOFO is to enhance the capability of NCATS CTSA Program KL2/K12 scholars and recipients of diversity and re-entry supplements (Research Supplements to Promote Diversity in Health-Related Research (Admin Supp – Clinical Trial Not Allowed) (PA-23-189 and PA-21-071)) and Research Supplements to Promote Re-Entry, Re-integration into, and Re-training in Health-Related Research Careers (Admin Supp – Clinical Trial Not Allowed) (NOT-OD-23-170 and NOT-OD-21-134)) supported by the CTSA Program to conduct translational science research as they complete the transition to fully independent academic translational scientists.

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Cellular and Molecular Biology of Complex Brain Disorders

Published by NIH

Exploratory/Developmental Research Grant

This Notice of Funding Opportunity (NOFO) encourages research on the biology of high confidence risk factors associated with complex brain disorders, with a focus on the intracellular, transcellular and circuit substrates of neural function. For the purposes of this NOFO , the term “complex” can refer to a multifactorial contribution to risk (e.g., polygenic and/or environmental) and/or highly distributed functional features of the brain disorders

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NINDS Exploratory Clinical Trials for Small Business (R43/R44 Clinical Trial Required)

Published by NIH

and Fast-TrackPhase IISmall Business Innovation Research (SBIR) Grant - Phase I

The purpose of this notice of funding opportunity (NOFO) is to provide a vehicle for Small Business Concerns (SBCs) submitting Small Business Innovation Research (SBIR) grant applications for investigator-initiated exploratory clinical trials to the National Institute of Neurological Disorders and Stroke (NINDS). The projects must focus on products related to the mission and goals of the NINDS and may evaluate drugs, biologics, devices, or diagnostics, as well as surgical, behavioral or rehabilitation therapies.

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Bio-printing of Living Cells for Regenerative Medicine

Published by European Commission (EC)

Regenerative Medicine

Regenerative medicine is a branch of translational research in tissue engineering and molecular biology which deals with the “process of replacing, engineering or regenerating human cells, tissues or organs to restore or establish normal function”. 3D-printing in general is considered an advanced manufacturing technique and 3D-printing of non-viable biomaterials to serve e.g. as scaffold for cell growth or as structure for medical devices is already broadly used.

However, bio-printing technology involving living cells is still in early stages of development, but has a huge potential for tissue engineering, drug testing and other biomedical applications. Tissue-specific functional 3D bio-printing is a new approach for transplantation applications in regenerative medicine, relying on the fabrication of tissues and organs with respect to the desired shape and function and their delivery and application in vivo. “In-situ bio-printing” known as printing cells and biomaterials directly onto or in a patient, or 4D bio-printing, which introduces a “time” variable that allows 3D printed materials to change shape or function when external stimulus is applied, are recent developments facing multiple additional challenges.

Despite some success of 3D bio-printing with thin tissue, thick tissue and complex organs remain a bottleneck because it is difficult to sufficiently mimic their metabolic needs, and the scientific knowledge about their intimate architecture and interplay with other tissues are not sufficiently elucidated. Next to these limitations are a lack of standardised manufacturing protocols and standardised bio-ink formulations with tuneable properties, unstable cellular behaviour, material biocompatibility and printability, etc. Taken together, 3D bio-printing is confronted with several challenges that currently hamper its large-scale deployment.

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Developing EU Methodological Frameworks for Clinical/Performance Evaluation and Post-market Clinical/Performance Follow-up of Medical Devices and In Vitro Diagnostic Medical Devices (IVDs)

Published by European Commission (EC)

Medical DeviceClinic

This topic aims at supporting activities that are enabling or contributing to one or several expected impacts of destination 6 “Maintaining an innovative, sustainable and globally competitive health industry”.

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