grant-funding

Sanfilippo 2018 Grant Competition Now!

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The Sanfilippo Children’s Foundation’s 2018 grant-funding round will open on March 1, 2018 for accepting “Expression of Interest” applications. The 2018 grant competition information will be available beginning March 1, 2018. They are accepting Expressions of Interest until April 3, 2018.

The Sanfilippo Children’s Foundation, located in Australia, funds Sanfilippo syndrome medical research internationally. They plan to invest between AUD$500,000 – $700,000 in their grant-funding program annually across four grant types:

• Translational research grants

• Incubator grants

• Scholarships and Fellowships

• Travel grants

The Sanfilippo Children’s Foundation is particularly interested in three focus areas:

• halt disease progression through therapies such as enzyme replacement, gene therapy and cell therapy, and employ strategies to enhance the effectiveness of such emerging therapies;

• repair and reverse the cell damage caused by Sanfilippo syndrome, which could include the application of neuro-regeneration advances made for other neuro-degenerative diseases; and

• improve quality of life through palliative care and symptom management specific to Sanfilippo syndrome.

Beginning March 1, 2018 be sure to visit their web site for more information. They are accepting e-mail inquiries also.

2017 Million Dollar Bike Ride Pilot Grant Program

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The University of Pennsylvania Orphan Disease Center’s 2017 Million Dollar Bike Ride Pilot Grant Program is now open! The Million Dollar Bike Ride (MDBR) Pilot Grant Program will provide a one-year grant to support research related to a rare disease represented in the 2017 Million Dollar Bike Ride. All individuals holding a faculty-level appointment at an academic institution, or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA. Interested applicants must first submit a Letter of Interest (LOI), which is done by completing a brief online form, and uploading a 1‐page Pre‐Application. This LOI is due by Monday, September 18, 2017 by 8:00 p.m. EST. Full grant applications are accepted by invitation only after LOIs are approved.

The following lysosomal diseases were represented in the 2017 Million Dollar Bike Ride last May in Philadelphia, and their available grants are detailed:

Mucolipidosis Type IV (ML4): One $71,939 pilot grant available. This grant is offered to investigators conducting research on all aspects of ML4 disease, including disease pathogenesis and clinical studies. Preference will be given to those research projects developing new therapies for ML4, and translational research projects that improve our understanding of the disease state and pathogenesis, such as identifying biomarkers or functional outcome measures to assess therapeutic impact. This grant is made possible by the intrepid riders of Together4Ido, TeamCureML4, Climb4Carin, Pedal4Paul, Danny4theGirls, and MayaanHikes4Meira.

Mucopolysaccharidoses (MPS): Two $59,449 pilot grants are available. Applications directed to treating the central nervous system manifestations or enzyme replacement therapy antibody responses are sought. These two grants are made possible by Team MPS, the National MPS Society, and The Ryan Foundation.

Niemann Pick Type C (NPC): Two $49,645 pilot grants are available. Consideration will be given to research projects developing new therapies for NPC, as well as those designed to complement therapies presently in the pipeline. Consideration will also be given to gene therapy proposals; studies focused on problems, including psychiatric issues, impacting quality of life through the lifespan of the patient population; and projects that improve our understanding of the biology, pathogenesis and disease state and that have a direct impact on translation of new treatments to patients. These two grants are made possible by Team NPC, the Andrew Coppola Foundation, Jammin’ for JP, Chase the Cure and iPedal4Chad.

Tay-Sachs, Sandhoff, GM-1, or Canavan Disease: One $42,419 pilot grant is available focusing on forms of Tay-Sachs, Sandhoff, GM-1, or Canavan disease. Proposals are solicited for innovative research projects that involve basic research, translational studies or clinical studies relevant to these diseases. Projects may be focused on (1) pre-clinical and clinical research needs, such as clinical outcome measures, registries, animal models, or biomarkers; or (2) technology approaches such as stem cells, molecular chaperones, substrate inhibitors, small molecule drug screening, gene therapy, or novel drug delivery to the brain. This grant is made possible by Team NTSAD and the National Tay-Sachs & Allied Diseases Association.

Questions regarding the scientific content of potential research projects can be directed to Monique Molloy; administrative queries should be sent to Samantha Charleston at the University of Pennsylvania’s Orphan Disease Center.

Funding Opportunity for Rare-disease Patient Support Groups

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Global Genes® Grants Available!

Global Genes® RARE Patient Impact Grant Program is an exclusive funding opportunity for rare-disease patient support groups and RARE Foundation Alliance partner organizations. (The RARE Foundation Alliance is a coalition of over 300 rare disease organizations that exchange best practices and share lessons learned to achieve better outcomes for the entire rare disease community.)

The first step in the application process is the submission of a Letter of Intent. Your Letter of Intent will be evaluated based on same selection criteria as the full grant application:
1) Innovative 2) Impactful 3) Achievable 4) Tangible and 5) Scalable. The Letter of Intent Deadline is Friday, August 11, 2017 at 11:59 p.m. Pacific Time. The Full Application is due (from those having accepted Letters of Intent only) by Monday, September 18, 2017 at 11:59 p.m. Pacific Time. RARE Patient Impact Grant applicant presentations will occur during the week of December 4, 2017. A handy online contact form will facilitate your questions reaching the correct person at Global Genes®.

Cystinosis Grants Seek Applicants!

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The Cystinosis Research Network (“CRN”) has issued its “Call for Proposals” for their 2017 research grant cycle. Priority is given to interventional research, both clinical and basic, that will lead to improved treatments for cystinosis. New investigators are particularly encouraged to apply! The CRN has a strong interest in funding projects related to advancing Newborn Screening for Cystinosis – applications regarding this subject will have priority.

Submission deadline is June 1, 2017. Proposals will be reviewed in July and notification will be made to applicants in August. After interested persons have read the above-linked web pages, they may direct their questions to Christy Greeley, Vice President for Research at the Cystinosis Research Network. Good luck, everyone!

Sanfilippo Grant Competition Now!

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The Sanfilippo Children’s Foundation’s 2017 grant-funding round is now open for medical research into Sanfilippo syndrome. They are accepting expressions of interest until April 3, 2017. Successful expressions of interest will be notified in mid-April 2017. The deadline for full applications is May 19, 2017. Funding decisions will be made in August 2017.

The Sanfilippo Children’s Foundation funds Sanfilippo syndrome medical research internationally, and has two types of grants available: ‘Incubator’ and ‘Translational.’ They also fund PhD scholarships within Australia, where the Foundation is located. The Sanfilippo Children’s Foundation is particularly interested in three focus areas:

• halt disease progression through therapies such as enzyme replacement, gene therapy and cell therapy, and employ strategies to enhance the effectiveness of such emerging therapies;

• repair and reverse the cell damage caused by Sanfilippo syndrome, which could include the application of neuroregeneration advances made for other neuro-degenerative diseases; and

• improve quality of life through palliative care and symptom management specific to Sanfilippo syndrome.

Be sure to visit their web page for more information. They are accepting e-mail inquiries also.

Penn Medicine Orphan Disease Center Grants!

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The Penn Medicine Orphan Disease Center at the University of Pennsylvania in Philadelphia has announced the recipients of the “2016 Improved Therapies for MPS I” pilot grants. They include two Lysosomal Disease Network physician-researchers: Igor Nestrasil, MD, PhD and Raymond Wang, MD:

• Igor Nestrasil, MD — University of Minnesota
Discovery of brain MRI signatures in infants with severe form of MPS I in the pre-HSCT and post-HSCT stage

• Raymond Wang, MD — CHOC Children’s Hospital (Orange County, California)
Intra-articular Gene Therapy for Canine Mucopolysaccharidosis Type I Joint Disease

• Joseph Anderson, PhD — University of California, Davis
Alpha-L-iduronidase Delivery via Human CD34+ Hematopoietic Stem Cells

Research is the Only Way Forward!

• Kim Keeling, PhD — University of Alabama at Birmingham
Identify Drugs to Treat MPS-IH Caused by Nonsense Mutations

• Lachlan Smith, PhD — University of Pennsylvania
Novel therapies to improve bone formation in Mucopolysaccharidosis I Dogs

• Drew Weissman, MD, PhD — University of Pennsylvania
Nucleoside modified mRNA therapy for MPS I

• Carmine Settembre, PhD —
Telethon Institute of Genetics and Medicine (Pozzuoli, Italy)
Modulation of autophagy to treat skeletal features in MPS I

Congratulations to these brilliant researchers!

Research Funding Opps Seeking Applicants!

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The Perelman School of Medicine at the University of Pennsylvania’s Orphan Disease Center (“ODC”) sponsors a Million Dollar Bike Ride each May in Philadelphia. As the result of this year’s successful Million Dollar Bike Ride, the ODC is offering several research funding opportunities in the lysosomal diseases. These grants are open to the international community (they are not limited to University of Pennsylvania or the Children’s Hospital of Philadelphia). These lysosomal disease research funding opportunities are:

1. For mucolipidosis type IV (ML4), one $45,000 pilot grant available to investigators conducting research on all aspects of disease including disease pathogenesis and clinical studies. This grant is made possible by Team Cure ML4, Pedal4Paul, Climb4Carin, and the ML4 Foundation.

2. For the MPS diseases, two $52,000 pilot grants available. ODC is seeking applications directed to treating the central nervous system manifestations of these diseases. These grants are made possible by Team MPS, the National MPS Society and The Ryan Foundation.

3. For Niemann-Pick disease type C (NPC), two $51,000 pilot grants are available. ODC will give preference to research projects developing new therapies for NPC, and translational research projects that improve our understanding of the biology, pathogenesis and disease state (i.e., biomarkers or functional outcome measures to assess therapeutic impact). This grant is made possible by Team NPC, the Andrew Coppola Foundation, Jammin’ for JP, Chase the Cure and iPedal4Chad.

4. For Tay-Sachs, Sandhoff, GM-1 or Canavan disease, one $42,000 pilot grant is available focusing on forms of Tay-Sachs, Sandhoff, GM-1, or Canavan disease. ODC is soliciting proposals for innovative research projects that involve basic research, translational studies or clinical studies relevant to the diseases mentioned above. Projects may be focused on (1) pre-clinical and clinical research needs, such as clinical outcome measures, registries, animal models, or biomarkers; or (2) technology approaches such as stem cells, molecular chaperones, substrate inhibitors, small molecule drug screening, gene therapy, novel drug delivery to the brain. This grant is made possible by Team NTSAD and the National Tay-Sachs & Allied Diseases Association.

Eligibility for these funding opportunities is restricted to individuals holding a faculty-level appointment at an academic institution, or a senior scientific position at a non-profit institution or foundation.

Deadline to Apply: Submit your Letter of Interest (“LOI”) in the required online form no later than Friday, September 16, 2016 by 8 p.m. EST. Full application is by invitation only, after review of LOIs.

For the downloadable Million Dollar Bike Ride RFA Guidelines, the link to the online Letter of Interest submission form that must be used, the full list of all available Million Dollar Bike Ride funding opportunities (this includes many non-lysosomal diseases) and other information, visit: current Orphan Disease Center funding opportunities and read the entry entitled “2016 Million Dollar Bike Ride Grants.”

Questions regarding the scientific content of potential projects can be directed to Ms. Molloy at moniquek@nullexchange.upenn.edu; administrative queries should be sent to Ms. Charleston at scharle@nullupenn.edu.

FDA to Spend $2M on Rare Disease Research

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The U.S. Food and Drug Administration (FDA) is making $2 million available in new research grants to fund natural history studies in rare diseases. The aim is to collect data on how specific rare diseases progress in individuals over time. This knowledge can inform and support drug product development and approval. This will be the first time the FDA will provide funding through its “Orphan Products Grants” to conduct natural history studies for rare diseases.

Natural history is the course a disease takes in affected individuals from the time immediately prior to its inception, progressing through a pre-symptomatic phase and different clinical stages, to a final outcome in the absence of treatment. This type of information is often not available, or incomplete, for many rare diseases, and this makes the drug-approval process additionally difficult.

The funding levels and duration of these grants will include:

● A maximum of $400,000 in total costs per year for up to five years for prospective (looking forward) natural history studies involving clinical examination of affected individuals.

● A maximum of $150,000 in total costs per year for up to two years for retrospective (looking back) natural history studies (i.e., chart review) or survey studies (i.e., questionnaire).

Grant applications are due not later than Oct. 14, 2016. All responsive applications will be reviewed and evaluated for scientific and technical merit by a panel of rare disease and natural-history experts. The anticipated start date of funding for grantees is March 2017.

Visit: FDA press release about $2m in new grants for natural history of rare diseases

Sanfilippo Children’s FDN in Australia

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The Sanfilippo Children’s Foundation in Australia is currently calling for “Expressions of Interest” for their 2016 Project Grant Round. This opportunity is not limited to researchers in Australia. These grants include translational research grants which provide funding for projects with significant potential to move through the translational pipeline towards treatment of Sanfilippo syndrome. This includes pre-clinical and clinical research to develop new treatments.

These grants also include incubator grants which focus on small-scale projects investigating bright new ideas in treatment of Sanfilippo syndrome. These small-scale projects should have the potential to generate the kind of results that can attract larger-scale funding.

Deadline for the submission of expressions of interest is April 29, 2016. Successful expressions of interest will be invited to submit a full application in mid-May. Deadline for full applications is June 25, 2016. Funding decisions will be made in August 2016. Visit their Web site: Click here for details.