The Society’s Board of Directors is pleased to announce the approval of $725,000 in new funding to be awarded as part of the Society’s competitive research grants program for 2022. These funds are made available through the great generosity of friends of the Society and especially through the efforts of our membership families. This year, the funds are dedicated to be used to further the research needs of syndromes as described below.

• General MPS/ML research
• MPS I research
• MPS II research
• MPS III research
• MPS IV research
• MPS VI research
• ML research

To give researchers and reviewers both the certainty of a set calendar of grant submission dates and to allow multiple submission cycles per year, the Society now has scheduled three grant proposal submission cycles per year. As the individual cycle proceeds, the details of a subsequent individual call for may be altered, or a call may be cancelled, pending the results of the previous cycle. The dates and additional details are below.

The Society Research Portal should be open three weeks before a LOI due date: https://mpssociety.org/research-grants/

• Research Tier I Grant
  • Research will address complex or multidisciplinary questions and/or unmet needs in basic, translational, clinical, or diagnostic aspects of the MPS and ML disorders
  • Up to a total of $100,000 US, disbursed over one to two years at the discretion of the PI
  • A competitive renewal under this mechanism is possible, but will only be funded once
  • Total funds of an original grant and its renewal will not exceed $200,000
  • Total funded period of a grant and renewal will not exceed 4 years (excluding NCE)
  • Restrictions apply (see below)

• Research Tier II Grant
  • Research is more defined than Tier I, and may address but is not limited to the following:
    • Discrete studies or deliverables involving drug repurposing, models, etc.
    • Research needs designed to make possible a significant and new research grant
    • Research needs designed to improve a significant research grant resubmission
    • Analysis of previously compiled data or research resources
    • Pre-clinical extension to an alternate syndrome of a previously validated therapy
  • Up to a total of $50,000 US, disbursed over one to two years at the discretion of the PI
  • A competitive renewal under this mechanism is possible but will only be funded once
  • Total funds of an original grant and its renewal will not exceed $100,000
  • Total funded period of a grant and renewal will not exceed 4 years (excluding NCE)
  • Restrictions apply (see below)

• Research/Clinical Scientist Fellow-Initiated Research Grant
  • The Fellow-Initiated application will, in most cases, address a Tier II level question
    • In rare cases the research may approach Tier I level research in scope
  • Awards will be limited to one year at $50,000 (US)
  • The basis of any award decision will rest on rigorous review of the research component
  • A required mentor’s statement must be included which address mentorship in research
  • Eligible for up to one renewal
  • Restrictions apply (see below)

• Research Tier III Grant
  • Addresses a discrete need not likely to be part of a larger research or grant initiative
  • Research is more defined than Tier II, and should address either:
    • A discrete study of an existing data or specimen resource
    • Be designed to establish a critical clinical practice consensus supporting a peer reviewed scholarly output such as a Delphi or modified Delphi consensus statement
  • Up to a total of $25,000 US, disbursed over one year
  • Restrictions apply (see below)
    
    

The Society welcomes submissions of any type that addresses any of the MPS or ML disorders (except as restricted below), but specifically seeks applications in the current cycle (Cycle II, 2022), that address those topics listed under #1 or #2 below. Note that an application is not required to address topics in both #1 and #2, but an application certainly may. Also please note that any application that addresses any topic important to MPS and ML disorders will receive full consideration as long as it is not an an area of research restricted in this cycle.

1. Applications addressing one the following disorders, areas, and/or research resource development:
   • Address ML II/III, MPS I, MPS II, MPS III, MPS IVA, or MPS VI
     • Alone or as part of a comparative approach involving other syndromes
   • Applications that address the lack of authentic large animal models of MPS II or IVA
   • Applications that address therapy for disorders of a membrane bound enzyme
   • Applications that involve pre-clinical/clinical assessment of repurposed drugs to address bone, connective tissue, cardio-respiratory, and/or immune/inflammatory disease
   • Applications that address the consequences of mutations affecting protein folding, protein maturation, and/or ER stress independent of enzyme activity or substrate accumulation
   • Discrete research or scholarly efforts addressing: either a previous existing data or specimen resources, or designed to establish a critical clinical practice consensus supporting a peer reviewed scholarly output.
   • Application to validate and implement RUSP enabling newborn screening of the NBSTRN RUSP Candidate Listed Conditions listed below. The proposed research may also involve simultaneous validation and implementation of the RUSP listed core conditions of MPS I and/or MPS II:
     • MPS IVA (Galactosamine 6-sulfatase (GALNS) deficiency, (Morquio A syndrome))
     • MPS VI (Arylsulfatase B (ARSB) deficiency, (Maroteaux-Lamy syndrome))
     • MPS VII (Beta-glucuronidase (GUSB) deficiency, (Sly syndrome))
2. Physiological systems with commonality between individual MPS and ML disorders:
   • Common bone and connective tissue pathology, treatment, and/or outcomes
   • Common cardio-respiratory pathology, treatment, and/or outcomes
   • Common CNS pathology, treatment, and/or outcomes
   • Common inflammatory/immune pathology, treatment, and/or outcomes

Approaches that will not be considered in Cycle III of 2022:

1. Proposals focusing on pharmacoperone or pharmacological chaperone therapy.
2. Proposals focusing on current or novel gene therapy vectors or gene therapy as a stand alone therapy evaluating therapeutic delivery of the normal cDNA to treat MPS I, II, IIIA, IIIB, VI, or VII.
   • Gene therapy exceptions:
     • Proposals focusing on treatment of MPS IIIC, IIID, IVA, or ML II/III will be considered.
     • Proposals focusing on treatment combining HSCT and gene therapy will be considered for all syndromes.
     • Also permitted: gene therapy techniques used as tools in proof of principle studies not focused on vector optimization or therapeutic delivery of the normal cDNA, where delivery of enzyme is most expediently delivered by the experimental use of a gene therapy vector.
3. Proposals focusing on current or novel gene editing techniques to correct mutations or to integrate a normal cDNA.
   • Permitted: gene editing techniques used as tools in proof of principle studies not focused on mutation correction or integration of a normal cDNA.

Eligible Institutions

• Any domestic US or foreign, public or private, non-profit or for-profit research organization is eligible to apply for and receive a Society Research Grant.

Eligible Investigators

• Research Tier I, II, or III Grants
  • Any individual with the skills, knowledge, and resources necessary to carry out the proposed research as a Principal Investigator (PI) is invited to apply.
  • A PI must be institutionally eligible to apply for, be awarded, and administer an extramural research grant.
• Research/Clinical Scientist Fellow-Initiated Research Grants
  • Eligible applicants include PhD candidates (preliminary exams completed) and medical professional students (to support a one-year hiatus from professional training to pursue research), and post-doctoral fellows (within 3 years of PhD or clinical degree).
    • Medical professional training programs and clinical degrees include the following training programs and degrees: DDS, DO, DVM, MD, VMD and equivalents.
  • Fellow applicants must have a principal mentor who satisfies the institutional requirements of a PI as defined above under the Research Tier I and II Grants heading.

• Budgetary restrictions include:
  • No indirect cost recovery charges are allowed by the National MPS Society.
  • No tuition is allowed.
  • No salary for a principal investigator, or any term or tenured faculty, researcher, or clinician who is institutionally eligible to serve as PI as defined under the Research Tier I and II Grants description.
  • No equipment over $5,000 is permitted, except once for a Tier I proposal and only by pre-approval (before submission of LOI) from the Society CSO. Such approval will only be considered for purchase of one piece of equipment between $5,000 and $10,000 US which is directly related to the proposed scope of work.
  • Funds for travel to conferences is restricted to 2% of the grant total.
    • Any such travel must be documented to be for the purpose of reporting on Society funded research.
    • Transportation that is integral to the research is not thus limited.
  • Fellow-Initiated research must budget 75% of the award toward Fellow salary support, with the balance dedicated to research costs.
• Additional Requirements and Restrictions
  • Only one research grant (Tier I, Tier II, or III, or Fellow-Initiated) application per independent laboratory group within an institution may be submitted per cycle.
    • Failure to adhere to this restriction or efforts to circumvent it may permanently restrict future applications from a given principal investigator or institution.
  • A condition of a research award will be the attendance and participation by the PI or a designated member of their laboratory at an entire Annual Family Conference of the National MPS Society once during the period of the award or within one year of the end of the award. Fellow grantees must fulfill this obligation by the attendance of the awardee within the year of their award unless the Notice of Grant Award (NOGA) is within one month of a scheduled Family Conference, in which case they may attend the following year’s conference. As part of participation at the meeting the attendee will be invited/expected to present on their research findings in the research sessions.
    • Failure to adhere to this restriction or efforts to circumvent it may permanently restrict future applications from a given principal investigator or institution.
  • No competitive renewal application may be made without a letter of request addressed to the CSO received 30 days prior to submission deadline of a competitive renewal LOI or fellow-initiated research grant.