BioMarin is a world leader in developing and commercializing first-in-class and best-in-class innovative biopharmaceuticals for rare diseases driven by genetic causes. BioMarin’s mission is to bring new treatments to market that will make a big impact on small patient populations. We are inviting scientists to submit short proposals to join our efforts in advancing treatments for rare diseases.

Focus Area:
BioMarin is looking for scientists who are developing innovative therapeutic approaches and enabling technologies that correct the underlying molecular defect in genetic-based rare diseases. Our standard boundary conditions are:

  • Rare mono genetic disease with a prevalence of at least 1:50,000
  • Broad therapeutic areas, but excluding oncology, infectious disease. Focus on CNS, muscular skeletal, hematology and cardiac diseases
  • Significant unmet clinical need with discernable clinical endpoints
  • Treatment addressing the underlying molecular pathology
  • Transformative impact – where treatment is expected to stop progression or provide reversal
    Enabling Technologies of interest are platform technologies for genome editing, vector engineering, new or improved modes of delivery and enhanced lead identification.

Technology Scope:
We are interested in a broad range of modalities and relevant enabling technologies including:

  • Small Molecule and Biologic therapeutic approaches
  • Gene Therapy and Gene Editing
  • Oligonucleotide and mRNA directed therapies
  • Cell-based therapies

Format and Criteria:
Proposals should be brief and not exceed 500 words total and should include the following:

  • Abstract on your proposal highlighting the potential impact in advancing treatments for rare diseases
  • High-level timeline to achievable goals (ideally the proposal would be no longer than 18 months, can include a possibility to extend based on the results)
  • High-level budget summary (Project Dependent)
  • Background on you and your team

Key Dates and Notes:

  • Proposal submissions are due by Feb. 1, 2022, but teams are encouraged to submit ASAP
  • Email submissions to tido.alliances@childrens.harvard.edu
  • Selected proposals will be reviewed and contacted in approximately 30 days post submission
  • Please note that based on the feedback received from this call for proposals, BioMarin could consider opening a portal to allow submissions year round to enable scientists to pitch new projects at any time.

    • We greatly appreciate and thank all applicants for their time and effort. We regrettably do not have the capacity to reach out to proposals that do not meet the criteria listed above.

    The proposal form is available for download here.

Deerfield has pledged up to $65 million with the goal of facilitating the translation and commercialization of novel biomedical research at Boston Children’s Hospital through a new collaboration called Blackfan Circle Innovations. Through Blackfan Circle, Boston Children’s Hospital scientists have the opportunity to submit therapeutic proposals for potential funding.

Deerfield will provide research funding and operational support in product development and commercialization to co-develop the jointly-selected research therapeutic candidates with the goal of achieving Investigational New Drug (IND) readiness. Successful projects that reach IND-enabled status may receive additional funding.

We would like to invite BCH investigators to begin the submission process to Blackfan Circle Innovations, the newly established entity created to accelerate research at BCH.

Areas of Interest:

  • Broad interest across all therapeutic areas and modalities
  • Early-stage projects with potential to progress to IND-enabled status, early clinical development, and human proof-of-concept

Proposal Process:

  • Proposal Concept Sheet
  • Letter of Intent
  • Full Proposal and Presentation

In order to engage initial interest with Blackfan Circle Innovations, TIDO encourages BCH investigators to submit one or more Proposal Concept Sheet(s), each of which will constitute a brief overview of the proposed project focusing on the areas listed below.

  • Genetic Evidence
  • Biologic Rationale
  • Preclinical Execution
  • Unmet Need
  • Clinical Development (if applicable)
  • Novelty/ Differentiation

A Proposal Concept Sheet submission template will be provided by TIDO. For more information on proposal submission to Blackfan Circle Innovations, a copy of the Proposal Concept Sheet, or questions related to the application process, please contact Sabrina Kamran, Strategic Alliance Manager at Sabrina.Kamran@childrens.harvard.edu.

If your Proposal Concept Sheet is selected for advancement, you will be asked to complete a Letter of Intent with a more detailed description of the proposed project. Finally, at the Full Proposal stage of evaluation, you will be asked to submit a detailed research plan and budget and meet with the Deerfield review team to discuss your proposal. Deerfield Discovery and Development experts will be available to consult with you on your proposal throughout the entire evaluation process.

Who Can Apply:

  • Full-time BCH Principal Investigators
  • Principal Investigators with faculty appointment who are subject to Boston Children’s policies regarding the conduct of research and ownership of intellectual property

Submissions:

Proposal Concept Sheets submitted to Blackfan Circle Innovations will be considered on a rolling basis, with operational and scientific guidance and support from TIDO during the submission process.