2021 BioMarin Request for Proposals 

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 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

Technology Scope:

BioMarin is 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
• Background on you and your team

Please submit your proposals by filling out the form on this page by August 30, 2021. Proposals will be reviewed by the Innovation Institute prior to being sent to the company.

If you are unable to respond in this timeframe please contact Amy Phillips and Paul-Valentin Pitou in the Office of Industry and Economic Partnerships.

Funding

Funding will be provided to support sponsored research for selected proposals. Funding levels will be commensurate with the scope of work. No limit on the number of awards given.

Eligibility

Applicants must be a University of Pittsburgh faculty member. An individual may submit multiple applications.