Externally sponsored research (esr)

Building on the extensive knowledge gained from years of research, GBT is focused on changing the treatment paradigm for SCD. As part of this goal, we continue to advance research and enhance understanding of diseases and therapeutics through externally sponsored research (ESR) studies that align to GBT’s Areas of Research Interest. These studies will add to the scientific understanding of the mechanism of action and the clinical application of our medicines.

GBT welcomes unsolicited proposals from qualified sponsor-investigators with promising ideas in our Areas of Research Interest. Well-written, scientifically sound studies that enhance the understanding of our medicine, improve patient care, and spark new ideas for disease-related research will be considered. Our medical science personnel are available to guide you through each step in the process, from concept pre-approval to submitting the final budget and protocol. GBT may support both interventional and non-interventional ESR studies in the form of financial support, product candidate/product supply, or both.

Types of ESR Studies Eligible for Support:

  • Clinical studies of approved and investigational uses, involving marketed GBT drugs or those still in development (interventional studies phase I to IV)
  • Clinical observational studies, real world evidence (non-interventional studies)
  • Preclinical studies

Categories of ESRs:

  • Investigator Initiated-Sponsored Research (IISR)
    Unsolicited research that is planned, designed, initiated, and conducted by a Sponsor-Investigator where GBT assumes no legal and/or regulatory accountabilities for such research.
  • Externally Sponsored Collaborative Research (ESCR)
    Unsolicited or solicited research that may be planned, designed, or initiated in conjunction with GBT personnel and is conducted by a Sponsor-Investigator where GBT does not assume legal or regulatory accountabilities for such research.

Sponsor-Investigators are welcome to submit their research proposals in their area of interest to GBT for consideration. The list of Areas of Research Interest below for voxelotor represents current data gaps identified and will be updated periodically. 

SCD Areas of Research Interest

  • Safety and tolerability of voxelotor in SCD patients with severe renal impairment (estimated glomerular filtration rate [eGFR] of < 30 mL/min/1.732 or on chronic dialysis)
  • Safety and efficacy of voxelotor in patients with Hemoglobin SC disease (also called sickle-hemoglobin C disease) 
  • Characterize the outcomes and impact of voxelotor on depression and mood in SCD patients using validated instruments and/or psychological evaluation
  • Characterize the impact of voxelotor on vision and retinal outcomes in SCD patients
  • Examine the clinical effect of voxelotor used in combination with other SCD therapies

Non-SCD Area of Research Interest

  • Clinical studies evaluating the impact of voxelotor on physiological and clinical endpoints in patients with hypoxemia in acute lung injury (ALI) or acute respiratory distress syndrome (ARDS) from any cause. See RFP below or click the link here.

2022 RFP Initiative

The 2022 RFP initiative is now open. Please click the link here to review areas of research interest.

If you have questions or would like to discuss your research concept, please reach out to your GBT Medical Science Liaison.

Do not know your GBT MSL? For general inquiries, please email us at: esr.gbt@gbt.com