Mass General Brigham Gene and Cell Therapy Institute

Gene and Cell Therapy Spark Grant: 2023

From groundbreaking research to successfully conducting clinical trials, Mass General Brigham researchers, scientists, and clinicians are continuously working to help patients globally with rare and complex disorders. Through groundbreaking gene and cell therapy research and treatments, we are always looking for potential cures. Our priorities are to imagine and create what is scientifically possible in conjunction with the highest clinical need.

With more than $2.3 billion in total research funding, Mass General Brigham has more than 400 researchers who are conducting gene and cell research in two categories:

  • Platforms (viral vector delivery, gene editing etc.), which apply across cohorts
  • Therapeutics

The Gene and Cell therapy Institute (GCTI) Spark grants aims to accelerate the translation of gene and cell therapies and improve patient health.  The program will enhance the innovative potential of the Mass General Brigham GCT research community, stimulate new inventive concepts, identify areas of commercially significant scientific strength, and accelerate commercialization of Mass General Brigham intellectual assets.  For fiscal year 2023, the GCTI funding opportunity is open to all clinical areas and technology types that include cell or gene therapies, with a focus on research and preclinical stage programs.  Multi-investigator collaborations are encouraged.  The GCTI program will fund projects that can demonstrate tangible advancements towards clinical applications as well as commercialization outcomes including licensing, commercial partnerships, or new spinoff companies.

We will continue to partner with industry to advance gene and cell therapy.

Gene Therapy

  • Many diseases can involve a genetic component — a missing gene, multiple copies of a gene, or a gene that is structurally defective. Gene therapy is the process of replacing or modifying genes to correct a deficiency, sometimes by altering the DNA sequence of the target gene or by modifying expression of a gene by turning it off or on. Gene therapy can have an especially profound effect in congenital diseases where the disease is associated with malfunction of a single gene. Researchers are working to identify such genes and develop therapies to replace or modify genes.

Cell Therapy

  • Cell therapy involves replacing or modifying cells to treat disease. Cell therapy can use cells from another person (allogenic) or the person’s own cells (autologous). These cells may or may not be genetically altered. Cell therapy is personalized to treat an individual’s condition, like CAR-T cell therapy, where scientists take the person’s own cells and genetically modify them outside the body, expand the number of modified cells and then re-infuse these modified cells to produce a therapeutic benefit. Such therapies alter the patient's cells so their immune system can now target and destroy previously stealth cancer cells.

Platform Technology

  • Refers to a technology that enables the clinical translation of gene and cell therapies and can be used as a base upon which other applications, processes, or technologies are developed.  Platform technologies in the gene and cell therapy space include genome editing, gene delivery modalities, manufacturing improvements, discovery techniques and processes, and biomarker assay development.

Gene editing technology directly edits entire genes or parts of genes within the cell either within (in vivo) or outside (ex vivo) the body and can be used both for gene and cell therapies.

Key Dates and Information:

  • Pre-proposals are due no later than May 10th, 2023 11:59pm EST
  • Full proposals will be invited June 2nd, 2023
  • Full proposals will be due no later than June 30th, 2023 11:59pm EST
  • Full proposals selected for technology development will be announced in early August, 2023.
  • The earliest possible start date is September 1, 2023.

Award Information:

  • No portion of the award may be subcontracted outside the Mass General Brigham community.  However, other costs for CRO or other commercial vendor services and GMP manufacturing are allowable and can be negotiated by GCTI.  All vendors and consultants must agree that all intellectual property rights will be assigned to the appropriate Mass General Brigham-affiliated institution.
  • Full proposals must include an itemized budget along with a budget justification that describes the basis for all estimated costs.
  • Salaries must include appropriate fringe benefit costs.
  • Awards may not be used for capital equipment, travel expenses, subcontract expenses, or patent expenses.
  • Award funding will be released in accordance with tranche plans developed by the PI in coordination with the GCTI Program Manager.  Tranche plans will be requested once full proposals are selected for award.  Tranche funding will be utilized to support GCTI awards in accordance with guidance provided by the GCTI Program Manager and progress achieved.
  • Each stage in translational and clinical research has different costs depending on the technology developed.  The following section outlines current thinking on standard academic costs for progressing technologies.   The following information provides amounts up to which funds may be requested in accordance with the technology development stage.  Indirect costs at 20% must be included in the budget as part of the funding limits provided below.

Two tiers of research support:

1. The Research/Preclinical Stage: includes concept development in research laboratories with an advanced medical therapy concept that is actionable and determines IND potential. The Research/Preclinical Stage may include safety and efficacy studies in an animal model (usually rodent); these studies also determine route, dose-response, frequency and duration of administration and enable at least provisional filing of IP and determine initial freedom to operate position.Data from preclinical studies justify advancing to the IND stage. [Funding maximum is $250,000 including 20% IDC]

2. *Please note: Funding for IND-enabling/Clinical stage projects will begin in the 2024 funding cycle and applications are not open at this time. The IND-Enabling/Clinical Stage: includes the work to complete to justify performing first in human, proof of concept clinical trials to the FDA.  The work includes all studies necessary to file an Investigative New Drug (IND) Application to the FDA, including GLP pharmacology and toxicology, preparation of clinical trial draft protocol and all manufacturing requirements for the Chemistry, Manufacturing, and Controls section of the IND; this work may employ external CROs.

The Clinical Stage is intended to show proof of concept. Assume treatment of up to 10 to 15 patients, depending on statistical significance. Any prior retrospective product and/or disease history studies can also be included as part of the Clinical Protocol draft and IND application.


  • To be eligible for a GCTI Spark award, lead applicants must have PI privileges at an institution within Mass General Brigham.
  • IP must be assigned to a Mass General Brigham entity.

Intellectual Property

IP must be assigned to a Mass General Brigham entity and awards will be governed by the Mass General Brigham Intellectual Property Policy.

Do not include unprotected proprietary information in your application.  Questions related to intellectual property and confidentiality should be directed to your Innovation Licensing Manager at 857-307-2400

Additional information

  • Additional information can be found in the Program Guidelines.
  • Questions related to GCTI Spark grants policies or procedures should be directed to Meini Shin, Gene and Cell Therapy Institute, Mass General Brigham
  • Questions related to the business opportunity should be directed to your Partners Innovation Licensing Manager.