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Open Call for New Projects: Invitation to join the Ladders to Cures Accelerator Community
The Ladders to Cures (L2C) Accelerator is an ambitious new initiative whose mission is to catalyze progress across the research ecosystem and accelerate advances leading to treatments and cures for patients with rare genetic diseases. Submit your project proposals by Sunday, June 30.
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The Ladders to Cures (L2C) Accelerator is an ambitious new initiative whose mission is to catalyze progress across the research ecosystem and accelerate advances leading to treatments and cures for patients with rare genetic diseases.

To fulfill our mission, we aim to develop and deploy scalable technologies to investigate disease mechanisms and to accelerate the development of therapeutic strategies. We are particularly interested in discovering shared “nodal” mechanisms between many rare – or rare and common – genetic diseases to accelerate our ability to develop targeted, precision therapies.

This is our first open call for transformative new ideas that will advance our mission. Selected projects will join the L2C Accelerator ecosystem and gain access to seed funding and scientific advisors with extensive experience in both industry and academia, to help advance therapeutic hypotheses from the lab to the clinic. Technology development projects that can be applied to rare disease discovery/therapeutics are also encouraged.

Ladders to Cures Accelerator Goals

We are focused on developing a deep mechanistic understanding of genetically-defined rare diseases. This may take many forms including:

  • Given a group of causal genes/variants, what is the disease mechanism?
  • Given a defined mechanism, can we establish a proof-of-concept therapeutic hypothesis?
  • Given a proof-of-concept therapy, can we develop a path to the clinic?

Successful L2C projects will interrogate multiple genes of interest in parallel, aiming to uncover shared, convergent or “nodal” mechanistic insights and potential therapies.

Proposers are encouraged to discuss plans for partnerships with patient organizations, and where appropriate, to outline the essential data that will be required to facilitate clinical development (i.e. status of patient registries, natural history studies, biomarkers, etc.).


We are encouraging ideas from all members of the Broad scientific community in partnership with collaborators across the Boston ecosystem and beyond. The only requirement is that one member of the team is a Broad Principal Investigator. You are welcome to bring in collaborators from anywhere, including patient groups and organizations. All Broadies with PI status are encouraged to submit their ideas. Scientists affiliated with the Broad but lacking standing-PI rights can easily apply for case-by-case PI rights for a particular L2C project - please reach out to if you are unsure of your status or need assistance acquiring case-by-case PI rights.

How to submit your project idea

Applicants are encouraged to discuss potential Ladders to Cures project ideas with Jillian Shaw (Scientific Advisor to L2C) at least two weeks prior to submission.

Seeking input from colleagues and platform or program leads is encouraged, but please note that letters of support are not required.

Project ideas will be evaluated by a committee composed of L2C steering committee members and L2C advisors with extensive experience in both academia and industry.

Successful teams will be required to showcase their work in an upcoming L2C Symposium!

Please email your project idea by Sunday, June 30, 2024 to as one merged PDF that includes the following sections:

Project Title

Principal Investigator(s)
Please include a preferred email address for the Lead PI. At least two PIs are required.

Impact Statement
How will this project make a difference?  Please describe in 3-5 sentences the impact this project will have on advancing a therapeutic hypothesis for genetically-defined rare diseases.

Project Idea
Please develop your project idea by answering the following questions in no more than 2 pages.  Specifically, please comment on the scalable capabilities you need to deploy or build to make progress toward multiple genes of interest driving rare genetic disease(s).

  1. the challenge – what obstacle are you attempting to overcome?
  2. the stakeholders – what patient populations would be directly impacted by your research? What are the specific genes of interest you intend to interrogate?
  3. the solution – how do you anticipate leveraging high-throughput, scalable, cross-cutting technology to advance our mechanistic understanding of and possible therapeutic approach to your disease(s) of interest? 

Project Team
Please list the collaborators and expertise required to bring your idea to fruition. 

Patient Foundation(s) and Patient Advocate(s)
Where appropriate, successful L2C projects will partner with patient stakeholders.

Broader Community Expertise and Input
Please seek feedback and guidance from the broader scientific community, e.g. Broad platform leaders, prior to submission. Please list the colleagues/experts you consulted.