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We are a community of scientists, physicians, patient advocacy groups, AI/machine learning experts and biopharma veterans working together to gain new mechanistic insights and advance treatments for rare genetic diseases.

Our Research

“Rare” genetic diseases are collectively common: they affect 1 in 10 people globally. The growing list of rare diseases stands at approximately 8,000, but fewer than 500 have an available treatment. The striking mismatch between these numbers underscores the urgent need to develop a new approach at scale to deliver precision cures at an accelerated pace.

What if we could systematically and simultaneously map the nodal biological mechanisms that underlie all known rare genetic diseases? Would this allow us to group diseases based on shared (nodal) biological mechanisms? Would targeting these biological nodes accelerate the path to treatments and cures?

Exploring Nodal Biology

A biological node is a point of convergence on a single druggable target. It is enabled by the discovery of previously unseen shared biological connections between seemingly disparate, but now newly connected, rare genetic diseases. 

The L2C Accelerator’s vision was informed by the lessons learned from the work of many investigators, including our research on one rare genetic disease that de-orphaned many others and was enabled by scalable resources at ӳ����ý. As a proof of concept, a drug designed to target a newly discovered  biological node is showing efficacy in treating rare genetic diseases in the kidney, the eye, and the brain and is now making its way to clinical trials in collaboration with a pharmaceutical partner. While this example validates the concept of targeting a biological node to simultaneously treat several rare genetic diseases, there are potentially hundreds of nodes to discover, underlying ~8000 human diseases caused by as many as 100,000 different mutations. 

Our vision is to uncover as many nodes as possible in a highly parallel manner. The L2C Accelerator is focused on vastly shortening the time to develop therapies for rare disease patients by grouping together hundreds (or even thousands) of rare genetic mutations in parallel and identifying many candidate therapeutic targets (i.e. druggable nodes)

Our Projects

The Ladders to Cures Accelerator will focus on building and supporting: 

1. an unprecedented platform to uncover nodal biological mechanisms across all rare genetic diseases
2. proof-of-concept therapeutic projects focusing on specific biological nodes, that is, druggable targets
3. close partnerships with patient-led advocacy groups
4. a rich, collaborative community empowered to solve biomedicine’s greatest challenges

We are proud to support our first cohort of L2C projects selected via an to the entire ecosystem. Please stay tuned for future open calls! 

News

•  - Anna Greka presents the L2C vision from the TED stage.
•  - Siddhartha Mukherjee (Columbia University) leads a conversation with panelists Anna Greka, Anne Vincent-Salomon (Institut Curie), and Dame Janet Thornton (European Bioinformatics Institute) at the AI for Science Forum, hosted by the Royal Society and Google DeepMind.
• - "Some researchers are trying to address the problem scientifically. Dr. Anna Greka said the ӳ����ý of MIT and Harvard has launched an effort to look at commonalities behind various conditions — or nodes, which can be likened to branches meeting at a tree trunk. Fixing the nodes with gene therapies or other treatments, rather than particular “misspellings” in DNA responsible for one disorder, could address multiple diseases simultaneously. “What this does is it increases the number of patients who can benefit from the therapy,” said Greka, a ӳ����ý member. “It also makes it infinitely easier or more attractive to anyone, like a biopharmaceutical company, to take the project forward and try to bring it toward the clinic, because they’re going to have a bigger market.”"
•  - We are proud to announce our first Open Call to the entire L2C ecosystem in search of the first cohort of L2C projects.

Our Partners