We aspire to vastly shorten the time it takes to develop therapies for patients with genetically-defined diseases.

Our vision is to uncover as many biological and technological nodes as possible to accelerate treatments and cures in patients with chronic genetic diseases.

We are focused on deploying cutting edge, scalable technologies – many invented at the Broad Institute – to investigate shared mechanisms of action and accelerate the development of therapeutic strategies for genetic diseases. In addition to developing “nodal” technologies, we are building a pipeline of key proof-of-concept studies called Leading Edge Projects. The discovery of nodal mechanisms shared between rare and common genetic diseases will revolutionize our ability to develop targeted, precision therapies.

OUR RESEARCH

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

NODAL BIOLOGY

Technological nodes are cutting-edge tools that drive rapid evaluation of therapeutic hypotheses, streamline experimental validation, and accelerate the deployment of therapies to manage or cure genetic diseases.

NODAL TECHNOLOGY

FLAGSHIP PROJECTS

In development, stay tuned!

LEADING EDGE PROJECTS

A glimpse at some current L2C Leading Edge Projects

  • Therapies to Target the Central Mechanism for Cerebral Cavernous Malformations

  • Advancing Liquid Biopsy to Resolve the Durability and Tissue Specificity of Genetic Medicine

  • Multiplexed Optical Pooled Screening for Proteinopathy Therapeutic Discovery

  • A Platform to Rapidly Identify Drugs for Many Rare Genetic Disorders

  • Next Generation Druggability Assessment for Rare Pediatric Cancers

  • Developing an Engine of Discovery for Devastating Diseases of Motor Neurons

  • Targeting DNA Repeat Expansion Disorders

  • A Platform for Cell-Specific Gene Therapy Delivery

  • A Prime Editing Incubator for Childhood Neurodevelopmental Disorders

  • Identifying Small Molecules for Neurodevelopmental Disorders

TOOLS-TO-THERAPIES PROJECTS

A glimpse at the current Tools-to-Therapies Projects

  1. A scalable platform to identify therapeutics for EPHB4-driven Vein of Galen Malformation.

  2. Developing a therapy for microvillus inclusion disease.

  3. Developing genetic medicine for Shwachman-Diamond syndrome.

  4. A scalable, cell-based platform to discover genetically-supported therapies for pediatric nephrotic syndrome.

  5. Nanobodies for improving muscle regeneration in Duchenne Muscular Dystrophy.

  6. Development of cell painting and therapeutic small molecule screening approaches for SELENON-Congenital myopathy as a model for rare disease discovery.