Dear colleagues and friends,

As 2025 comes to a close, all of us at Broad’s Ladders to Cures (L2C) Accelerator are proud to look back on a year of meaningful progress toward our mission: accelerating bold, high-impact efforts aimed at developing therapies for the world’s most challenging genetic diseases. This year, the Ladders to Cures Accelerator grew in scale, expanded its impact, and supported an extraordinary cohort of researchers pushing the boundaries of what is possible.

We are looking forward to a productive 2026 filled with new and exciting discoveries!

Scientific Achievements

• We received philanthropic support to build a bridge between Boston Children’s Hospital (BCH) and Broad to develop the Tools-to-Therapies (T^3) program. After a rigorous selection process, we identified 6 scientific teams that will use Broad platforms to more effectively advance therapeutics-focused projects.

• We established CellWorks, a new capability to produce genetic disease-relevant cell models at scale, with a focus on induced pluripotent stem cells (iPSCs) and gene-edited cell lines for high-throughput screens.

• We are proud to support 16 seed projects involving nearly 200 scientists actively working to advance therapeutic strategies for genetic diseases.

• We launched the Ladders to Cures Accelerator Meeting, a highly-interactive scientific forum focused on bringing together scientists, physicians, Broad platform directors, AI/ML experts, and patient advocacy groups to work together on therapeutic-focused programs for rare genetic diseases. All are welcome to join for these hybrid meetings in 2026!

• We celebrate David Liu, Alex Sousa, Cat Lutz, Holt Sakai and colleagues for publishing the results of their L2C Accelerator supported project focused on developing gene editing approaches to correct mutations in a gene that causes a childhood neurodevelopmental disorder. Read more about it here.

• We congratulate L2C Accelerator awardee Jen Pan for her work on the clinical impact of pathogenic variants found in neurodevelopmental disorders, with a focus on CACNA1A–appearing in Science Translational Medicine. Learn more here.

Community and Collaboration

• Scientific leaders from ten L2C Accelerator seed projects presented their progress during the 2nd Annual Ladders to Cures Symposium that also included a scientific keynote by Jonathan Weissman discussing brainwide silencing of prion protein by AAV-mediated delivery of an engineered epigenetic editor. A symposium highlight was a keynote by patient advocate Jimi Olaghere who presented his personal story, “From Casgevy to Kilimanjaro.” 

• To bring attention to the importance of patient-led research, the L2C Accelerator teamed up with the Termeer Institute to host a symposium focused on this important topic: An Era of Innovation for Rare Diseases.

On the Horizon

We are entering 2026 with tremendous momentum! We are profoundly grateful for major philanthropic support this past year, as we redouble our fundraising efforts in the year ahead. We are thus laying the foundation to support promising L2C Accelerator projects as they advance toward the clinic.

Stay tuned for additional updates throughout the year, and in the meantime, please register for…

11th Annual Rare Disease Day Event: Advancing Neurodegenerative Disease Research

Wednesday, February 25, 2026: In collaboration with the Termeer Institute, we will host our 11th Annual Rare Disease Day Event: Advancing Neurodegenerative Disease Research focused on patient voices and the technological innovations driving discovery. The 2026 program includes Broad Scientists, Termeer Fellows and Mentors, and Patient Advocates. We hope you’ll join us!

To register for this event, please visit this link.

Save the Date!

L2C Accelerator Scientific Symposium

Tuesday, October 27, 2026: All L2C supported project teams will present exciting progress towards their scientific milestones at the upcoming L2C Accelerator Scientific Symposium on Tuesday, October 27, 2026 from 9:00am - 5:00pm ET.

Save the date and we’ll be in touch once registration is live!

We appreciate your support and want to hear from you! As we continue to grow our community, please join us by visiting our website or get in touch with us at L2C@broadinstitute.org.

We are sending you our warmest wishes for the holiday season and cannot wait to see what 2026 will bring!

Best wishes,

The L2C Accelerator Team