Converging on Cures:
Nodal Biology meets
Transformative Technology

3RD ANNUAL LADDERS TO CURES SYMPOSIUM

OCTOBER 27, 2026

8:30AM - 6:00PM ET

BROAD INSTITUTE OF MIT AND HARVARD

AND STREAMING ONLINE

REGISTER NOW!

9:10 - 9:55am

SESSION 1 | Scaling Rare Disease Therapeutics: Disease-Agnostic Editing, Patient Data, and Clinical-Stage Pipelines

9:10am Disease agnostic editing modalities | David Liu (Broad Institute)

9:35am Patient Data as a Drug Development Engine: Lessons from FOXG1 | Nasha Fitter (Citizen Health)

9:55 - 10:30am PANEL 1 | Advancing Clinical-Stage Assets

Moderator David Meeker (Rhythm Pharmaceuticals)

Panelists: Meg Alexander (Ovid Therapeutics), David Goldstein (Actio Biosciences), Winston Yan (N=1; Center for Therapeutic Genetics)

10:30 - 10:45 am Coffee Break/Networking

10:45 - 11:30am

SESSION 2 | AI-Driven Target Discovery and Drug Design Chair: Caroline Uhler (Broad)

10:45 - 11:05am Optical Pooled Screening and AI-driven Target Discovery | Olivia Wiles (Google Deep Mind) + Matt Brown (Broad Institute)

11:05 - 11:30am From Target Discovery to Clinical Impact | Andrew Adams (Eli Lilly)

11:30 - 11:55am

SESSION 3 | Metabolic Nodes as Therapeutic Targets in Mitochondrial Disease Chair: Jillian Shaw

11:30 - 11:55am Hypoxia as a Nodal Therapeutic Strategy for Mitochondrial Diseases | Vamsi Mootha

11:55 - 1:00 pm Lunch/Networking

1:00 - 2:00pm

SESSION 4 | Mechanism-Driven Small-Molecule Discovery Chair: Raj Gupta

1:00 - 1:20pm Small-molecule Discovery for Microvillus Inclusion Disease |

Jay Thiagarjah (BCH/Tools-to-Therapies Investigator)

1:20 - 1:40pm Scalable Screening Approaches for Rare Pediatric Kidney Disease |

Magdalena Riedl Khursigara (Broad Institute; Tools-to-Therapies Investigator)

1:40 - 2:00pm Generalizable approaches to Collagenopathies: proteostasis as a nodal target across connective tissue disorders | Christina Jacobsen (BCH)

2:00 - 2:40pm

SESSION 5 | Scalable Cell Platforms for Disease Modeling and Screening

Chair: Stacey Gabriel (Broad Institute)

2:00 - 2:20pm Cellular Barcoding at Scale: Mapping Rare Cardiovascular Disease Mechanisms with iPSCs

Sarah Morton (BCH/Broad Institute)

2:20 - 2:40pm CellWorks: Cell Line Engineering and High-Throughput Screening |

Sarra Merzouk (Broad Institute/Tools-to-Therapies/CellWorks)

2:40 - 3:00pm Coffee + Networking Break

3:00 - 3:15pm CNBC Cures: Becky Quick in Conversation with Anna Greka

3:15 - 3:50pm

PANEL 2 | The Reimbursement Gap: Can the Economics of Rare Diseases Keep Pace with

Scientific Discovery?

Moderator: Annie Kennedy (RARE Foundation), Panelists: Andrew Lo (MIT), William Shrank (Aradigm), Duncan McKechnie, Ekemini Riley (Coalition for Aligning Science)

3:50 - 4:25pm

PANEL 3 | Clearing the Path: How Advocates and Regulators Get Rare Disease Therapies Approved

Moderator: Charlene Son Rigby (Global Genes)

Panelists: Cat Lutz (Jackson Laboratory), Terry Pirovolakis (Elpida Therapeutics), Sunitha Malepati

4:25 - 4:30pm Concluding Remarks | Anna Greka

4:30 - 6:00pm Poster Session, Reception, Networking

8:30am Registration

8:50am Opening Remarks | Todd Golub

9:00am State of the L2C Scientific Accelerator | Anna Greka and Jillian Shaw

Learn More

Speakers

  • Andrew Adams, PhD

    Eli Lilly and Company

  • Christina Jacobsen, MD, PhD

    Boston Children’s Hospital

  • David Liu, PhD

    Broad Institute

  • Magdalena Riedl Khursigara, MD, PhD

    Broad Institute

  • Nasha Fitter, MBA

    Citizen Health

  • Sarah Morton, MD, PhD

    Boston Children’s Hospital

  • Vamsi Mootha, MD

    Broad Institute

  • Annie Kennedy

    RARE Foundation

  • Sunitha Malepati

    Renaissance Philanthropy

FAQs

  • After you complete your registration to attend the symposium, please apply to present a poster here.

    Deadline to apply for the poster session is Tuesday, October 20, 2026.

  • Paid parking is available in the Kendall Center Yellow Garage (75 Ames St, Cambridge, MA 02142) and the Kendall Center Green Garage (90 Broadway, Cambridge, MA 02142). Limited, metered street parking is available nearby.

    The nearest MBTA station is the Kendall Square stop on the Red Line.

    1. Log in to Eventbrite: Go to Eventbrite and log into your account using the email address you registered with.

    2. Navigate to Tickets: Click on your profile icon in the top right corner and select Tickets from the dropdown menu (or go directly to eventbrite.com/tickets).

    3. Select Your Order: Find our event in your list of upcoming events and click on it to view your order details.

    4. Cancel Order: Click Cancel Order (usually located on the left side). Follow the prompts to confirm. You will receive an email confirmation once the cancellation is complete.

  • You can reach the L2C Accelerator Team via our contact page or email us directly at <l2c@broadinstitute.org>.

FAQs