Cellarity, Inc.

Flagship Pioneering Innovations Vi, LLC

Ragy Haddad

Atli Thorarensen

Na Li

Nicholas McCartney Plugis

Fabian Alexander Wolf

Mauricio Cortes

Robert Brian Nicewonger

Kristen Marino

David Zhang

Colin Diner

Samuel Miller

Frank Lovering

Ravi Ujjinamatada

Juan Corchado Garcia

Diogo Camacho

The present disclosure provides DCN-1 modulating compounds, pharmaceutically acceptable salts thereof, pharmaceutical compositions, and their use for treating sickle cell disorders, diseases, and conditions. Such compounds are of Formula I: or a pharmaceutically acceptable salt thereof.

DCN-1 modulating compounds and methods of use thereof

Systems and methods of associating a test compound with a reference compound are provided. A fingerprint of a chemical structure of the test compound is obtained. Abundance values for a set of cellular constituents are also obtained, from one or more reference assays, across a plurality of cells that have been exposed to a control solution free of the test compound. The fingerprint and the abundance values are inputted into a model that outputs a predicted similarity between (i) a predicted perturbational effect of the test compound across the set of cellular constituents and (ii) a measured cell-based perturbational effect of the reference compound across the set of cellular constituents. When the predicted similarity achieves a threshold similarity, the test compound is associated with the reference compound.

Systems and methods for predicting compounds associated with transcriptional signatures

Systems and methods for associating a compound with physiological conditions are provided. A fingerprint of a compound chemical structure is obtained and inputted to a model that outputs one or more calculated activation scores. Each activation score represents a cellular constituent module in a set of modules, where each module includes a subset of cellular constituents and a first module in the set of modules is associated with the physiological condition. When the activation score for the first module satisfies a threshold criterion, the compound is identified as associated with the physiological condition. In some aspects, each activation score represents a perturbation signature associated with the physiological condition and the compound is identified when the activation score for a first perturbation signature satisfies a threshold criterion. Systems and methods for training a model that associates compounds with physiological conditions are also provided.

Systems and methods for associating compounds with physiological conditions using fingerprint analysis

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Cambridge, MA, United States of America

Ragy Haddad