Massachusetts Institute of Technology

The Ohio State University

Translate Bio, Inc.

Shire Human Genetic Therapies, Inc.

Yizhou Dong

Daniel Griffith Anderson

Robert Samuel Langer

Michael W Heartlein

Frank DeRosa

Delai Chen

Akinleye C Alabi

Bin Li

Yi Heng Chen

Kevin Thomas Love

Arturo Jose Vegas

Yunlong Zhang

Xinfu Zhang

Darrell J Irvine

Ron Weiss

Karl Dane Wittrup

Joseph R Dorkin

Yingzhong Li

Noor Momin

Omid Veiseh

Eric Andrew Appel

Hao Yin

Matthew J Webber

Weiheng Wang

Benjamin C Tang

Kun Xue

Xiao Luo

Ahmed A Eltoukhy

The present disclosure relates to synthetic oncolytic viruses comprising a lipid nanoparticle comprising one or more types of lipid and a self-amplifying replicon RNA comprising a sequence that encodes an immunomodulatory molecule.

Synthetic oncolytic LNP-replicon RNA and uses for cancer immunotherapy

The present disclosure generally relates to genome editing systems and methods and compounds and compositions for use in Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) genome editing systems. Disclosed herein are modified nucleic acids that modulate the activity of genome editing.

Modified nucleic acids, hybrid guide RNAS, and uses thereof

The present disclosure generally relates to systems, methods and compositions for use in Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cpf1 genome editing systems.

Engineered guide RNA and uses thereof

The present disclosure generally relates to systems, methods and compositions for use in Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cpf1 genome editing systems. Disclosed herein are modified Cpf1 mRNAs, modified guide RNAs, and combinations thereof, that confer increased levels of genome editing.

Modified Cpf1 MRNA, modified guide RNA, and uses thereof

The present disclosure relates to biomimetic nanomaterials, compounds, compositions, and methods for delivery of therapeutic, diagnostic, or prophylactic agents (for example, a nucleic acid).

Biomimetic nanomaterials and uses thereof

The present invention provides, among other things, methods of delivering mRNA in vivo, including administering to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome such that the administering of the composition results in the expression of the protein encoded by the mRNA in vivo, wherein the liposome comprises a cationic lipid of formula I-c: or a pharmaceutically acceptable salt thereof.

Lipid formulations for delivery of messenger RNA

Modified nucleic acids, hybrid guide RNAs, and uses thereof

The present disclosure relates to benzene-1,3,5-tricarboxamide derived ester lipid compounds, compositions, lipid-like nanoparticles, and methods for delivery of therapeutic, diagnostic, or prophylactic agents.

Benzene-1,3,5-tricarboxamide derived ester lipids and uses thereof

Described herein are compounds and compositions characterized, in certain embodiments, by conjugation of various groups, such as lipophilic groups, to an amino or amide group of an amino acid, a linear or cyclic peptide, a linear or cyclic polypeptide, or structural isomer thereof, to provide compounds of the present invention, collectively referred to herein as 'APPLs'. Such APPLs are deemed useful for a variety of applications, such as, for example, improved nucleotide delivery. Exemplary APPLs include, but are not limited to, compounds of Formula (I), (II), (III), (IV), (V), and (VI), and salts thereof, as described herein: wherein m, n, p, R', R, R, R, R, R, R, Z, W, Y, and Z are as defined herein.

Amino acid-, peptide- and polypeptide-lipids, isomers, compositions, and uses thereof

The present disclosure provides brush-poly(glycoamidoamine)-lipids (PGALs) (e.g., polymers of any one of Formulae (I)-(IV)) and methods of preparing the PGALs. A described PGAL may include poly(glycoamidoamine)-derived moieties (e.g., such as which may assist the PGAL and/or a complex of the PGAL and an agent to pass through cell membranes or be taken up by cells. Also provided are compositions including a described PGAL and an agent (e.g., polynucleotide, small molecule, peptide, or protein). The present disclosure also provides methods, kits, and uses that include or involve the PGALs or compositions for delivering an agent to a subject, tissue, or cell and/or for treating and/or preventing in a subject a range of diseases, such as genetic diseases, proliferative diseases, hematological diseases, neurological diseases, immunological diseases, gastrointestinal diseases, respiratory diseases, painful conditions, psychiatric disorders, musculoskeletal diseases, genitourinary diseases, and metabolic disorders 

Brush-poly (glycoamidoamine)-lipids and uses thereof

Described herein are polymers, polymeric gels, or a composition thereof, for drug delivery. The polymers, which include boronic-acid containing moieties (e.g., and polyol-containing moieties (e.g., are prepared by free-radical polymerization and can self-assemble into polymeric gels such as hydrogels. Also provided are methods or preparing the polymers, kits involving the polymers and/or polymeric gels or a composition thereof, for use as materials or delivery applications of an agent to a subject.

Polymers, hydrogels, and uses thereof

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Dublin, OH, United States of America

Yizhou Dong