Forma Therapeutics, Inc.

Biogen Ma Inc.

Valo Health, Inc.

Forma Tm2, Inc.

Valo Early Discovery, Inc.

Q32 Bio Inc.

Nimbus Wadjet, Inc.

Camp4 Therapeutics Corporation

Justin Andrew Caravella

Zhongguo Wang

David R Lancia, Jr

Ann-Marie Campbell

Anna M Ericsson

Jian Lin

Gary Gustafson

R Bruce Diebold

Susan Ashwell

Wei Lu

Stephanos Ioannidis

David Joseph Guerin

Alexandre Joseph Buckmelter

Minghua Wang

Bruce Follows

Hongbin Li

David James Richard

Steven Gregory Mischke

Tatiana Shelekhin

Pui Yee Ng

Frederick Robbins Taylor

Adam Charles Talbot

Shawn E R Schiller

Darby Schmidt

Angela V West

Edward L Fritzen

Kenneth W Bair

Matthew W Martin

Susan L Kalled

Shelia Marie Violette

Vernon Michael Holers

Michael Storek

Michael Steven Curtis

Ellen Garber Stark

Kelly C Fahnoe

Cheng Ran Huang

Robert L Dow

Mary-Margaret Zablocki

Derun Li

Silvana Marcel Leit De Moradei

Kenneth J Simon

Mia Marie Rushe

Thomas O Cameron

Nikolay Sitnikov

Nathan E Genung

Florian Bartels

Alex J Buckmelter

David R Lancia

Bingsong Han

Cuixian Liu

David A Bumcrot

Alexey Alexandrovich Lugovskoy

Ellen A Garber

Deping Wang

Alfica Sehgal

Scott Glaser

Brian Miller

Jennifer R Downing

Joseph Arndt

Tigran Aivazian

Subhadeep Roy

Ellen A Garber Stark

Scot Mente

Yun Joon Jung

Susan Foley

Yuchun Guo

Bryan J Matthews

Yuting Liu

Rachana S Kelkar

Mario Esteban Contreras Gamboa

Rutuja Sudhakar Pai

Dania Mounir Rabah

Amna Saeed-Kothe

George Campbell Kaynor

Eric Day

Xiaoping Hronowski

Brian Robert Miller

David Lancia

Anna Ericsson

David R Lancia Jr

Shawn ER Schiller

Jr David R Lancia

Tigran Dikran Aivazian

The present disclosure is directed to compounds of Formula I: and pharmaceutically acceptable salts thereof, and compositions thereof, as well as methods of treatment of cancers such as those involving WRN protein.

WRN inhibitors

The present disclosure is directed to compounds of Formula II-e: and pharmaceutically acceptable salts thereof, and compositions thereof, as well as methods of treatment of cancers such as those involving WRN protein.

Described herein are methods of modulating gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for modulating the levels of gene products, for example, increasing expression of Carbamoyl-Phosphatase Synthetase 1 (CPS1), thereby treating diseases associated with aberrant gene expression.

Modulation of gene transcription using antisense oligonucleotides targeting regulatory RNAs

The invention relates to inhibitors of mutant isocitrate dehydrogenase (mt-IDH) proteins with neomorphic activity useful in the treatment of cell-proliferation disorders and cancers, having the Formula: where A, U, W, W, W, R-R, and Rare described herein.

Pyridin-2(1H)-one quinolinone derivatives as mutant-isocitrate dehydrogenase inhibitors

The present disclosure relates to modulators, such as inhibitors, of at least one pathway chosen from USP28 and USP25, pharmaceutical compositions comprising the inhibitors, and methods of using the inhibitors. The modulators, such as inhibitors, of at least one pathway chosen from USP28 and USP25 can be useful in the treatment of cancers, among other ailments. The present disclosure provides compounds of Formula (I).

Inhibiting deubiquitinase USP25 and USP28

The present disclosure relates to modulators, such as inhibitors, of at least one pathway chosen from USP28 and USP25, pharmaceutical compositions comprising the inhibitors, and methods of using the inhibitors. The modulators, such as inhibitors, of at least one pathway chosen from USP28 and USP25 can be useful in the treatment of cancers, among other ailments.

Carboxamides as ubiquitin-specific protease inhibitors

The application relates to inhibitors of USP1 useful in the treatment of cancers, and other USP1 associated diseases and disorders, having the Formula: where R, R, R, R, R, R, X, X, X, X, and n are described herein.

Purinones as ubiquitin-specific protease 1 inhibitors

The invention relates to inhibitors of USP7 inhibitors useful in the treatment of cancers, neurodegenerative diseases, immunological disorders, inflammatory disorders, cardiovascular diseases, ischemic diseases, viral infections and diseases, and bacterial infections and diseases, having the Formula: where m, n, X, X, R-R, Rand Rare described herein.

Pyrrolo and pyrazolopyrimidines as ubiquitin-specific protease 7 inhibitors

Provided herein are fusion protein constructs that can bind a complement-associated antigen, comprising a targeting moiety and a complement modulator protein, or a fragment thereof or a variant thereof. The targeting moiety is an antibody or an antigen binding fragment thereof, in some examples. Further provided are methods of using the fusion protein constructs, for example, in treating complement mediation conditions.

Methods of treating complement mediated diseases with fusion protein constructs comprising anti-C3d antibody and a complement modulator

The disclosure relates to USP30 Inhibitor Compounds, pharmaceutically acceptable salts thereof, pharmaceutical compositions comprising same, and medical uses involving same.

Fused pyrrolines which act as ubiquitin-specific protease 30 (USP30) inhibitors

The disclosure relates to USP30 Inhibitor Compounds I, pharmaceutically acceptable salts thereof, pharmaceutical compositions comprising same, and medical uses involving same. (I) 

The disclosure relates to inhibitors of USP28 and/or USP25 useful in the treatment of cancers, inflammation, autoimmune diseases, and infectious diseases, having the Formula: (I), where R, R, R, R, R, R, R, X, and n are described herein. 

Thienopyrazine carboxamides as ubiquitin-specific protease inhibitors

The present disclosure relates to chemical entities useful as inhibitors of Ubiquitin Specific Protease 30 (USP30), pharmaceutical compositions comprising the chemical entities, and methods of using the chemical entities. The chemical entities as disclosed herein can be useful in the treatment of a disease, disorder, or condition involving mitochondrial dysfunction, including neurodegenerative diseases, motor neuron diseases, metabolic disorders, and cancers, among other ailments. Chemical entities disclosed herein include compounds of Formula (II): wherein A, R, R, R, R, R, R, R, R, and m are defined herein.

Inhibiting ubiquitin specific peptidase 30

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Cambridge, MA, United States of America

Justin Andrew Caravella