The Brigham and Women's Hospital, Inc.

Massachusetts Eye and Ear Infirmary

Sapphiros Ai Bio LLC

Dana-Farber-Cancer Institute Inc.

Yuma Therapeutics, Inc.

Duke University

The Ohio State University

Indiana University

Aquinnah Pharmaceuticals, Inc.

Orig3n, Inc.

University of Massachusetts

Marcie A Glicksman

Gregory D Cuny

Lixin Qiao

Albert Edge

Sang-Jun Jeon

Ross L Stein

Kathleen Seyb

Robin Young Smith

Jun Xian

Min Huang

Alan D D'Andrea

Kevin J Hodgetts

David Wilson

Steven L Mathieu

David Cohen

Yukari Y Perrella

Neil Wagle

Ekaterina Y Shishova

Vincent Darmency

Hrvoje Lusic

April Case

Elliot J Androphy

Donald C Lo

Chien-Liang Glenn Lin

Xeuchao Xing

Benjamin Wolozin

Justin Boyd

Jonathan Cherry

Nikhat F Zaidi

Thomas Gainer

Sungwoon Choi

Alan D D'andrea

Jonathon J Cherry

Reserves of immortalized genetic material are stored in a bank for providing a resource (e.g., artificial gametes) for couples (e.g., same sex couples) to produce biologically-related children. The reserves provide the ability to derive sperm from induced pluripotent stem cells (iPSCs) of one partner and/or eggs from iPSCs of the other partner. For example, a biological sample is stored as iPSCs that can be used to generate an unlimited supply of genetic material (e.g., artificial gametes for conception) when needed by a user (e.g., a year or more after the sample is provided, e.g., 5 years or more after the sample is provided). Such a bank is helpful for an individual in a same sex (or infertile) couple who desires to have biological children at some point during his/her lifetime, but does not plan to have children in the immediate timeframe, for example.

Systems and methods of assisted reproduction and prevention of genetic defects in offspring using induced pluripotent stem cells

Described herein are various systems and methods for customizing cell culture media for optimized cell proliferation based on genetic traits of the cells. Factors of the cells, such as genetics, can determine the optimum cell culture conditions for growth and use of individual cells in vitro. In certain embodiments, genetics can determine sensitivity from a toxic perspective. In certain embodiments, genetics can determine what concentration(s) of growth factor(s) and nutrient(s) are required for optimum growth.

Systems and methods for customizing cell culture media for optimized cell proliferation based on genetic traits of cells

Described herein are various systems, methods, and apparatus for systematic creation of isolated homogeneous colonies of cells from vector-based lineages. The vector-based lineages may originate from multiple types of viral vector families (e.g., Paramyx-oviridae, Retroviridae, Parvoviridae) or non-natural engineered vectors or a plurality of vector combinations, for example. In certain embodiments, the isolated homogeneous colonies of cells are vector-free sub-colonies; in other embodiments, the isolated homogeneous colonies of cells are homogeneous vector sub-colonies. In other embodiments, vector mixed sub-colonies are created. The disclosed systems, methods, and apparatus are useful for inducible pluripotent stem cell (iPSC) production and work by selectively binding to one or more corresponding protein markers expressed on the surface of a cell that indicate that cellular reprogramming has occurred. Software is used to automate the purification and isolation of the iPSCs produced.

Systems, methods, and apparatus for induced pluripotent stem cell isolation and combinatorial production

This invention generally provides compounds, pharmaceutical compositions, and methods for their use, which include methods that result in increased expression in an Atoh1 gene (e.g., Hath1) in a biological cell. More specifically, the invention relates to the treatment of diseases and/or disorders that would benefit from increased Atoh1 expression, e.g., a hearing impairment or imbalance disorder associated with a loss of auditory hair cells, or a disorder associated with abnormal cellular proliferation.

Compounds that enhance Atoh1 expression

The present invention relates to 2-amino-4-arylpyrimidine and 2-amino-4-aryltriazine compounds as inhibitors of heat shock protein 90 family of chaperone proteins. The invention also features pharmaceutical compositions and kits that include the compounds and compositions of the invention. The invention further relates to the medical use of these compounds and compositions for the treatment of a disorder in a subject. For example, the disorder is a neurodegenerative disease.

Pyrimidine compounds and methods using the same

Disclosed are methods for diagnosing Parkinson's disease, or identifying a risk of developing Parkinson's disease, comprising measuring the amount of a biomolecule in a blood sample, liver sample, or hepatocyte. Also disclosed are methods for preventing or treating Parkinson's disease, comprising administering a therapeutically effective plurality of hepatocytes to a subject in need thereof.

Diagnosis and treatment of parkinson's disease based on identification and amelioration of liver dysfunction

Provided are small molecule inhibitors of ubiquitin specific protease 1 (USP1) activity and methods for their use in treating and characterizing cancers. The small molecule USP1 inhibitors of the invention are particularly useful in the treatment of cancers that are resistant to DNA cross-linking agents.

Small molecule inhibitors of USP1 deubiquitinating enzyme activity

This invention relates to compounds of Formulas I, II, and III, and their use as inhibitors of phosphatidylcholine transfer protein (PC-TP). The invention further relates to pharmaceutical compositions and methods of treatment of disorders related to the inhibition of PC-TP using the compounds of Formulas I, II, and III. Such disorders include obesity and disorders associated with obesity.

Phosphatidylcholine transfer protein inhibitors

Pyridazine derivatives that activate the excitatory amino acid transporter 2 (EAAT2), and methods use thereof for treating or preventing diseases, disorders, and conditions associated with glutamate excitotoxicity.

Pyridazine derivatives as EAAT2 activators

Herein, methods of modulating inclusion formation and stress granules in cells are described. The methods comprise contacting a cell with an inclusion inhibitor. Methods for screening for modulators of TDP-43 aggregation are also described.

Identification of compounds that disperse TDP-43 inclusions

Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.

Screening methods for spinal muscular atrophy

Growing community of inventors

Boston, MA, United States of America

Marcie A Glicksman