Bial - R&d Investments, S.a.

Lysosomal Therapeutics Inc.

Bial-biotech Investments, Inc.

Massachusetts Institute of Technology

Children's Hospital

Link Medicine Corporation

Peter T Lansbury

Renato T Skerlj

Elyse Marie Josee Bourque

Andrew Charles Good

William J Greenlee

Richard B Silverman

Dimitri Krainc

Jianbin Zheng

Robert Samuel Langer

Mark E Duggan

Joseph P Vacanti

James Douglas Harper

Alan L Davison

Peter Lin

Robert S Langer

Hogyu Han

Robin Kate Meray

Ross A Fredenburg

Craig J Justman

Cheon-Gyu Cho

Weiguo Zhen

Denise Barrera

Elyse Marie Josée Bourque

The invention provides substituted pyrazolo[1,5-a]pyrimidinyl carboxamide and related organic compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted pyrazolo[1,5-a]pyrimidinyl carboxamide compounds described herein include 2-heterocyclyl-4-alkyl-pyrazolo[1,5-a]pyrimidine-3-carboxamide compounds and variants thereof. The invention provides, in part, a compound of Formula (IV): or a pharmaceutically acceptable salt thereof, wherein the variables are as defined herein.

Substituted pyrazolo[1,5-a]pyrimidines as glucocerebrosidase activators

The invention provides substituted pyrazolo[1,5-a]pyrimidine and related organic compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted pyrazolo[1,5-a]pyrimidine compounds described herein include 5,7-dimethyl-N-phenylpyrazolo[1,5-a]pyrimidine-3-carboxamide compounds and variants thereof.

Substituted pyrazolo[1,5-a]pyrimidines and their use in the treatment of medical disorders

The invention provides substituted imidazo[1,2-b]pyridazine compounds, substituted imidazo[1,5-b]pyridazine compounds, related compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted imidazo[1,2-b]pyridazine compounds described herein include substituted imidazo[1,2-b]pyridazine-3-carboxamide compounds and variants thereof.

Substituted imidazo[1,2-b]pyridazines, substituted imidazo[1,5-b]pyridazines, related compounds, and their use in the treatment of medical disorders

The invention provides substituted imidazo[1,5-a]pyrimidinyl carboxamide and related organic compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted imidazo[1,5-a]pyrimidinyl carboxamide compounds described herein include substituted 2-heterocyclyl-4-alkyl-imidazo[1,5-a]pyrimidine-8-carboxamide compounds and variants thereof.

Imidazo[1,5-a]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders

The invention provides substituted imidazo[1,5-a]pyrimidines and related organic compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted imidazo[1,5-a]pyrimidine compounds described herein include substituted 2,4-dimethyl-N-phenylimidazo[1,5-a]pyrimidine-8-carboxamide compounds and variants thereof.

Substituted imidazo[1,5-a]pyrimidines and their use in the treatment of medical disorders

The invention provides substituted pyrrolo[1,2-a]pyrimidines and related organic compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted pyrrolo[1,2-a]pyrimidines compounds described herein include substituted 2,4-dimethyl-N-phenylpyrrolo[1,2-a]pyrimidine-8-carboxamide compounds and variants thereof.

Substituted pyrrolo[1,2-a]pyrimidines and their use in the treatment of medical disorders

The invention provides substituted imidazo[1,2-a]pyridine compounds, substituted imidazo[1,2-a]pyrazine compounds, related compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary imidazo[1,2-a] pyridine compounds described herein include substituted 5-methyl imidazo[1,2-a]pyridine-8-carboxamide compounds and variants thereof.

Substituted imidazo[1,2-a]pyridines, substituted imidazo[1,2-a]pyrazines, related compounds, and their use in the treatment of medical disorders

The invention provides substituted pyrazolo[1,5-a]pyrimidinyl carboxamide and related organic compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted pyrazolo[1,5-a]pyrimidinyl carboxamide compounds described herein include 2-heterocyclyl-4-alkyl-pyrazolo[1,5-a]pyrimidine-3-carboxamide compounds and variants thereof. The invention provides, in part, a compound of Formula (I): or a pharmaceutically acceptable salt thereof, wherein the variables are as defined herein.

Substituted pyrazolo[1,5-a]pyrimidines for the treatment of medical disorders

The invention provides substituted pyrrolo[1,2-a]pyrimidinyl carboxamide and related organic compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted pyrrolo[1,2-a]pyrimidinyl carboxamide compounds described herein include substituted 2-heterocyclyl-4-alkyl-pyrrolo[1,2-a]pyrimidine-8-carboxarnide compounds and variants thereof.

Pyrrolo[1,2-A]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders

The invention provides substituted pyrazolo[1,5-a]pyrimidinyl carboxamide and related organic compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted pyrazolo[1,5-a]pyrimidinyl carboxamide compounds described herein include 2-heterocyclyl-4-alkyl-pyrazolo[1,5-a]pyrimidine-3-carboxamide compounds and variants thereof. The invention provides, in part, a compound of formula (I-A): or a pharmaceutically acceptable salt thereof, wherein the variables are as defined herein.

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Brookline, MA, United States of America

Peter T Lansbury