1. the cause of the disease is well-defined
2. there is high unmet need, there are no approved therapies, or there are significant shortcomings with existing treatment paradigms
Cerebral Cavernous Malformation
Neurofibromatosis Type 2
Familial Adenomatous Polyposis
GM2 Gangliosidosis
Clostridium difficile Colitis
KRAS/STK11-driven checkpoint resistance
MYC-driven oncology
Cancer immunotherapy target alpha
Cancer Immunotherapy Target Beta
Hepatocellular carcinoma
HRD-negative ovarian cancer target gamma
Ovarian cancer
Charcot-Marie-Tooth Disease Type 2A
Batten Disease
Several dozen early discovery programs
Neurofibromatosis Type 2
Cerebral Cavernous Malformation
GM2 Gangliosidosis
Solid Tumors
Dilated Cardiomyopathy
Clostridium difficile Colitis
Neuroinflammation
Batten Disease
Charcot-Marie-Tooth Disease
Pulmonary Arterial Hypertension
Lung Disease
Senescence
Vascular Disease
+ 18 early
discovery programs
Oncology 1
Oncology 2
Neurofibromatosis Type 2
Cerebral Cavernous Malformation
GM2 Gangliosidosis
*Undisclosed indication
Metabolic Disorders
Tumor Suppression
Cancers
Mitochondrial Diseases
Lysosomal Storage Disorders
Nervous System Disorders
Neurodevelopment Disorders
Neurodegenerative Disorders
Inflammation
Lung Disease
Senescence
Vascular Dysplasia
Tumor Suppression
Dialated Cardiomyophathy
Clostridium Difficile Colitis
Amitrophic Lateral Sclerosis
Batten Disease
Charcot-Marie-Tooth Disease
Pulmonary Arterial Hypertension
Neurofibromatosis Type 2
Cerebral Cavernous Malformation
*Undisclosed indication