Diseases (100)
| Indication | Programs | Companies | Trials |
|---|
| Pompe | 82 | 79 | 68 |
| NB | 78 | 75 | 76 |
| LGS | 74 | 69 | 72 |
| FSGS | 73 | 72 | 45 |
| Endometrial Ca | 72 | 68 | 66 |
| HS | 69 | 67 | 64 |
| Celiac | 69 | 66 | 58 |
| Schizophrenia | 68 | 65 | 53 |
| Bladder Ca | 67 | 66 | 74 |
| PBC | 67 | 65 | 53 |
| Heart Failure | 66 | 63 | 54 |
| PNH | 66 | 65 | 48 |
| RB | 66 | 63 | 50 |
| SMA | 65 | 62 | 62 |
| MG | 65 | 64 | 68 |
| MCL | 65 | 61 | 59 |
| LN | 65 | 62 | 62 |
| Parkinson's | 64 | 63 | 59 |
| Migraine | 64 | 64 | 58 |
| OCD | 64 | 63 | 65 |
| MS | 63 | 62 | 52 |
| RSV | 63 | 62 | 39 |
| IPF | 62 | 58 | 67 |
| FTD | 62 | 61 | 75 |
| CLL | 61 | 60 | 53 |
| Psoriasis | 61 | 60 | 59 |
| HNSCC | 61 | 59 | 49 |
| HCC | 61 | 59 | 49 |
| GBM | 61 | 55 | 53 |
| CF | 61 | 58 | 49 |
| CKD | 61 | 60 | 56 |
| MDS | 60 | 58 | 49 |
| AS | 60 | 58 | 46 |
| Thymoma | 60 | 60 | 55 |
| MM | 59 | 55 | 55 |
| T2D | 59 | 58 | 56 |
| NMOSD | 59 | 58 | 64 |
| ADPKD | 59 | 58 | 66 |
| Ovarian Ca | 58 | 58 | 53 |
| MDD | 58 | 58 | 50 |
| TTR Amyloidosis | 58 | 56 | 36 |
| Fabry | 58 | 55 | 46 |
| Rett | 58 | 57 | 56 |
| Wilms | 58 | 53 | 56 |
| Prostate Ca | 57 | 56 | 55 |
| Crohn's | 57 | 56 | 45 |
| FL | 57 | 55 | 46 |
| Urothelial Ca | 57 | 52 | 43 |
| Angelman | 57 | 55 | 40 |
| CSU | 57 | 57 | 53 |
| EoE | 57 | 57 | 46 |
| MCC | 57 | 55 | 44 |
| Ewing Sarcoma | 57 | 54 | 59 |
| DLBCL | 56 | 55 | 55 |
| SCD | 56 | 54 | 57 |
| Hemophilia A | 56 | 55 | 57 |
| SCLC | 56 | 53 | 47 |
| Huntington's | 56 | 55 | 61 |
| Dravet | 56 | 54 | 47 |
| CML | 56 | 53 | 56 |
| NSCLC | 55 | 55 | 44 |
| Melanoma | 55 | 55 | 69 |
| RA | 55 | 52 | 57 |
| GA | 55 | 52 | 52 |
| PsA | 55 | 53 | 58 |
| PSP | 55 | 53 | 49 |
| CTCL | 55 | 53 | 51 |
| Meso | 55 | 55 | 42 |
| NASH | 54 | 52 | 47 |
| Asthma | 54 | 51 | 45 |
| PV | 54 | 53 | 53 |
| PAH | 54 | 52 | 51 |
| ADHD | 54 | 53 | 40 |
| Cervical Ca | 54 | 54 | 57 |
| ACC | 54 | 52 | 46 |
| CRC | 53 | 52 | 50 |
| SLE | 53 | 52 | 42 |
| Obesity | 53 | 52 | 44 |
| ALL | 53 | 51 | 49 |
| Gastric Ca | 52 | 52 | 43 |
| AML | 51 | 50 | 46 |
| UC | 51 | 50 | 45 |
| Epilepsy | 51 | 49 | 44 |
| BCC | 51 | 49 | 47 |
| RCC | 50 | 47 | 46 |
| GIST | 50 | 47 | 43 |
| Cholangiocarcinoma | 49 | 46 | 43 |
| Breast Ca | 48 | 47 | 47 |
| DMD | 48 | 44 | 37 |
| ALS | 48 | 46 | 41 |
| Myelofibrosis | 48 | 47 | 34 |
| IgAN | 47 | 46 | 41 |
| ET | 47 | 47 | 43 |
| WM | 47 | 46 | 45 |
| Pancreatic Ca | 46 | 46 | 42 |
| Wet AMD | 46 | 45 | 35 |
| Narcolepsy | 46 | 44 | 34 |
| Atopic Derm | 43 | 43 | 40 |
| PTSD | 43 | 43 | 35 |
| Alzheimer's | 35 | 35 | 35 |