| Date | Product | Disease | Phase | Company | Therapeutic area |
|---|---|---|---|---|---|
| 2022-05-16 | ATA-100 | limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) | 1-2 | Atamyo Therapeutics (France) | Rare diseases - Genetic diseases - Neuromuscular diseases |
| 2022-04-13 | sepofarsen (formerly named QR-110) | CEP290-mediated Leber congenital amaurosis 10 (LCA10) | 2-3 | ProQR Therapeutics (The Netherlands) | Rare diseases - Genetic diseases - Ophthlalmological diseases |
| 2022-04-12 | StemCART | cartilage damage | preclinical | Cline Scientific (Sweden) | Regenerative medicine |
| 2022-04-11 | EDIT-101 | Leber congenital amaurosis 10 | 1-2 | Editas Medicine (USA - MA) | Rare diseases - Genetic diseases - Ophthlalmological diseases |
| 2022-04-08 | AFM24 | EGFR-expressing solid malignancies | 1 | Affimed (Germany) | Oncology |
| 2022-04-01 | leniolisib | activated phosphoinositide 3-kinase delta (PI3K?) syndrome (APDS) | 2-3 | Pharming (The Netherlands) | Immunological diseases |
| 2022-03-28 | MaaT013 | steroid-resistant gastrointestinal acute graft-versus-host disease (SR GI-aGvHD) | 3 | Maat Pharma (France) | Transplantation |
| 2022-03-28 | MIC-Lx cell treatment | 2b | TolerogenixX (Germany) | ||
| 2022-03-23 | apraglutide | acute graft-versus-host disease (aGVHD) | preclinical | Vectivbio (Switzerland) | Transplantation |
| 2022-03-22 | ATR-258 | type 2 diabetes | 1 | Atrogi | Metabolic diseases |
| 2022-03-02 | LX1001 | Alzheimer's disease | 1-2 | Lexeo Therapeutics (USA-NY) | Neurodegenerative diseases |
| 2022-02-16 | LX2006 | Friedreich's ataxia cardiomyopathy | 1-2 | Lexeo Therapeutics (USA-NY) | Rare diseases - Genetic diseases -Neurodegenerative diseases - Cardiovascular diseases |
| 2021-12-21 | ADVM-022 | wet age-related macular degeneration (wet AMD) | I | Adverum Biotechnologies (USA - CA) | Ophtalmological diseases |
| 2021-11-15 | gene therapy based on E2F4DN | Alzheimer's disease | preclinical | Tetraneuron | Neurodegenerative diseases |
| 2021-10-20 | apraglutide | acute graft-versus-host disease | 2 | Vectivbio (Switzerland) | Transplantation |
| 2021-06-15 | apraglutide | short bowel syndrome with intestinal failure (SBS-IF) | 2 | Vectivbio (Switzerland) | Inflammatory diseases - Gastrointestinal diseases |
| 2021-05-14 | cotoretigene toliparvovec (BIIB112) | X-linked retinitis pigmentosa (XLRP) | 2-3 | Biogen (USA - MA) | Rare diseases - Genetic Diseases - Ophthalmological diseases |
| 2021-02-03 | apraglutide | short bowel syndrome | 3 | Vectivbio (Switzerland) | Inflammatory diseases - Gastrointestinal diseases |
| 2020-01-09 | GEM103 | geographic atrophy (GA) secondary to dry AMD. | 1 | Gemini Therapeutics (USA) | Ophthalmological diseases |
| 2019-12-07 | SB-525 | hemophilia A | 1-2 | Sangamo Therapeutics (USA - CA) | Rare diseases - Genetic diseases - Hematological diseases |
