Last Update: Aug 5, 2023
| Company | Product | MOA | Indication | Approval |
|---|---|---|---|---|
| Novartis | Adakveo (crizanlizumab) | Anti-P-selectin mAb | Add-on or monotherapy to reduce the frequency of VOCs in SCD patients aged 16 and older. | 2019 (US), 2020 (EU, revoked 2023), 2021 (UK) |
| Global Blood Therapeutics | Oxbryta (voxelotor) | HbS stabiliser/polymerisation inhibitor | Treatment of SCD in adults and pediatric patients 4 years of age and older. Ages 12+ in UK. | 2019 (US), 2022 (EU), 2022 (UK) |
| Emmaus Life Sciences | Endari (L-glutamine) | Antioxidant/unclear | Reduce acute complications of SCD | 2017 (US) |
| Multiple | Hydroxyurea/ Hydroxycarbamide | Increase HbF production, immunomodulation | Reduce painful crises and blood transfusions in pts w/ SCA w/recurrent mod-severe painful crises | 1998 (US) |
| - | Allogeneic HSCT | Replace beta globin-defective stem cells | Severe SCD - eligible patients | - |
| Company | Candidate | MOA | Indications | Phase |
|---|---|---|---|---|
| Global Blood Therapeutics | Inclacumab | Anti P-selectin mAb | SCD - reduce frequency of VOCs | III |
| Forma Therapeutics | Etavopivat (FT-4202) | PKR activator | SCD - Hb+VOCs | II/III |
| Agios Pharma | Mitapivat | PKR activator | SCD, thalessemias | II/III |
| Global Blood Therapeutics | GBT601 | Next gen HbS polymerisation inhibitor | SCD - disease modifying | II/III |
| EpiDestiny/Novo Nordisk | EPI01 | DNMT1 inhibitor / increase expression of HbF | SCD | II |
| Fulcrum Therapeutics | FTX-6058 | EED inhibitor / increase expression of HbF | SCD | Ib |
| Agios Pharma | AG-946 | PKR activator | SCD, hemolytic anemias | I |
| CSL | CSL889 | Plasma-derived hemopexin | Treatment of VOC in SCD | I |
| Glycomimetics | GMI-1687 | E-selectin antagonist | SCD - reduce VOC | IND-C |
| Company | Candidate | MOA | Indications | Phase |
|---|---|---|---|---|
| Vertex/CRISPR Therapeutics | CTX001 (Exa-cel) | Ex vivo autologous CRISPR/Cas9 BCL11A edited HSC | Severe hemoglobinopathies (SCD) cure | BLA |
| Bluebird bio | LentiGlobin BB305 (bb1111) | Ex vivo autologous HSC lentiviral therapy delivering functional HBB | SCD cure | III, I/II |
| Sangamo/Bioverativ (Sanofi) | BIVV-003 | ZFN-modified autologous stem cells w/ disrupted BCL11A gene | SCD cure, B-thalessemia | I/II |
| Intellia Therapeutics | OTQ923 | Ex vivo HSC gene therapy to increase HbF expression | Severe SCD | I/II |
| Editas Medicine | EDIT-301 | Ex vivo HSC gene therapy to increase HbF expression | Severe SCD | I/II |
| Aruvant Sciences | RVT-1801 | Ex vivo autologous HSC lentiviral therapy delivering gamma-globin gene | SCD cure, beta-thalessemia | I |
| Bluebird bio | BCH-BB694 | shRNA miR lentiviral vector to knockdown BCL11A expression | Severe SCD | I |
| CSL/Calimmune | CSL200 (CAL-H) | Ex vivo HSC gene therapy delivering gamma-globin gene | SCD cure, B-thalessemia | I |
| Novartis | ADPT03 | Ex vivo CRISPR | SCD cure | I |
| Beam Therapeutics | BEAM-101 | Ex vivo base-edited HSC cell therapy (corrected HbS) | SCD, Beta Thalassemia | I/II |
| Prime Medicine | - | Ex vivo base-edited HSC cell therapy (corrected HbS) | SCD | Preclin |
| Company | Candidate | MOA | Indications | Phase |
|---|---|---|---|---|
| Pfizer/Glycomimetics | Rivipansel | Pan-selectin antagonist | SCD - reduce VOCs | III |
| Imara | Tovinontrine (IMR-687) | PDE9 inhibitor | SCD | IIb |
| Modus Therapeutics | Sevuparin | Polysaccharide, anti-inflammatory | SCD - reduce VOCs | II |
| Merck | Montelukast | CysLT1 receptor inhibitor | SCD | II |
| Graphite Bio | GPH101 | Autologous HbA CRISPR-corrected HSCT | Severe SCD cure | I/II |
| Beam Therapeutics | BEAM-102 | Ex vivo base-edited HSC cell therapy (corrected HbS) | SCD | IND-enabling |