| Disease family/type | Condition | Treatment name, Manufacturer | Therapy type | Population |
|---|---|---|---|---|
Cancers |
Blood cancer (Leukaemia; lymphoma; myeloma; myelodysplastic syndrome; myeloproliferative disorders)
|
Provided in hospitals
|
Stem cell therapy (Haematopoietic stem cell transplant) |
All ages |
| Cancers |
Blood cancer; for patients without access to donor haematopoietic stem cells | Zemcelpro, ExCellThera | Stem cell therapy (Expanded haematopoietic stem cells) | Adults |
| Cancers | Diffuse large B-cell lymphoma | Kymriah, Novartis Yescarta, Kite Pharma Breyanzi, Bristol Myers Squibb | Hybrid gene-cell therapy (CAR-T Therapy) | Adults |
Cancers | High-grade B-cell lymphoma | Yescarta, Kite Pharma Breyanzi, Bristol Myers Squibb | Hybrid gene-cell therapy (CAR-T Therapy) | Adults |
Cancers | Follicular Lymphoma | Yescarta, Kite Pharma Breyanzi, Bristol Myers Squibb | Hybrid gene-cell therapy (CAR-T Therapy) | Adults |
| Cancers | Mantle cell lymphoma | Tecartus, Kite Pharma / Gilead Breyanzi, Bristol Myers Squibb | Hybrid gene-cell therapy (CAR-T therapy) | Adults |
| Cancers | Primary mediastinal large B-cell lymphoma | Yescarta, Kite Pharma Breyanzi, Bristol Myers Squibb | Hybrid gene-cell therapy (CAR-T Therapy) | Adults |
| Cancers | B-Cell Acute Lymphoblastic Leukaemia | Kymriah, Novartis | Hybrid gene-cell therapy (CAR-T Therapy) | < 25 years |
| Cancers | Adult B-Cell Acute Lymphoblastic Leukaemia | Yescarta, Kite Pharma Tecartus, Kite Pharma / Gilead | Hybrid gene-cell therapy (CAR-T Therapy) | Adults |
Cancers | B-cell precursor acute lymphoblastic leukaemia (ALL) | Aucatzyl, Autolus Therapeutics | Hybrid gene-cell therapy (CAR-T Therapy) | > 26 years |
| Cancers | Multiple myeloma (adult) | Abecma, Bristol Myers Squibb Carvykti, Janssen Biotech | Hybrid gene-cell therapy (CAR-T Therapy) | Adults |
Cancers
| Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) | Ebvallo, Atara Biotherapeutics | Cell therapy (Donor T-cell immunotherapy) | > 2 years |
| Cancers | Melanoma | Imlygic, Amgen | Gene therapy | Adults |
| Immune-mediated blood disorders | Severe Combined Immunodeficiency (SCID) syndrome | Strimvelis, Orchard Therapeutics/ GlaxoSmithKline | Hybrid gene-cell therapy (Haematopoietic stem cells containing altered genetic material) | Children |
| Immune-mediated blood disorders | T-Cell deficiency (DiGeorge Syndrome, ataxia-telangiectasia) | Provided in hospitals | Stem cell therapy (Haematopoietic stem cell transplant) | All ages |
| Immune-mediated blood disorders | Acquired loss of immune function | Provided in hospitals | Stem cell therapy (Haematopoietic stem cell transplant) | All ages |
| Blood disorders (other) | Anaemias (aplastic anaemia; pure red cell anaemia; paroxysmal nocturnal haemoglobinuria) | Provided in hospitals | Stem cell therapy (Haematopoietic stem cell transplant) | All ages |
| Blood disorders (other) | Sickle cell disease | Provided in hospitals | Stem cell therapy (Haematopoietic stem cell transplant) | All ages |
Blood disorders (other) | Sickle cell disease | Casgevy, Vertex Pharmaceuticals | Gene therapy | > 12 years |
| Blood disorders (other) | Haemoglopinopathies | Provided in hospitals | Stem cell therapy (Haematopoietic stem cell transplant) | All ages |
| Blood disorders (other) | Haemophilia A | Roctavian, BioMarin | Gene therapy | Adults |
Blood disorders (other) | Haemophilia B | Hemgenix, UniQure | Gene therapy | Adults |
| Blood disorders (other) | Immune-mediated cytopenia | Provided in hospitals | Stem cell therapy (Haematopoietic stem cell transplant) | All ages |
| Blood disorders (other) | Haemophagocytic lymphohistiocytosis (HLH syndrome) | Provided in hospitals | Stem cell therapy (Haematopoietic stem cell transplant) | All ages |
| Infectious diseases | HIV/AIDS | Provided in hospitals | Stem cell therapy (Haematopoietic stem cell transplant) | All ages |
| Neurological disorders | Cerebral adrenoleukodystophy | Skysona, Bluebird Bio | Gene therapy | Children |
Neurological disorders
| Metachromatic leukodystrophy | Libmeldy, Orchard Therapeutics |
Combined gene-cell therapy (Genetically modified haematopoietic stem cells)
| Children |
| Neurological disorders | Multiple sclerosis | Provided in hospitals | Stem cell therapy (Haematopoietic stem cell transplant) | All ages |
| Neurological disorders | AADC deficiency | Upstaza, PTC Therapeutics | Gene therapy | > 18 months |
| Neurological disorders | Spinal muscular atrophy | Zolgensma, Novartis | Gene therapy | Children |
| Other disorders | Dystrophic epidermolysis bullosa | Vyjuvek, Krystal Biotech | Gene therapy | > 6 months |
| Vision loss | Retinal dystrophy | Luxturna, Novartis | Gene therapy | All ages |
| Acquired injury, eye | Corneal damage (e.g. chemical burn) | Holoclar, Chiesi | Tissue Therapy (Limbal stem cells) | All ages |
| Acquired injury, orthopaedic | Cartilage damage (knee joint) | Spherox, CO.DON AG | Cell therapy | Adults |
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