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What conditions can currently be treated using gene and cell therapy? (Full table)

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 cells)
All ages

Cancers

Diffuse large B-cell lymphoma
 
Kymriah, Novartis
Yescarta, Kite Pharma
Hybrid gene-cell therapy (CAR-T Therapy)
Adults

Cancers

 
Mantle cell lymphoma
 
Tecartus, Kite Pharma / Gilead 
 
Hybrid gene-cell therapy (CAR-T therapy)
 
Adults

Cancers

 
Primary mediastinal B-cell lymphoma
 
 
Yescarta, Kite Pharma
 
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
Hybrid gene-cell therapy (CAR-T Therapy)
Adults

Cancers

Melanoma
Imlygic, Amgen
Gene therapy
Adults

Cancers

Multiple myeloma (adult)
Abecma, Bristol-Myers-Squib
Hybrid gene-cell therapy (CAR-T 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 cells)
All ages

Immune-mediated blood disorders

Acquired loss of immune function
Provided in hospitals
Stem cell therapy (Haematopoietic stem cells)
All ages

Blood disorders (other)

Anaemias (aplastic anaemia; pure red cell anaemia; paroxysmal nocturnal haemoglobinuria)
Provided in hospitals
Stem cell therapy (Haematopoietic stem cells)
 
All ages

Blood disorders (other)

Beta-Thalassemia
Zynteglo, Bluebird Bio
Hybrid gene-cell therapy (Haematopoietic stem cells)
< 12 years

Blood disorders (other)

Sickle cell disease
Provided in hospitals
Stem cell therapy (Haematopoietic stem cells)
All ages

Blood disorders (other)

Haemoglopinopathies
Provided in hospitals
Stem cell therapy (Haematopoietic stem cells)
All ages

Blood disorders (other)

 
Immune-mediated cytopenia
 
Provided in hospitals
Stem cell therapy (Haematopoietic stem cells)
All ages

Blood disorders (other)

 
Haemophagocytic lymphohistiocytosis (HLH syndrome)
Provided in hospitals
Stem cell therapy (Haematopoietic stem cells)
All ages

Immune-related disorders

Crohn’s Disease (anal fistula)
Alofisel, Takeda
Hybrid gene-cell therapy (Haematopoietic stem cells)
All ages

Infectious diseases

HIV/AIDS
Provided in hospitals
Stem cell therapy (Haematopoietic stem cells)
All ages

Neurological disorders

Cerebral adrenoleukodystophy
Skysona, Bluebird Bio
Gene therapy
Children

Neurological disorders

Metachromatic leukodystrophy
Libmeldy, Orchard Therapeutics
Hybrid gene-cell therapy (Haematopoietic stem cells)
Children

Neurological disorders

Multiple sclerosis
Provided in hospitals
Stem cell therapy (Haematopoietic stem cells)
All ages

Neurological disorders

Spinal muscular atrophy
Zolgensma, Novartis
Gene therapy
Children

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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