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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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