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

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