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Orchard Therapeutics
Orchard Therapeutics
(ORTX)
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Drug Name
Phase 1
Phase 2
Phase 3
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Indication
Atidarsagene autotemcel
metachromatic leukodystrophy
,
lysosomal storage diseases
Antithrombin iii
streptococcal infections
,
rectal neoplasms
,
meningioma
,
atrial fibrillation
,
mantle-cell lymphoma
,
multiple myeloma
,
plasma cell neoplasms
,
gliosarcoma
,
central nervous system neoplasms
,
nervous system neoplasms
,
colonic neoplasms
,
streptococcus agalactiae
,
breast neoplasms
,
neoplasms
,
arthritis
,
ankylosing spondylitis
,
spondylarthritis
,
spondylitis
,
non-small-cell lung carcinoma
,
lung neoplasms
,
prostatic neoplasms
,
psoriatic arthritis
,
meningococcal infections
,
leukemia
,
carcinoma
,
bcr-abl positive chronic myelogenous leukemia
,
lymphoma
,
sickle cell anemia
,
melanoma
,
renal cell carcinoma
,
macular edema
,
edema
,
vision disorders
,
low vision
,
meningitis
,
meningococcal meningitis
,
uveitis
,
thrombocytopenia
,
sarcoma
,
neuroendocrine tumors
,
glioblastoma
,
myeloid leukemia
,
ovarian neoplasms
,
human influenza
,
myelodysplastic syndromes
,
preleukemia
,
syndrome
,
gastrointestinal neoplasms
,
digestive system neoplasms
,
b-cell chronic lymphocytic leukemia
,
lymphoid leukemia
,
hepatocellular carcinoma
,
carcinoid tumor
,
malignant carcinoid syndrome
,
serotonin syndrome
,
astrocytoma
,
hypercholesterolemia
,
hyperlipoproteinemia type ii
,
homozygous familial hypercholesterolemia
,
chronic obstructive pulmonary disease
,
lung diseases
,
obstructive lung diseases
,
gastrointestinal stromal tumors
,
philadelphia chromosome
,
ovarian epithelial carcinoma
,
axial spondyloarthritis
,
allergic rhinitis
,
rhinitis
,
allergic rhinitis seasonal
,
graft vs host disease
,
pituitary acth hypersecretion
,
acth-secreting pituitary adenoma
,
rheumatoid arthritis
,
psoriasis
,
hepatitis b
,
whooping cough
,
tetanus
,
diphtheria
,
haemophilus influenzae
,
non-hodgkin lymphoma
,
b-cell lymphoma
,
acromegaly
,
hodgkin disease
,
recurrence
,
arteritis
,
giant cell arteritis
,
polymyalgia rheumatica
,
follicular lymphoma
,
pancreatic neoplasms
,
fistula
,
myeloid leukemia chronic-phase
,
neuroendocrine carcinoma
,
heart failure
,
hypertension
,
essential hypertension
,
systolic murmurs
,
multiple sclerosis
,
neoplasm metastasis
,
chronic progressive multiple sclerosis
,
sclerosis
,
small cell lung carcinoma
Simoladagene autotemcel
severe combined immunodeficiency
,
immunologic deficiency syndromes
Hsct
hematologic neoplasms
,
precursor cell lymphoblastic leukemia-lymphoma
,
lymphoid leukemia
,
neoplasms
,
syndrome
,
graft vs host disease
,
bcr-abl positive chronic myelogenous leukemia
,
b-cell chronic lymphocytic leukemia
,
bronchiolitis obliterans syndrome
,
anemia
,
diabetes mellitus
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
thrombocytopenia
,
leukopenia
,
cytopenia
,
hematopoietic stem cell transplantation
,
lung diseases
,
bronchiolitis obliterans
,
bronchiolitis
,
type 1 diabetes mellitus
,
renal insufficiency
,
stem cell transplantation
,
aplastic anemia
,
leukemia
,
multiple myeloma
,
myeloid leukemia acute
,
lymphoma
,
plasma cell neoplasms
,
myeloid leukemia
,
myelodysplastic syndromes
,
non-hodgkin lymphoma
,
hodgkin disease
,
preleukemia
,
transplantation
,
residual neoplasm
,
autologous transplantation
Arm1
myeloid leukemia acute
,
myeloid leukemia
,
healthy volunteers/patients
,
myelodysplastic syndromes
,
preleukemia
,
renal cell carcinoma
,
streptococcal infections
,
chronic obstructive pulmonary disease
,
lung diseases
,
obstructive lung diseases
,
b-cell chronic lymphocytic leukemia
,
lymphoid leukemia
,
glioblastoma
,
non-small-cell lung carcinoma
,
leukemia
,
colorectal neoplasms
,
lung neoplasms
,
sjogren's syndrome
,
pancreatic ductal carcinoma
,
adenocarcinoma
,
multiple myeloma
,
primary myelofibrosis
,
lymphoma
,
pancreatic neoplasms
,
amyotrophic lateral sclerosis
,
motor neuron disease
,
sclerosis
,
triple negative breast neoplasms
,
ovarian neoplasms
,
plasma cell neoplasms
,
iron overload
,
glioma
,
astrocytoma
,
oligodendroglioma
,
sarcoma
,
chronic hepatitis c
,
chronic hepatitis
,
osteoarthritis
,
graft rejection
,
malaria
,
falciparum malaria
,
sarcopenia
,
idiopathic pulmonary fibrosis
,
pulmonary fibrosis
,
uveitis
,
hematologic neoplasms
,
meningococcal infections
,
ovarian epithelial carcinoma
,
carcinoma
,
hepatocellular carcinoma
,
liver neoplasms
,
obesity
,
heart failure
,
colonic neoplasms
,
neoplasm micrometastasis
,
uterine cervical neoplasms
,
rheumatoid arthritis
,
autoimmune diseases
,
systemic lupus erythematosus
,
breast neoplasms
,
chronic urticaria
,
urticaria
,
arthritis
,
melanoma
,
syndrome
,
neoplasms
,
haemophilus infections
,
liver transplantation
,
diphtheria
,
covid-19
,
chronic renal insufficiency
,
kidney diseases
,
psoriatic arthritis
,
hepatitis b
,
tetanus
,
influenza in birds
,
chronic disease
,
thrombocytopenic purpura idiopathic
,
thrombocytopenia
,
gouty arthritis
,
macular edema
,
edema
,
autoimmune hepatitis
,
juvenile arthritis
,
whooping cough
,
haemophilus influenzae
,
hyperphosphatemia
,
chronic hepatitis b
,
eosinophilia
,
cryopyrin-associated periodic syndromes
,
cellulitis
,
lupus nephritis
,
nephritis
,
vascular diseases
,
polypoidal choroidal vasculopathy
,
h5n1 subtype influenza a virus
,
disease
,
human influenza
,
kidney transplantation
,
infections
,
hepatitis
,
hepatitis a
,
hepatitis c
,
fibrosis
,
tick-borne encephalitis
,
encephalitis
,
communicable diseases
,
cystic fibrosis
,
h1n1 subtype influenza a virus
,
cytomegalovirus infections
,
viremia
,
rabies
Arm2
healthy volunteers/patients
,
myeloid leukemia acute
,
myeloid leukemia
,
myelodysplastic syndromes
,
preleukemia
,
chronic obstructive pulmonary disease
,
lung diseases
,
obstructive lung diseases
,
congenital abnormalities
,
chromosome aberrations
,
b-cell chronic lymphocytic leukemia
,
lymphoid leukemia
,
renal cell carcinoma
,
non-small-cell lung carcinoma
,
leukemia
,
colorectal neoplasms
,
multiple myeloma
,
lung neoplasms
,
pancreatic neoplasms
,
primary myelofibrosis
,
sjogren's syndrome
,
lymphoma
,
amyotrophic lateral sclerosis
,
motor neuron disease
,
sclerosis
,
plasma cell neoplasms
,
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
astrocytoma
,
oligodendroglioma
,
sarcoma
,
carcinoma
,
hepatocellular carcinoma
,
liver neoplasms
,
chronic hepatitis c
,
chronic hepatitis
,
pancreatic ductal carcinoma
,
adenocarcinoma
,
osteoarthritis
,
graft rejection
,
malaria
,
falciparum malaria
,
sarcopenia
,
triple negative breast neoplasms
,
gastrointestinal neoplasms
,
gastrointestinal stromal tumors
,
idiopathic pulmonary fibrosis
,
pulmonary fibrosis
,
neoplasm micrometastasis
,
uterine cervical neoplasms
,
uveitis
,
hematologic neoplasms
,
meningococcal infections
,
obesity
,
heart failure
,
human influenza
,
breast neoplasms
,
chronic urticaria
,
urticaria
,
syndrome
,
arthritis
,
melanoma
,
haemophilus infections
,
liver transplantation
,
infections
,
covid-19
,
chronic renal insufficiency
,
kidney diseases
,
thrombocytopenic purpura idiopathic
,
thrombocytopenia
,
psoriatic arthritis
,
neoplasms
,
influenza in birds
,
diphtheria
,
gouty arthritis
,
macular edema
,
edema
,
autoimmune hepatitis
,
juvenile arthritis
,
h5n1 subtype influenza a virus
,
disease
,
hyperphosphatemia
,
tetanus
,
communicable diseases
,
hepatitis b
,
chronic hepatitis b
,
chronic disease
,
eosinophilia
,
cryopyrin-associated periodic syndromes
,
cellulitis
,
lupus nephritis
,
nephritis
,
hepatitis
,
hepatitis a
,
kidney transplantation
,
hepatitis c
,
fibrosis
,
tick-borne encephalitis
,
encephalitis
,
viremia
,
cystic fibrosis
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