autologous cd34+ cells encoding arsa gene
Nervous system (N07)Nervous system (N07) compound in ORCHARD THERAPEUTICS's portfolio. Molecular target: ARSA.
- Target
- ARSA
- Mechanism
- ARSA exogenous gene
pharma · Metachromatic Leukodystrophy (MLD) · Mucopolysaccharidosis type I, Hurler Syndrome
ORCHARD THERAPEUTICS
Orchard Therapeutics (Netherlands) B.V.
ORCHARD THERAPEUTICS drug portfolio — 12 indexed compounds with therapeutic class and molecular targets from public FDA and regional approval metadata.
London, GB HQ
12 Employees
EMA registrant Type
Company details
- Status
- Public
- HQ
- London, GB
- Employees
- 12
- Programs
- 5
- Drugs
- 12
- Patents
- 0
ORCHARD THERAPEUTICS pipeline
Portfolio lens on 12 drugs and development assets — regulatory status, targets, and deep links to NovaPharmaNews drug profiles.
ORCHARD THERAPEUTICS drug portfolio — 12 indexed compounds with therapeutic class and molecular targets from public FDA and regional approval metadata.
Drug portfolio
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busulfan
Antineoplastic and immunomodulating agents (L01)US regulatory status: approved. Molecular target: DNA.
- Target
- DNA
- Mechanism
- DNA inhibitor
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US regulatory status: approved. Mechanism: Unknown.
- Mechanism
- Unknown
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Development asset linked to ORCHARD THERAPEUTICS in public FDA and regional records.
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US regulatory status: approved. Molecular target: Granulocyte colony-stimulating factor receptor.
- Target
- Granulocyte colony-stimulating factor receptor
- Mechanism
- Granulocyte colony stimulating factor receptor agonist
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Development asset linked to ORCHARD THERAPEUTICS in public FDA and regional records.
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US regulatory status: approved. Molecular target: DNA polymerase (alpha/delta/epsilon).
- Target
- DNA polymerase (alpha/delta/epsilon)
- Mechanism
- DNA polymerase (alpha/delta/epsilon) inhibitor
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Development asset linked to ORCHARD THERAPEUTICS in public FDA and regional records.
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laronidase
Alimentary tract and metabolism (A16)US regulatory status: approved. Molecular target: Dermatan sulfate.
- Target
- Dermatan sulfate
- Mechanism
- Dermatan sulfate hydrolytic enzyme
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plerixafor
Antineoplastic and immunomodulating agents (L03)US regulatory status: approved. Molecular target: C-X-C chemokine receptor type 4.
- Target
- C-X-C chemokine receptor type 4
- Mechanism
- C-X-C chemokine receptor type 4 partial agonist
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rituximab
Antineoplastic and immunomodulating agents (L01)US regulatory status: approved. Molecular target: B-lymphocyte antigen CD20.
- Target
- B-lymphocyte antigen CD20
- Mechanism
- B-lymphocyte antigen CD20 binding agent
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Development asset linked to ORCHARD THERAPEUTICS in public FDA and regional records.