CTX001

This drug entry is a stub and has not been fully annotated. It is scheduled to be annotated soon.

Identification

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Name
CTX001
Accession Number
DB15572
Type
Biotech
Groups
Investigational
Biologic Classification
Gene Therapies
Other gene therapies
Description

CTX001 is being developed by CRISPR Therapeutics and Vertex. It is an autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells in clinical trials for the treatment of severe sickle cell disease and severe beta-thalassemia.1

Synonyms
Not Available
External IDs
CTX-001 / CTX001
Categories
Not Available
UNII
Not Available
CAS number
Not Available

Pharmacology

Indication
Not Available
Pharmacodynamics
Not Available
Mechanism of action
Not Available
Absorption
Not Available
Volume of distribution
Not Available
Protein binding
Not Available
Metabolism
Not Available
Route of elimination
Not Available
Half life
Not Available
Clearance
Not Available
Toxicity
Not Available
Affected organisms
Not Available
Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Not Available
Food Interactions
Not Available

References

General References
  1. BioPharma Dive: First look at CRISPR, Vertex gene-editing therapy hints at treatment potential [Link]
External Links
Not Available

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
1, 2RecruitingTreatmentBeta-Thalassemia / Genetic Diseases, Inborn / Haemoglobinopathies congenital / Hematologic Diseases / Thalassaemic disorders1
1, 2RecruitingTreatmentHaemoglobinopathies congenital / Hematological Diseases / Sickle Cell Disorders1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage forms
Not Available
Prices
Not Available
Patents
Not Available

Properties

State
Not Available
Experimental Properties
Not Available

Taxonomy

Classification
Not classified

Drug created on November 19, 2019 09:39 / Updated on February 02, 2020 03:24