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Displaying drugs 1351 - 1375 of 1988 in total
Investigational
NGGT-002 is a recombinant adeno-associated virus serotype 8 vector encoding human phenylalanine hydroxylase (PAH).
Investigational
Investigational
Investigational
ADVM-062 is an adeno-associated virus vector carrying complementary deoxyribonucleic acid encoding the human long-wave-sensitive opsin 1 (AAV.7m8-L-opsin)
Investigational
AVR-RD-04 is an investigational gene therapy for cystinosis. It consists of autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin.
Investigational
SB-218078 is a specific inhibitor of CHK1.
Investigational
Matched Iupac: … 28-oxa-4,14,19-triazaoctacyclo[12.11.2.1^{15,18}.0^{2,6}.0^{7,27}.0^{8,13}.0^{19,26}.0^{20,25}]octacosa …
Pegadricase is under investigation in clinical trial NCT01021241 (Safety and Efficacy Study of Intravenous Uricase-PEG 20).
Investigational
Gavorestat is under investigation in clinical trial NCT04902781 (Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia).
Investigational
DG031, deCODE genetics's lead compound, is being developed for the prevention of myocardial infarction, or heart attack.
Investigational
KB002 is an engineered human IgG1k antibody engineered human. It is developed for the treatment of autoimmune diseases, initially rheumatoid arthritis.
Investigational
Investigational
GLPG-0259 is under investigation in clinical trial NCT01024517 (GLPG0259 Solid Formulation Bioavailability and Food Effect).
Investigational
PR006 is a non-replicating recombinant adeno-associated virus serotype 9 containing the progranulin gene. It is being investigated for the treatment of dementia.
Investigational
Investigational
Investigational
SNS01-T is a gene therapy nanoparticle. It is composed of three components: a B cell-specific plasmid expressing eIF5AK50R; an siRNA that targets the native eIF5A that promotes growth/anti-apoptosis of cancer cells; and polyethylenimine.
Investigational
RR001 is an investigational gene therapy consisting of autologous human adipose perivascular stromal cells genetically modified to secrete soluble tumour necrosis factor-related apoptosis-inducing ligand (AD-PC sTRAIL).
Investigational
TTAX01 is a cryopreserved human umbilical cord product being developed by TissueTech.
Investigational
ALMB-0166 is a umanized connexin 43 (Cx43) monoclonal antibody being investigated for the treatment of acute spinal cord injury. It was granted FDA orphan designation in November 2018.
Investigational
Investigational
EB003 is a CRISPR-based sequence-specific antimicrobial (SSAM) targeted against shiga toxin-producing E. coli.
Investigational
PBGM01 is a non-replicating recombinant adeno-associated virus serotype hu68 vector, which contains human GLB1 transgene encoding the galactosidase beta 1 enzyme.
Investigational
VGB-R04 is an adeno-associated viral vector delivering human FIX Padua gene.
Investigational
Mavelertinib is under investigation in clinical trial NCT02349633 (Study for Patients With NSCLC EGFR Mutations (Del 19 or L858R +/- T790M)).
Investigational
Displaying drugs 1351 - 1375 of 1988 in total