Displaying drugs 14701 - 14725 of 14969 in total
mRNA-3745
mRNA-3745 is a lipid nanoparticle (LNP) encapsulated with nucleoside-modified, codon-optimized mRNAs encoding human glucose 6-phosphatase (G6Pase).
Investigational
PVX-410 component XBP1 US184-192(YISPWILAV)
Investigational
PVX-410 component CD138 260-268(GLVGLIFAV)
Investigational
MB-107
MB-107 consists of mobilized peripheral blood-derived autologous CD34+ hematopoietic stem cells that are genetically modified to express the IL2RG gene.
Investigational
B7-H3-specific chimeric antigen receptor T cells
Investigational
SPL84-23-1
SPL84-23-1 is a sodium salt of 19-bp single-stranded RNA uniformly modified 2'-O-(2-methoxyethyl) phosphorothioate Antisense Oligonucleotide (ASO) consisting of 19 nucleotide residues with the sequence 5'-5MeC-5MeU-G-5MeC-A-A-5MeC-A-G-A-5MeU-G-G-A-A-G-A-5MeC-5MeU-3'
Investigational
PBFT02
PBFT02 is a non-replicating recombinant adeno-associated virus serotype 1 vector delivering human GRN gene encoding the protein progranulin.
Investigational
scAAV9.U7.ACCA
scAAV9.U7.ACCA is a non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding RNA. Developed by Nationwide Children’s Hospital, it is being investigated for the treatment of Duchenne muscular dystrophy.
Investigational
AAV9/MFSD8
AAV9/MFSD8 is a gene therapy developed by Neurogene Inc. It is an adeno-associated virus serotype 9 (AAV9) vector with an engineered transgene encoding the human CLN7/MFSD8 gene for expression of active human major facilitator superfamily domain containing 8. It was investigated for the treatment of neuronal ceroid lipofuscinosis type 7.
Investigational
WU-CART-007
WU-CART-007 consists of allogeneic, fratricide-resistant genetically modified T cells transduced with a 2nd generation 4-1BB-CD3z chimeric antigen receptor targeting human CD7.
Investigational
CMG901
CMG901 is an anti-Claudin 18.2 monoclonal antibody conjugated with MMAE payload.
Investigational
CTX120
CTX120 is an allogeneic CRISPR/Cas9-mediated genetically modified CAR T cell therapy targeting B-cell maturation antigen.
Investigational
CTX110
CTX110 is an allogeneic CRISPR/Cas9-mediated genetically modified CAR T cell therapy targeting CD19 antigen.
Investigational
ALLO-715
ALLO-715 is an allogeneic chimeric antigen receptor T Cell therapy targeting B cell maturation antigen (BCMA).
Investigational
ALLO-605
ALLO-605 is an allogeneic turbo chimeric antigen receptor (Turbocar) T Cell therapy targeting B Cell maturation antigen (BCMA).
Investigational
AVR-RD-04
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
Allogenic fetal mesenchymal stem cells
Investigational
HUB-101
Investigational
AMP-201
AMP-201 comprises an adeno-associated virus type 8 vector (AAV8) encoding the collagen Q gene (COLQ). It is under investigation for the treatment of collagen Q congenital myasthenia.
Investigational
BGT-DTDS
BGT-DTDS is an investigational gene therapy comprising an adeno-associated viral vector type 2 (AAV2) encoding the human SLC6A3 gene. It is under investigation for the treatment of dopamine transporter deficiency syndrome (DTDS).
Investigational
RGG0853
RGG0853 is an E1A lipid complex, a repressor gene complexed with a cationic lipid vector.
Investigational
Ozekibart
Ozekibart is a tetravalent recombinant humanized IgG1 agonist antibody targeting the human TRAIL death receptor 5 (DR5). It is being investigated for the treatment of chondrosarcoma.
Investigational
Displaying drugs 14701 - 14725 of 14969 in total