Displaying drugs 13501 - 13525 of 15142 in total
RP-L201
Investigational
OCE-205
Investigational
Clervonafusp alfa
Investigational
AEM-28
AEM-28 is a 28 amino acid mimetic of apolipoprotein E being investigated for lipid disorders, such as homozygous familial hypercholesterolemia. It possesses anti-inflammatory properties.
Investigational
TAK-611
TAK-611, formerly known as SHP611, is a recombinant human arylsulfatase A. First developed by Takeda, it is being investigated for the treatment of metachromatic leukodystrophy.
Investigational
AVR-RD-02
AVR-RD-02 is an investigational ex vivo lentiviral gene therapy developed by AVROBIO Inc. It consists of autologous CD34+ enriched hematopoietic stem cells (HSCs) that have been genetically modified ex vivo with a lentiviral vector (LV) to contain a ribonucleic acid (RNA) transcript that, after reverse transcription, results in codon-optimized, complementary...
Investigational
ABX1100
ABX1100 is an investigational Centyrin-siRNA conjugate that targets the Gys1 gene, which encodes glycogen synthase 1 (Gys1), an enzyme that is responsible for glycogen synthesis in the muscle. It was first developed by Aro Biotherapeutics and is being investigated for the treatment of Pompe disease.
Investigational
Matched Description: … It was first developed by Aro Biotherapeutics and is being investigated for the treatment of Pompe disease …
PEG-Glucocerebrosidase
Investigational
rAAV9-rsATP7A
A recombinant adeno-associated viral vector gene therapy under investigation for the treatment of Menkes disease.
Investigational
Reveglucosidase alfa
Investigational
mRNA-3705
mRNA-3705 is an investigational mRNA encoding methylmalonic CoA mutase (MUT) enzyme. Developed by Moderna TX Inc., it is being investigated for the treatment of methylmalonic academia.
Investigational
Recombinant human alkaline phosphatase
Investigational
LV-FAH
Investigational
NZ-1002
Investigational
Heterologous human adult liver derived progenitor cells
Investigational
BBM-H901
BBM-H901 is a liver-tropic adeno-associated viral (AAV) vector carrying cassette coding hyperactive Padua factor IX (FIX) protein. It is being investigated for the treatment of hemophilia B.
Investigational
Dalcinonacog alfa
Dalcinonacog alfa is a recombinant human factor IX (FIX) variant. It is being investigated for the treatment of hemophilia B.
Investigational
mRNA-3351
mRNA-3351 is a modified mRNA encoding the UGT1A1 gene. Developed by Moderna, it is currently being investigated as the treatment for Crigler-Najjar syndrome
Experimental
OMS906
OMS906 is a humanized Igg4 monoclonal antibody against mannan-binding lectin-associated serine protease-3 (MASP-3) currently being developed by Omeros.
Investigational
CSL-689
CSL689 is a recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP).
Investigational
Allogeneic cytomegalovirus-specific cytotoxic T lymphocytes
Investigational
Exbivirumab
Exbivirumab is an investigational monoclonal antibody. It is one of the active ingredients in HepeX-B, a drug product containing two human monoclonal antibodies to the hepatitis B virus (HBV) surface antigen.
Investigational
Libivirumab
Libivirumab is an investigational monoclonal antibody. It is one of the active ingredients in HepeX-B, a drug product containing two human monoclonal antibodies to the hepatitis B virus (HBV) surface antigen.
Investigational
Bos taurus colostrum immunoglobulins
Investigational
MCMV3068A
MCMV3068A is an anticytomegalovirus monoclonal IgG1 antibody used in combination with MCMV5322A to form RG7667.
Investigational
Displaying drugs 13501 - 13525 of 15142 in total