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Displaying drugs 14376 - 14400 of 15419 in total

WVE-003

WVE-003 is an allele-selective antisense oligonucleotide that works to selectively reduce the toxic mutant huntingtin (mHTT) protein.

Investigational

NGGT-002

NGGT-002 is a recombinant adeno-associated virus serotype 8 vector encoding human phenylalanine hydroxylase (PAH).

Investigational

Expanded human allogeneic neural retinal progenitor cells extracted from neural retina

Investigational

Autologous adipose derived mesenchymal stromal cells

Investigational

INT41

INT41 (also known as rAAV6-INT41) is a recombinant adeno-associated virus vector containing DNA encoding INT41 intrabody.

Investigational

RGX-181 consists of an adeno-associated virus serotype 9 (AAV9) vector designed to deliver the soluble lysosomal enzyme tripeptidyl peptidase I (TPP1) gene. Developed by REGENXBIO, it is being investigated for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.

Investigational

Matched Description: … RGX-181 consists of an adeno-associated virus serotype 9 (AAV9) vector designed to deliver the soluble ... Developed by REGENXBIO, it is being investigated for the treatment of late-infantile neuronal ceroid …