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Displaying drugs 13526 - 13550 of 15164 in total

SRP-6004

A recombinant AAVrh74 gene therapy expressing the dysferlin transgene under control of a muscle-specific promoter (rAAVrh74.MHCK7.DYSF.DV).

Investigational

VY-HTT01 is a gene therapy designed to reduce the expression of huntingtin, thereby altering disease progression. VY-HTT01 is comprised of an adeno-associated virus capsid (AAV1) and a proprietary transgene that harnesses the canonical RNA interference pathway to selectively knock down levels of HTT mRNA.

Investigational

Matched Description: … VY-HTT01 is comprised of an adeno-associated virus capsid (AAV1) and a proprietary transgene that harnesses …

BMN-052

Experimental

Withdrawn

BMN-053

Experimental

Withdrawn

Autologous Hematopoietic Peripheral Blood Stem Cells

Autologous hematopoietic peripheral blood stem cells were tested in a clinical trial (NCT00424489) by Northwestern University for the treatment of refractory myasthenia gravis.

Investigational

αAβ–Gas6

αAβ–Gas6 is a chimeric fusion protein being investigated in preclinical studies as an immunotherapeutic agent for Alzheimer's disease (AD). It is made of a single chain variable fragment (scFv) of an Amyloid β (Aβ)-targeting monoclonal antibody fused with a truncated receptor binding domain of growth arrest-specific 6 (Gas6), which is...

Experimental

Matched Description: … inducer of Aβ,[A253203] which is the main component of the amyloid plaques found in individuals with AD and …