Resources Tag: Gene Therapy

An autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase (ADA) cDNA sequence from human haematopoietic stem/progenitor (CD34+ ) cells.

Zynteglo is an autologous, advanced therapy medicinal product (ATMP) suitable for infusion and conveys functional copies of the βA-T87Q-globin gene in HSCs to myeloablated patients who have TDT.

TECARTUS is a CD19-directed genetically modified autologous T cell immunotherapy. To prepare TECARTUS, a patient’s own T cells are harvested and genetically modified ex vivo by retroviral transduction to express a chimeric antigen receptor (CAR) comprising a murine anti-CD19 single-chain variable fragment (scFv) linked to CD28 and CD3-zeta co-stimulatory domains. The anti-CD19 CAR T cells are expanded and infused back into the patient, where they can recognize and eliminate CD19-expressing target cells.

PROVENGE consists of autologous peripheral blood mononuclear cells, including antigen presenting cells (APCs), that have been activated during a defined culture period with a recombinant human protein, PAP-GM-CSF, consisting of prostatic acid phosphatase (PAP), an antigen expressed in prostate cancer tissue, linked to granulocyte-macrophage colony-stimulating factor (GM-CSF), an immune cell activator.

KYMRIAH™ (tisagenlecleucel) is a CD19-directed genetically modified autologous T cell immunotherapy comprised of autologous T cells that are genetically modified using a lentiviral vector to encode an anti-CD19 chimeric antigen receptor (CAR). The CAR is comprised of a murine single-chain antibody fragment (scFv) specific for CD19, followed by a CD8 hinge and transmembrane region that is fused to the intracellular signaling domains for 4-1BB (CD137) and CD3 zeta.

IMLYGIC was derived from a novel primary HSV-1 isolate (JS1, ECACC Accession Number 01010209) that demonstrates enhanced oncolytic activity towards tumor cells, as compared to the commonly used laboratory strains (e.g., 17syn+).

LUXTURNA (voretigene neparvovec-rzyl) is a recombinant adeno-associated virus serotype 2 (AAV2) vector with a cytomegalovirus (CMV) enhancer and chicken beta actin (CβA) promoter driving expression of the gene for human retinal pigment epithelium 65 kDa protein (hRPE65), which is an isomerohydrolase converting all-trans-retinyl ester to 11-cis-retinol in the retinoid visual cycle.

LAVIV is an autologous cellular product composed of fibroblasts suspended in Dulbecco’s Modified Eagle’s Medium (DMEM) without phenol red. Dermal fibroblasts from post-auricular skin biopsy tissue are aseptically expanded using standard tissue-culture procedures until sufficient cells for three doses are obtained.

ZOLGENSMA is a suspension of an adeno-associated viral vector-based gene therapy for intravenous infusion. It is a recombinant self-complementary AAV9 containing a transgene encoding the human survival motor neuron (SMN) protein, under the control of a cytomegalovirus enhancer/chicken-β-actin hybrid promoter.

YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.