APR Nov/Dec 2022 - 48

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BIOPHARMACEUTICALS
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ABECMA (idecabtagene vicleucel)
Multiple Myeloma
ALLOCORD (HPC, Cord Blood)
SSM Cardinal Glennon Children's Medical Center
BREYANZI
B-cell lymphoma
CARVYKTI (ciltacabtagene autoleucel)
Janssen Biotech, Inc.
CLEVECORD (HPC Cord Blood)
Disorders affecting the hematopoietic system
Ducord, HPC Cord Blood
Disorders affecting the hematopoietic system
GINTUIT (Allogeneic Cultured Keratinocytes and Fibroblasts in
Bovine Collagen)
Treatment of mucogingival condition
HEMACORD (HPC, cord blood)
Disorders affecting the hematopoietic system
HPC, Cord Blood
Disorders affecting the hematopoietic system
HPC, Cord Blood - MD Anderson Cord Blood Bank
Disorders affecting the hematopoietic system
HPC, Cord Blood - LifeSouth
Disorders affecting the hematopoietic system
HPC, Cord Blood - Bloodworks
Disorders affecting the hematopoietic system
IMLYGIC (talimogene laherparepvec)
Melanoma
KYMRIAH (tisagenlecleucel)
Acute Lymphoblastic Leukemia & B cell lymphoma
LAVIV (Azficel-T)
Nasolabial fold wrinkles
LUXTURNA
Retinal dystrophy
MACI (Autologous Cultured Chondrocytes on a Porcine
Collagen Membrane)
Autologous Cultured Chondrocytes on a Porcine Collagen Membrane
PROVENGE (sipuleucel-T)
Prostate Cancer
RETHYMIC
Congenital athymia
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SKYSONA (elivaldogene autotemcel)
Active cerebral adrenoleukodystrophy (CALD).
STRATAGRAFT
Allogeneic cultured keratinocytes and dermal fibroblasts in
murine collagen
TECARTUS (brexucabtagene autoleucel)
Acute Lymphoblastic Leukemia & Mantle Cell Lymphoma
YESCARTA (axicabtagene ciloleucel)
Follicular Lymphoma & B cell lymphoma
ZYNTEGLO (betibeglogene autotemcel)
ß-thalassemia
ZOLGENSMA (onasemnogene abeparvovec-xioi)
Spinal Muscular Atrophy
A Lexicon for Main Types of Gene
Therapy Products
Viral Vectors is the term that the FDA uses when referring to viruses
that are modified to remove their ability to cause infectious disease.
Viruses have a natural ability to deliver genetic material into cells, and
therefore some gene therapy products are derived from viruses.8
These
modified viruses can be used as vectors (vehicles) to carry therapeutic
genes into human cells (Figure 1).
Source: Food & Drug Administration. https://www.fda.gov/consumers/
consumer-updates/how-gene-therapy-can-cure-or-treat-diseases?%20
how%20does%20it%20work?
Figure 1.
Patient-derived cellular gene therapies refer to products where cells are
removed from the patient, genetically modified (often using a viral
vector) and then returned to the patient.6
Plasmid DNA describes circular DNA molecules that can be genetically
engineered to carry the good therapeutic genes into human cells.
https://www.fda.gov/consumers/consumer-updates/how-gene-therapy-can-cure-or-treat-diseases?how-does-it-work?
APR Nov/Dec 2022

Table of Contents for the Digital Edition of APR Nov/Dec 2022
