APR Nov/Dec 2022 - 46

»
BIOPHARMACEUTICALS
»
Potential of Gene and Cell
Therapies for Patients with
Rare/Orphan Diseases,
Ensuring Access
to Treatment
Andy Szczotka, PharmD
Chief Pharmacy Officer
AscellaHealth
For the estimated 25-30 million Americans1
and 400 million people
worldwide2 living with a rare disease, news of potential new gene
therapies is a powerful message that resonates personally among
individuals and families. Approximately 80% of rare diseases are
caused by a single-gene defect in a person's genes,3
and unfortunately,
rare diseases often are difficult to diagnose, with few or no treatment
options available. In fact, it is estimated that 95% of rare diseases do
not yet have a single FDA-approved treatment.3
impact more people than cancer and AIDS combined.3
and rare diseases
According to the U.S. Food & Drug Administration (FDA), gene therapy
is a technique4
that modifies a person's genes to treat or cure disease.
Gene therapy (GT) is the process of replacing defective genes with
healthy ones, adding new genes to help the body fight or treat disease,
or turn off genes that are causing problems.
Gene therapies use a target gene that expresses protein products at
a sufficient level to cure, or at least ameliorate, a disease caused by a
genetic defect. This involves the transfer of genetic material, usually in
a carrier or vector, and the uptake of the gene into the appropriate cells
of the body. Cell therapy also has the potential to treat the inherent
cause of both genetic and acquired diseases but involves the transfer
of cells with the relevant function into the patient.
Both approaches have the potential to alleviate the underlying
causes5
by replacing the missing protein(s) or cells causing the disease
symptoms, suppressing expression of proteins which are toxic to cells,
or eliminating cancerous cells. Many different types of cells6
may be
used as part of a therapy or treatment for a variety of diseases and
conditions, including hematopoietic (blood-forming) stem cells (HSC),
skeletal muscle stem cells, mesenchymal stem cells, lymphocytes,
dendritic cells, and pancreatic islet cells.
As of this writing, below is a list of licensed products from the Office of
Tissues and Advanced Therapies (OTAT), alphabetical by brand name
and uses:7
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| November/December 2022
APR Nov/Dec 2022

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