22 BioPharm International
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Emerging Therapies eBook September 2024 www.biopharminternational.com
W
hen entering the discovery and early
development phases of a medication,
a company needs to look beyond sim-
ply detecting a disease or ailment that
could benefit from a novel therapy. There are several
factors that should be considered when developing a
new modality.
One of these considerations is manufacturing, ac-
cording to Wade Macedone, CEO of Andelyn Biosci-
ences. "Translating a potential treatment from the
laboratory to a therapeutic requires deep knowledge of
manufacturing science and technology (MS&T) as well
as process development, analytical development, qual-
ity and regulatory disciplines," says Macedone. Viral
vector delivery of gene therapies, for example, have
manufacturing requirements that are different from
antibodies and other modalities. "Everything from the
delivery vector, targeting specificity of the serotype,
the size of the therapeutic payload and dosing, to the
particulars of upstream cell culture and downstream
purification will vary from therapy to therapy," says
Macedone. "This is why expertise across the manu-
facturing disciplines is so important."
Cell and gene therapies require balance
There are two types of cell therapies, explains Michael
Pinaud, senior vice president, Business Operations,
Ascend Advanced Therapies; autologous cell ther-
apies use patient cells and allogeneic cell therapies
use donor cel ls. Autologous products have more
specific logistical challenges. "Material is first col-
lected at an apheresis center, then sent to a central
manufacturing location, processed, and finally back
to the patient for administration. All this movement
takes time and cost and requires maintenance of the
chain of identity to ensure the right product gets to
the right patient," says Pinaud. Allogeneic, in com-
parison, have fewer steps but are costly to produce.
"For either type of cell therapy, costs will be driven
by indication, dose required, in vitro or in vivo therapy,
Susan Haigney
SNEAKYPEAKPOINTS/PEOPLEIMAGES.COM
-
STOCK.ADOBE.COM
The Logistics of Developing
an Emerging Therapy
Emerging therapies, such as cell and gene therapies, come
with unique challenges that must be planned for in advance.
