Regulation of Regenerative Medicines: A Global Perspective
1
The term “regenerative medicine” has become
globally recognized and accepted, and the science
driving discovery and development of regener-
ative medicines is rapidly evolving. The types of
biopharmaceutical products classified as regen-
erative medicines differs by country but typically
are made up of cellular or genetic therapies or
tissue engineered therapies. New technologies are
constantly being developed, and there is a need
for more flexibility in the regulatory governance
of these innovative products.
Regulation of Regenerative Medicines: a Global
Perspective is a comprehensive examination of the
regulatory framework of regenerative medicines
for those regulatory professionals working in the
biopharmaceutical industry, the government, or
academia. As the regenerative medicine land-
scape continues to expand and evolve, there is a
growing need for regulatory innovation of these
complex therapies. This book strives to provide
wide-ranging information on the regulations
governing regenerative medicines from develop-
ment to commercialization.
This book is divided into three distinct
sections. Section I contains general information
about regenerative medicines from definitions
to the unique properties of regenerative medi-
cines. Section II discusses special considerations,
manufacturing concepts, and distribution chal-
lenges. Lastly, Section III covers the regulatory
framework in multiple global regions including
Australia, Canada, China, Europe, Japan,
Singapore, and the US and looks forward to the
future of regenerative medicine.
Chapter 2 provides an overview of the
history of regenerative medicines. The chapter
provides insight into the evolution of regener-
ative medicine from cloning and stem cells to
recombinant DNA technology to the
“tissue rules” and gene therapy. The chapter
discusses the evolving US regulatory land-
scape and the involvement of the National
Institutes of Health and the US Food and Drug
Administration in the early regulation of regen-
erative medicines. There are many similarities
and differences between small molecules and
large molecules (i.e., biological products) but the
differences become more glaring when talking
about regenerative medicines which are com-
prised of cell therapy (i.e., CAR-T), gene therapy,
and tissue engineering. Chapter 3 provides an
overview of the unique properties that make up
certain types of regenerative medicines including
a brief summary on manufacturing, off-target
effects of gene therapies, and immunogenicity.
Manufacturing of regenerative medicines has
unique challenges which are covered extensively
in Chapter 8. Additionally, in Chapter 4, good
manufacturing practice and current good tissue
practices are examined when human cell and
tissues are a constituent part of a combination
product. Chemistry, manufacturing, and controls
1
Introduction
By Jocelyn Jennings, MS, RAC (US, Drugs, Devices)
1
The term “regenerative medicine” has become
globally recognized and accepted, and the science
driving discovery and development of regener-
ative medicines is rapidly evolving. The types of
biopharmaceutical products classified as regen-
erative medicines differs by country but typically
are made up of cellular or genetic therapies or
tissue engineered therapies. New technologies are
constantly being developed, and there is a need
for more flexibility in the regulatory governance
of these innovative products.
Regulation of Regenerative Medicines: a Global
Perspective is a comprehensive examination of the
regulatory framework of regenerative medicines
for those regulatory professionals working in the
biopharmaceutical industry, the government, or
academia. As the regenerative medicine land-
scape continues to expand and evolve, there is a
growing need for regulatory innovation of these
complex therapies. This book strives to provide
wide-ranging information on the regulations
governing regenerative medicines from develop-
ment to commercialization.
This book is divided into three distinct
sections. Section I contains general information
about regenerative medicines from definitions
to the unique properties of regenerative medi-
cines. Section II discusses special considerations,
manufacturing concepts, and distribution chal-
lenges. Lastly, Section III covers the regulatory
framework in multiple global regions including
Australia, Canada, China, Europe, Japan,
Singapore, and the US and looks forward to the
future of regenerative medicine.
Chapter 2 provides an overview of the
history of regenerative medicines. The chapter
provides insight into the evolution of regener-
ative medicine from cloning and stem cells to
recombinant DNA technology to the
“tissue rules” and gene therapy. The chapter
discusses the evolving US regulatory land-
scape and the involvement of the National
Institutes of Health and the US Food and Drug
Administration in the early regulation of regen-
erative medicines. There are many similarities
and differences between small molecules and
large molecules (i.e., biological products) but the
differences become more glaring when talking
about regenerative medicines which are com-
prised of cell therapy (i.e., CAR-T), gene therapy,
and tissue engineering. Chapter 3 provides an
overview of the unique properties that make up
certain types of regenerative medicines including
a brief summary on manufacturing, off-target
effects of gene therapies, and immunogenicity.
Manufacturing of regenerative medicines has
unique challenges which are covered extensively
in Chapter 8. Additionally, in Chapter 4, good
manufacturing practice and current good tissue
practices are examined when human cell and
tissues are a constituent part of a combination
product. Chemistry, manufacturing, and controls
1
Introduction
By Jocelyn Jennings, MS, RAC (US, Drugs, Devices)