Human Gene Therapy - April 2023 - 256

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GENE THERAPY BRIEFS
''We have compared, side-byside,
fused and unfused prime editors
plus integrases at several different
target sites in human cells, and we
have never observed any benefit
from fusing the prime editor and integrase,''
says Liu, who is also the
director of the Merkin Institute of
Transformative Technologies in
Healthcare and a Howard Hughes
Medical Institute investigator.
Asked about the potential for a
challenge to the PASTE patent,
Gootenberg acknowledged: ''It's
obviously going to be a complex
situation with intersecting IP, as
has been with Cas9 and other
nucleases from the start. But that
said, we're really excited about how
we can develop these technologies
and move these technologies forward
to develop actual cures for
patients.''1
ABBVIE, CAPSIDA EXPAND
EYE DISEASE
COLLABORATION TO THREE
PROGRAMS, UP TO $665M
AbbVie and Capsida Biotherapeutics
have expanded a 2-year-old
strategic collaboration to develop
genetic medicines for eye diseases
with high unmet need, with Capsida
standing to potentially gain up to
$665 million.
The companies have agreed to
identify and advance three programs
by combining AbbVie's development
capabilities with Capsida's
novel adeno-associated virus (AAV)
engineering platform and manufacturing
capability.
''This expanded collaboration
with Capsida has the potential to
develop transformative therapies for
patients with serious eye diseases,''
said Jonathon Sedgwick, PhD, AbbVie's
vice president and global head
of discovery research. ''In pursuing
the promise of genetic medicinebased
therapeutics, AbbVie continues
to expand our capabilities, and
we are pleased to have Capsida as a
partner.''2
Capsida agreed to lead capsid
discovery efforts for all programs
using its high-throughput AAV
engineering platform and will
oversee process development and
early clinical manufacturing.
AbbVie will lead innovative therapeutic
cargo approaches and be
responsible for development and
commercialization.
AbbVie has agreed to pay Capsida
$70 million consisting of upfront
payments and a potential equity
investment. For the three programs,
AbbVie also agreed to pay Capsida
up to $595 million in option fees
and research and development milestones,
with potential for further
commercial milestones. Capsida is
also eligible to receive mid-to-high
single-digit royalty payments on
future product sales.
The companies launched their
neurodegenerative disease partnership
in 2021.
AERA THERAPEUTICS
LAUNCHES WITH $193M
AND DELIVERY PLATFORM
Aera Therapeutics has launched
with $193 million in combined
Series A and Series B financings,
and the goal of applying its platform
for delivering genetic medicines
across a variety of modalities and
therapeutic areas.
Aera's delivery platform is based
on research by the laboratory of the
company's scientific founder Feng
Zhang, PhD, the CRISPR pioneer
and core member of the Broad
Institute of MIT and Harvard. Zhang
is also an investigator with MIT's
McGovern Institute for Brain
Research, as well as the James and
Patricia Poitras Professor of Neuroscience
at MIT and an investigator
at the Howard Hughes Medical
Institute.
Aera said its proprietary protein
nanoparticle genetic medicine delivery
platform combines the benefits of
an endogenous self-assembling system
with the engineerability of a
protein-based system and has the
potential to enable the delivery of
various genetic medicine modalities.
The platform is designed to leverage
the discovery of endogenous human
proteins derived from retroelements
that can self-assemble to form
capsid-like structures and which can
package and transfer nucleic acid
cargo.
Aera's technology also includes a
licensed therapeutic enzyme platform
based on the discovery of
novel, compact, and programmable
gene editing enzymes. The company
reasons that the compact size
of the enzymes may help overcome
the packaging and delivery
challenges of current gene editor
systems.
''With access to enabling delivery
and payload technology, we
aim to expand the reach of genetic
medicines to different tissues and
applications to benefit more patients
across more disease areas,'' said
Aera CEO Akin Akinc, PhD.3
Akinc is a member of Aera's
board, which is chaired by John
Maraganore, PhD, Alnylam Pharmaceuticals'
founding CEO who
stepped down at the end of 2021.
Other leaders and board members
include Robert Nelsen, managing
director, ARCH Venture Partners;
Issi Rozen, board observer and
venture partner, GV (Google Ventures);
Vicki Sato, PhD, former
president, Vertex Pharmaceuticals;
David Schenkein, MD, general
partner, GV; and Josh Wolfe,
cofounder and managing partner,
Lux Capital.
ARCH Venture Partners, GV
(Google Ventures), and Lux
Capital co-led the $193 million in
financings.
The company's name comes
from Aerarium, the Roman word
for the public treasury. Akinc noted
that Aera is founded around the
discovery of human proteins that
package genetic cargo, adding that
the proteins are ''treasure in our
genome.''4
Human Gene Therapy - April 2023

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