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4D Molecular Therapeutics Announces Presentation of Preclinical Data at the 6th International Update on Fabry Disease and Provides Clinical Update

May 30, 2019 Off By BusinessWire

– Novel vector, 4D-C102, demonstrates superior performance compared
to commonly used AAV1, AAV8, and AAV9 –

– Fabry product candidate, 4D-310, is on track for anticipated
clinical trial initiation in 2020 –

EMERYVILLE, Calif.–(BUSINESS WIRE)–4D Molecular Therapeutics, Inc. (4DMT), a leader in the discovery and
development of targeted, customized and proprietary next-generation
adeno-associated virus (AAV) vectors and gene therapy products,
announced new preclinical data with its novel vector, 4D-C102, broadly
for neuromuscular diseases and specifically for the product candidate
incorporating 4D-C102 for Fabry Disease, 4D-310. The data was
highlighted in a poster presentation at the 6^th International
Update on Fabry Disease held May 26 to 28, 2019 in Prague, Czech
Republic.

The data presented demonstrated:

Superior transduction of 4DMT’s novel vector, 4D-C102, in human
cardiomyocytes in vitro as compared to AAV1, AAV8 and AAV9
Dose dependent GLA expression and function after administration of
4D-310, that incorporates the 4D-C102 vector, in vitro in Fabry
patient fibroblasts
Superior delivery of the 4D-C102 vector in non-human primate cardiac
tissue in vivo as compared to AAV8 and AAV9

Fabry disease is a rare, X-linked, monogenic disorder caused by
mutations in the GLA gene that results in storage and accumulation of
lipids and leads to debilitating effects on a wide range of organs and
systems, including the heart. Current enzyme replacement therapy
partially clears accumulated lipids from the endothelial cells of
affected organs; however, clearance in other cell types, including
cardiomyocytes, appears incomplete. As a result, heart disease is the
most common cause of mortality in these patients. 4DMT’s Fabry product
candidate, 4D-310, aims to preferentially express the GLA enzyme in
cardiomyocytes, in addition to other tissues including kidney and liver,
to directly address the cause of Fabry-related heart disease.

“The data generated using the novel vector, 4D-C102, and the 4D-310
Fabry product candidate using that vector, helps validate 4DMT’s
Therapeutic Vector Evolution approach for in vivo cardiac tissue
targeting. This data package also provides the basis of a preclinical
data package that we believe will enable commencement of clinical
development of our Fabry product candidate,” said Dr. Gabriel Brooks, VP
Clinical Research and Development, and Program Leader, Neuromuscular and
LSD Therapeutic Areas at 4DMT.

“4DMT is building upon our Therapeutic Vector Evolution platform with
the goal of ultimately bringing gene therapy products to patients with
severe genetic diseases. We are pleased to present these data as an
early demonstration of our platform’s ability to potentially address
neuromuscular diseases,” said Dr. Peter Francis, Chief Medical Officer
at 4DMT.

A copy of the poster presentation can be found in the “Investors & News”
section of the company website, under “Events & Presentations”: www.4dmoleculartherapeutics.com

About 4D Molecular Therapeutics

4DMT is focused on the discovery and development of targeted, customized
and proprietary next-generation AAV gene therapy products for use in
patients with severe genetic diseases with high unmet medical need.
4DMT’s robust discovery platform, termed Therapeutic Vector Evolution,
empowers it to create customized gene delivery vehicles that can be
designed to deliver genes specifically to any tissue or organ in the
body, by optimal clinical routes of administration, at lower doses and
with resistance to pre-existing antibodies. 4DMT believes the product
candidates it has in development may enable it to treat both rare
genetic diseases and complex large market diseases. 4DMT is creating a
diverse and deep product pipeline through its own internal 4DMT
products, as well as partnered programs.

About 4DMT’s Therapeutic Vector Evolution

4DMT is advancing the field of targeted and optimized AAV vector
technology by deploying principles of evolution and natural selection to
create vectors that are designed to efficiently and selectively target
the desired cells within the diseased human organ via clinically optimal
routes of administration, at manageable doses and with resistance to
pre-existing antibodies in the population. 4DMT’s Therapeutic Vector
Evolution platform can deploy over 35 unique and proprietary AAV
libraries comprised of an estimated 1 billion vector capsid sequences.
After defining the target product profile, and the associated target
vector profile, 4DMT then applies proprietary methods to identify lead
vectors from within our AAV libraries. The result is a customized,
novel, and proprietary pharmaceutical-grade product candidate designed
for targeted therapeutic gene delivery and efficacy in humans.