4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT), a leading
clinical-stage genetic medicines company focused on unlocking the
full potential of genetic medicines to treat large market diseases,
and Arbor Biotechnologies, Inc. (Arbor), a next-generation gene
editing company developing potentially curative therapeutics for
patients with serious diseases based on its proprietary and modular
toolbox of DNA-editing technologies, today announced a strategic
partnership focused on advancing new AAV-based gene-editing
therapies for central nervous system (CNS) diseases with high unmet
medical needs in both rare and common disease populations. 4DMT and
Arbor will co-develop and co-commercialize up to six AAV-delivered
CRISPR/Cas-based therapeutic candidates, with the costs and profits
shared evenly based on mutually agreed plans.
Arbor will lead research, development and, if approved,
commercialization efforts on the first product candidate in the
collaboration, which will address a molecular target implicated in
amyotrophic lateral sclerosis (ALS). 4DMT will lead research,
development and, if approved, commercialization efforts on the
second product candidate (target and disease to be disclosed at a
future date).
Arbor utilizes its artificial intelligence and machine learning,
or AI/ML, driven discovery engine and protein engineering
capabilities to identify and optimize genomic editors with the
potential to treat a broad range of genetic diseases. Arbor’s
proprietary portfolio of novel genomic editors has therapeutic
potential due to, among other things, their unique cut patterns and
protospacer adjacent motifs (PAMs). The PAMs, for example, may
enable Arbor to access greater than 93% of all sites in the human
genome which allows it to target nearly any genetic locus. The
small size of Arbor’s genomic editing technology allows the use of
additional delivery mechanisms with their high specificity, which
can enable improved safety profiles. Arbor also has expertise in
CNS biology, CNS drug development and CNS therapeutic
development.
4DMT’s proprietary invention platform, Therapeutic Vector
Evolution, has yielded customized and evolved, highly targeted AAV
vectors in ophthalmology, pulmonology and cardiology that have
demonstrated differentiation compared to naturally occurring AAVs
in clinical studies. Initial evidence includes strong clinical
activity in wet age-related macular degeneration (wet AMD) with low
dose, intravitreal delivery using the R100 vector (4D-150 product
candidate), and unprecedented transgene expression was achieved in
the lungs of people with cystic fibrosis using the A101 vector
(4D-710 product candidate). 4DMT has utilized its platform to
invent customized AAV vectors for CNS tissues, and these vectors
will be deployed in the partnership. In addition, the partnership
will leverage 4DMT’s AAV product design and engineering,
manufacturing, clinical and regulatory development expertise.
“CNS disorders include some of the most devastating diseases,
many of which have a genetic origin. The technology to effectively
edit the underlying genetic mutations within these diseases did not
exist until an AAV compatible genomic editing technology was
developed. Utilizing our expansive toolbox of AAV compatible
genomic editors, we aim to advance the development of potential
lifesaving therapies for CNS diseases, and we’re delighted to
partner with 4DMT as a part of this mission,” said Devyn Smith,
Ph.D., Chief Executive Officer of Arbor. “4DMT’s proprietary,
customized and evolved AAV vectors potentially provide the best
delivery vehicles for our gene editing payloads to target select
regions of the brain that we believe could provide meaningful
benefits to patients in both rare and large CNS patient
populations.”
“The recent landmark FDA approval of the world’s first
CRISPR-based treatment is a revolutionary event for genetic
medicines and the biotech industry. Gene editing technology
continues to rapidly advance and we believe the safe and efficient
delivery of these potentially transformative therapies can address
CNS diseases of high unmet need,” said David Kirn, M.D., Co-Founder
and Chief Executive Officer of 4DMT. “Arbor is at the forefront of
this rapid innovation, and they have discovered and optimized gene
editing payloads that are compact enough to be delivered within a
single AAV vector. We look forward to combining our complementary
technologies and capabilities to power a new generation of
potential genetic medicines. This collaboration provides us with
the opportunity for next-generation gene editing capabilities and
CNS products, while simultaneously allowing us to maintain our
focus on clinical development in large market ophthalmology and
pulmonology.”
About Amyotrophic Lateral Sclerosis
ALS, often called Lou Gehrig’s disease, is a progressive
neurodegenerative disease in which the motor neurons atrophy and
die, resulting in loss of the ability to speak, move, eat and,
eventually, breathe. ALS is typically fatal within approximately
two to five years of symptom onset. The disease is estimated to
impact approximately 79,000 people in the United States, EU, and
UK. There are multiple approved treatments available for ALS and
its symptoms, but there currently is no cure.
About Arbor Biotechnologies
Arbor Biotechnologies® is a next-generation gene editing company
based in Cambridge, MA. Combining the promise of CRISPR with
advanced computational AI-driven discovery, high-throughput
screening, and robust protein engineering approaches, our
scientific co-founders Feng Zhang and David Walt laid the
groundwork for our proprietary discovery engine, that has yielded
an extensive portfolio of novel genomic editors. We envision a
future of gene editing that extends beyond simple knockdowns to
include reverse transcriptases, nuclease excisions and large
insertions. This affords us the potential to treat a broad spectrum
of patients, from those with ultra-rare to the most common
genetically defined diseases. Guided by a deep understanding of the
molecular basis of disease and our access to a unique suite of
optimized genomic editors, we are rapidly advancing our development
programs with an initial focus on genetically defined
liver-mediated and CNS diseases. As we advance toward the clinic
with our first therapeutic candidate in primary hyperoxaluria type
I, we look to expand our strategic partnerships around in vivo
genomic editing across multiple therapeutic areas and ex vivo cell
therapy programs to broaden the reach of our novel gene editing
technologies. For more information, please visit: arbor.bio.
About 4DMT
4DMT is a leading clinical-stage genetic medicines company
focused on unlocking the full potential of genetic medicines to
treat large market diseases in ophthalmology and pulmonology.
4DMT’s proprietary invention platform, Therapeutic Vector
Evolution, combines the power of the Nobel Prize-winning
technology, directed evolution, with approximately one billion
synthetic AAV capsid-derived sequences to invent customized and
evolved vectors for use in our wholly owned and partnered product
candidates. Our product design, development, and manufacturing
engine helps us efficiently create and advance our diverse product
pipeline with the goal of revolutionizing medicine with potential
curative therapies for millions of patients. Currently, 4DMT is
advancing five clinical-stage and two preclinical product
candidates, each tailored to address rare and large market diseases
in ophthalmology, pulmonology, and cardiology. In addition, 4DMT is
also advancing programs in CNS through a gene editing partnership.
4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™,
and the 4DMT logo are trademarks of 4DMT.
All of our product candidates are in clinical or preclinical
development and have not yet been approved for marketing by the FDA
or any other regulatory authority. No representation is made as to
the safety or effectiveness of our product candidates for the
therapeutic uses for which they are being studied.
Learn more at www.4DMT.com and follow us on LinkedIn.
4DMT’s Forward Looking Statements:
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, including, without limitation, implied and
express statements regarding the therapeutic potential, abilities,
safety and efficacy of 4DMT’s CNS vectors, including in connection
with Arbor’s use thereof pursuant to the Collaboration Agreement,
the potential benefits or applications of 4DMT’s Therapeutic Vector
Evolution platform, including any other vectors developed through
the Therapeutic Vector Evolution platform, and the amount of
potential payments under the Collaboration Agreement. The words
"may," “might,” "will," "could," "would," "should," "expect,"
"plan," "anticipate," "intend," "believe," “expect,” "estimate,"
“seek,” "predict," “future,” "project," "potential," "continue,"
"target" and similar words or expressions are intended to identify
forward-looking statements, although not all forward-looking
statements contain these identifying words. Any forward looking
statements in this press release are based on management's current
expectations and beliefs and are subject to a number of risks,
uncertainties and important factors that may cause actual events or
results to differ materially from those expressed or implied by any
forward-looking statements contained in this press release,
including risks and uncertainties that are described in greater
detail in the section entitled "Risk Factors" in 4D Molecular
Therapeutics’ most recent Annual Report on Form 10-K and Quarterly
Report on Form 10-Q, as well as any subsequent filings with the
Securities and Exchange Commission. In addition, any
forward-looking statements represent 4D Molecular Therapeutics'
views only as of today and should not be relied upon as
representing its views as of any subsequent date. 4D Molecular
Therapeutics explicitly disclaims any obligation to update any
forward-looking statements. No representations or warranties
(expressed or implied) are made about the accuracy of any such
forward looking statements.
Contacts:
Arbor Media:
Peg RusconiVerge Scientific
Communicationsprusconi@vergescientific.com
4DMT Media:
Katherine SmithEvoke CanaleKatherine.Smith@evokegroup.com
4DMT Investors:
Julian PeiHead of Investor Relations and Corporate
CommunicationsInvestor.Relations@4DMT.com267-644-5097
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