Abeona Therapeutics to Report New Preclinical Data Demonstrating Therapeutic Potential of ABO-401 for Treatment of Cystic Fib...
April 30 2019 - 8:00AM
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today will report new preclinical data
demonstrating that ABO-401, the Company’s novel gene therapy for
cystic fibrosis (CF), efficiently delivered a highly-expressed,
functional copy of human mini-CFTR (hCFTR) to the lung of CF mice
and restored CFTR function in human CF patient nasal and bronchial
epithelial cells. The data will be presented this evening at the
American Society of Gene and Cell Therapy 22nd Annual Meeting in
Washington, D.C.
“These encouraging preclinical data add to the growing body of
evidence suggesting ABO-401 may address the challenges in lung
delivery and transgene expression that have limited the advancement
of gene therapy for CF patients,” said Timothy J. Miller, Ph.D.,
President and Chief Scientific Officer. “ABO-401 is a promising
candidate from our AIMTM capsid library en route to IND-enabling
studies that may ultimately change the landscape of CF treatment by
introducing one-time gene therapy.”
ABO-401 has a regulatable human mini-CFTR gene that is
efficiently packaged into one of the Company’s next-generation AIM™
library capsids, AAV204. In this and other preclinical studies,
ABO-401 restored CFTR expression and chloride conductance in airway
epithelia, the main cells of the lung that contribute to CF
pathology in humans.
These new data demonstrated that ABO-401 efficiently delivered a
highly-expressed, functional copy of hCFTR to the lungs of CF mice
and restored CFTR function in nasal and bronchial epithelial cells
of human donor cells with the delta-F508 mutation, the most common
mutation of CF. ABO-401 transduced human CF nasal and bronchial
epithelial cells, with CFTR-specific change in short-circuit
current that was comparable or superior to existing modulator
therapy in these same cells. Robust expression of AAV204 in the
lungs of CF mice was observed and demonstrated that the AAV204
capsid was equally or more efficient at delivering gene expression
cassettes to the lung compared to other naturally-occurring AAV
capsids. Further, the data demonstrated that ABO-401 restored
CFTR-specific nasal potential difference in CF mice, and that the
ABO-401 gene expression cassette makes a fully-processed CFTR.
About ABO-401ABO-401 is a novel gene therapy in
development for the treatment of patients with cystic fibrosis
(CF), a progressive genetic disease that results in persistent lung
infections and limits the ability to breathe over time. Preclinical
data demonstrate that ABO-401 is delivered to the lung using a
next-generation AIMTM vector. ABO-401 targets airway cells and
corrects the underlying CF chloride channel deficit and addresses
all CF mutations, including the most common CF mutation,
delta-F508.
About the AIMTM Vector PlatformAbeona is
developing the AIM™ Vector Platform: a next generation of
adeno-associated virus (AAV) capsids for use in gene therapies. The
AIM™ capsid library can utilize AAV biology to selectively target
delivery of genetic payloads to the central nervous system, lungs,
eye, muscle, liver and other tissues. AIMTM vectors are
non-virus-producing and have shown the potential to evade the
immune responses generated by exposure to naturally-occurring AAV
vectors. The Company’s AIMTM library contains more than 100 capsids
with tissue tropisms selected for their potential to target a wide
range of organs and multiple routes of delivery.
About Abeona Therapeutics Abeona Therapeutics
Inc. is a clinical-stage biopharmaceutical company developing gene
and cell therapies for serious diseases. The Company’s clinical
programs include EB-101, its autologous, gene-corrected cell
therapy for recessive dystrophic epidermolysis bullosa, as well as
ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo
syndrome types A and B (MPS IIIA and MPS IIIB). The Company’s
portfolio of AAV9-based gene therapies also features ABO-202 and
ABO-201 for CLN1 disease and CLN3 disease, respectively. Its
preclinical assets include ABO-401, which uses the novel AIM™ AAV
vector platform to address all mutations of cystic fibrosis. Abeona
has received numerous regulatory designations from the FDA and EMA
for its pipeline candidates and is the only company with
Regenerative Medicine Advanced Therapy designation for two
investigational therapies (EB-101 and ABO-102). For more
information, visit www.abeonatherapeutics.com.
Forward Looking StatementThis
press release contains certain statements that are forward-looking
within the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended, and that involve risks and uncertainties. These
statements include statements regarding our pipeline including the
potential for the AIM™ vector platform in the treatment of cystic
fibrosis, including the results of pre-clinical studies and
initiation of clinical studies, and the company’s goals and
objectives. We have attempted to identify forward looking
statements by such terminology as “may,” will,” “anticipate,”
“believe,” “estimate,” “expect,” “intend,” and similar
expressions.
Actual results may differ materially from those indicated by
such forward-looking statements as a result of various important
factors, numerous risks and uncertainties, including but not
limited to: continued interest in our rare disease portfolio, our
ability to initiate and enroll patients in clinical trials, the
impact of competition, the ability to secure licenses for any
technology that may be necessary to commercialize our products, the
ability to achieve or obtain necessary regulatory approvals, the
impact of changes in the financial markets and global economic
conditions, risks associated with data analysis and reporting, and
other risks as may be detailed from time to time in the Company’s
annual reports on Form 10-K and quarterly reports on Form 10-Q and
other reports filed by the Company with the Securities and Exchange
Commission. The Company undertakes no obligation to revise
the forward-looking statements or update them to reflect events or
circumstances occurring after the date of this presentation,
whether as a result of new information, future developments or
otherwise, except as required by the federal securities laws.
Investor Contact: Sofia Warner Senior Director,
Investor Relations Abeona Therapeutics +1 (646) 813-4710
swarner@abeonatherapeutics.com
Media Contact: Scott Santiamo Director,
Corporate Communications Abeona Therapeutics +1 (718) 344-5843
ssantiamo@abeonatherapeutics.com
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