Abeona Therapeutics to Present at the Jefferies 2017 Global Healthcare Conference
June 02 2017 - 8:05AM
Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage
biopharmaceutical company focused on developing novel gene
therapies for life-threatening rare diseases, today announced
President and CEO, Timothy J. Miller, Ph.D., will present at the at
the Jefferies 2017 Global Healthcare Conference in New York City,
NY.
Event: Jefferies 2017 Global Healthcare
ConferenceDate: Wednesday, June
7thPresenter: Timothy J. Miller, Ph.D., President
& CEOPresentation Time: 3:30 PM – 3:55 PM
ETRoom Name: 1 BallroomLocation:
New York City, NYWebcast:
http://wsw.com/webcast/jeff105/abeo
Abeona Recent Highlights
ABO-102 gene therapy program for
Sanfilippo syndrome Type A:
- Abeona announced top-line data for Phase 1/2 MPS IIIA gene
therapy trial at ASGCT -- Positive dose response in central
nervous system with 60.7% (+/- 8.8%) reduction of disease-causing
heparan sulfate GAG observed in Cohort 2 -- Reduction of
disease manifestation observed in decreased liver volume
of 14.81% (+/- 1.2%) -- ABO-102 well-tolerated in six
subjects through more than 1,100 days cumulative follow-up with no
Serious Adverse Events -- Cohort 1 demonstrated stabilized or
improved Leiter Nonverbal IQ scores at six months
- Abeona received regulatory approval to initiate ABO-102 gene
therapy clinical trial in Australia for patients with MPS IIIA
EB-101 gene therapy program for severe
form of Epidermolysis Bullosa:
- Abeona provided an update for Phase 1/2a gene therapy to the
Society for Investigative Dermatology Conference -- EB-101
demonstrated significant wound healing (defined as greater than 50%
healed) in 100% of treated wounds (36/36) at 3 months; 89% (32/36)
at 6 months, 83% (20/24) at 12 months, 88% (21/24) at 24 months and
100% (6/6) at 36 months post-administration -- Clinical
endpoints supported by data from Natural History Study observations
from 1,436 wounds in 128 patients with Recessive Dystrophic
Epidermolysis Bullosa (RDEB)
- Abeona was granted Orphan Drug Designation for EB-101 in
Epidermolysis Bullosa by the European Medicines Agency (EMA)
Committee for Orphan Medicinal Products
- Abeona was granted Orphan Drug Designation and Rare Pediatric
Designations for EB-101 in Epidermolysis Bullosa by the FDA,
enabling Priority Review Voucher
About Abeona: Abeona
Therapeutics Inc. is a clinical-stage biopharmaceutical company
developing gene therapies for life-threatening rare genetic
diseases. Abeona's lead programs include ABO-102 (AAV-SGSH), an
adeno-associated virus (AAV) based gene therapy for Sanfilippo
syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts)
for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is
also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B
(MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten
disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile
Batten disease (INCL), EB-201 for epidermolysis bullosa (EB),
ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302
using a novel CRISPR/Cas9-based gene editing approach to gene
therapy for rare blood diseases. In addition, Abeona has a
plasma-based protein therapy pipeline, including SDF Alpha™
(alpha-1 protease inhibitor) for inherited COPD, using its
proprietary SDF™ (Salt Diafiltration) ethanol-free process. For
more information, visit www.abeonatherapeutics.com.
Investor Contact:Christine Silverstein Vice
President, Investor Relations Abeona Therapeutics Inc. +1
(212)-786-6212 csilverstein@abeonatherapeutics.com
Media Contact:Andre’a Lucca Vice President,
Communications & Operations Abeona Therapeutics Inc. +1
(212)-786-6208alucca@abeonatherapeutics.com
This press release contains certain
statements that are forward-looking within the meaning of Section
27a of the Securities Act of 1933, as amended, the expected receipt
of a Priority Review Voucher and that involve risks and
uncertainties. These statements include, without limitation, our
plans for continued development and internationalization of our
clinical programs, that patients will continue to be identified,
enrolled, treated and monitored in the EB-101 clinical trial, and
that studies will continue to indicate that EB-101 is
well-tolerated and may offer significant improvements in wound
healing. These statements are subject to numerous risks and
uncertainties, including but not limited to continued interest in
our rare disease portfolio, our ability to enroll patients in
clinical trials, the impact of competition; the ability to develop
our products and technologies; the ability to achieve or obtain
necessary regulatory approvals; the ability to secure licenses for
any technology that may be necessary to commercialize our
products; the impact of changes in the financial markets
and global economic conditions; and other risks as may be detailed
from time to time in the Company's Annual Reports on Form 10-K and
other reports filed by the Company with the Securities and Exchange
Commission. The Company undertakes no obligations to make any
revisions to the forward-looking statements contained in this
release or to update them to reflect events or circumstances
occurring after the date of this release, whether as a result of
new information, future developments or otherwise.
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