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Abeona Therapeutics Announces Support of EB Research Partnership's Venture Into Cures Event to Raise Awareness for Education and Research of Epidermolysis Bullosa

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Abeona Therapeutics (Nasdaq: ABEO) announced its sponsorship of the virtual event Venture Into Cures on November 18, 2021, supporting the EB Research Partnership's mission to find a cure for epidermolysis bullosa (EB). The event will feature stories from EB patients, world-renowned speakers, and celebrity performers to raise funds for research. Abeona is advancing its Phase 3 VIITAL™ study of EB-101, a promising treatment for recessive dystrophic epidermolysis bullosa, with the goal of addressing the urgent medical need faced by patients.

Positive
  • Sponsorship of Venture Into Cures raises awareness and funds for EB research.
  • Advancing Phase 3 VIITAL™ study of EB-101, targeting a severe form of EB with no current approved treatment.
  • EB-101 has received Rare Pediatric Disease Designation from the U.S. FDA.
Negative
  • The absence of approved treatments for RDEB indicates a high unmet medical need.

NEW YORK and CLEVELAND, Nov. 18, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced its sponsorship of the second annual Venture Into Cures, a virtual event supporting EB Research Partnership's mission to find a cure for epidermolysis bullosa (EB). The event will take place virtually on November 18, 2021 at 8:00 p.m. ET/ 5:00 p.m. PT.

The one-hour show will feature inspiring stories about individuals and families living with EB and bring together world-renowned speakers alongside appearances and performances from a lineup of celebrity supporters, to educate viewers about EB and raise funds for critical research towards finding treatments and cures for EB. Venture Into Cures will stream globally for free at www.ventureintocures.org.

“We are thankful for the opportunity to continue collaborating with EB Research Partnership and are excited to support this important event,” said Vish Seshadri, Ph.D., M.B.A., Chief Executive Officer of Abeona. “Venture Into Cures provides the general public with an engaging opportunity to learn about this devastating disease and shines a light on the urgent unmet medical need the EB community faces. We are working with urgency to meet this need by investigating EB-101, an innovative treatment for patients with recessive dystrophic epidermolysis bullosa, the most severe form of EB. We are advancing our pivotal Phase 3 VIITAL™ study of EB-101 in collaboration with researchers at Stanford University Medical Center and UMass Memorial Medical Center and aim to bring this promising investigational therapy to RDEB patients who currently have no adequate treatment options.”

About Recessive Dystrophic Epidermolysis Bullosa
Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

About EB-101
EB-101 is an autologous, gene-corrected cell therapy currently being investigated in Abeona’s pivotal Phase 3 VIITAL™ study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITAL™ study is a randomized clinical trial enrolling 10 to 15 RDEB patients with approximately 35 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver the COL7A1 gene into a patient’s own skin cells (keratinocytes and its progenitors) and transplanting those cells back to the patient. EB-101 is designed to enable normal Type VII collagen expression, which is believed to facilitate wound healing. The U.S. FDA has granted Rare Pediatric Disease Designation for EB-101. Abeona produces EB-101 for the VIITAL™ study at its fully integrated gene and cell therapy manufacturing facility in Cleveland, OH.

About EB Research Partnership
Founded in 2010 by a dedicated group of parents and Jill and Eddie Vedder, EB Research Partnership (EBRP) is the largest global non-profit dedicated to funding research aimed at treating and ultimately curing Epidermolysis Bullosa (EB), a group of devastating and life-threatening skin disorders that affect children from birth. Working around the clock with offices in the US and Australia, EBRP utilizes an innovative venture philanthropy business model. When making a grant to a research project, they retain the added upside of generating a recurring revenue stream if the therapy or product is commercially successful, then use the return on investment to fund additional EB research until a cure is found. To learn more, visit www.ebresearch.org.

About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona’s clinical programs include EB-101, its investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel investigational AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeona’s fully integrated gene and cell therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and planned commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

Forward-Looking Statements
This 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. We have attempted to identify forward-looking statements by such terminology as “may,” “will,” “believe,” “estimate,” “expect,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. 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 the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, 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 disclosed in the Company’s most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.


FAQ

What is the date of the <em>Venture Into Cures</em> event sponsored by Abeona Therapeutics?

The Venture Into Cures event will take place on November 18, 2021.

What is the purpose of the <em>Venture Into Cures</em> event?

The event supports the EB Research Partnership's mission to find a cure for epidermolysis bullosa (EB) and raises funds for critical research.

What is EB-101 and its significance in the treatment of recessive dystrophic epidermolysis bullosa?

EB-101 is a gene-corrected cell therapy being investigated in the Phase 3 VIITAL™ study for treating recessive dystrophic epidermolysis bullosa, a severe form of EB.

What designation has the U.S. FDA granted to EB-101?

The U.S. FDA has granted EB-101 Rare Pediatric Disease Designation.

How is Abeona Therapeutics involved with the EB Research Partnership?

Abeona Therapeutics sponsors the Venture Into Cures event, collaborating with EB Research Partnership to raise awareness and funds for EB research.

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