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NEW YORK and CLEVELAND and DALLAS, August 17, 2020 (GLOBE NEWSWIRE) – Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully integrated leader in gene and cell therapy, and Taysha Gene Therapies, a patient-centered gene therapy company mission to eradicate monogenic CNS disease, today announced that it has entered into licensing and inventory purchase agreements for ABO-202, an adeno-associated virus (AAV) gene therapy for CLN1 disease (also known as childhood Batten disease). The agreements grant Taysha exclusive worldwide rights to intellectual property developed by scientists at the University of North Carolina at Chapel Hill (UNC) and Abeona, as well as Abeona’s expertise in research, development and the manufacture of ABO-202. The therapy was originally developed in the lab of Steven Gray, Ph.D., associate professor at UT Southwestern Medical Center (formerly with UNC) and chief scientific advisor for Taysha Gene Therapies. Abeona continued to advance the program, including the development of the Phase 1/2 clinical trial protocol and manufacturing process, and received FDA clearance for its Investigational New Drug (IND) application for a phase 1/2 clinical trial which should enter the clinic. in 2021.

Under the terms of the deal, Taysha will make upfront cash payments to Abeona of $ 7 million, including an initial license fee of $ 3 million and an inventory purchase price of $ 4 million, including including Abeona’s CLN1 plasmid of GMP origin. In addition, Abeona is eligible to receive up to $ 56 million from Taysha upon completion of certain clinical, regulatory and commercial milestones, as well as high single-digit royalties on net sales of Taysha’s CLN1 product.

“We are delighted to partner with Taysha in the development of a potential treatment for children living with Batten disease,” said João Siffert, MD, CEO of Abeona. “At the same time, these agreements allow Abeona to continue to focus its resources on advancing its key clinical programs in the RDEB, MPS IIIA and MPS IIIB areas towards biologic license application submissions with the aim of to provide safe and effective gene and cell therapies to patients who currently have no approved treatment options.

“CLN1 is a progressive monogenic CNS disease with significant unmet medical needs, and we believe the ABO-202 data generated so far demonstrates great translational potential and offers hope for children with this devastating disorder.” Said RA Session II, President, CEO Officer and Founder of Taysha. “We are delighted to continue to work closely with Dr. Gray to rapidly advance this promising gene therapy in the clinic. “

ABO-202 is a unique, self-complementary AAV gene therapy (serotype 9) designed to deliver a functional copy of the palmitoyl-protein thioesterase 1 (PPT1) gene. Preclinical studies in a CLN1 animal model demonstrated that ABO-202 normalized survival and led to improved neurological function in affected mice. The therapy has received orphan drug and rare pediatric disease designations by the United States Food and Drug Administration (FDA) and has received orphan medicinal product designation from the European Medicines Agency.

About CLN1 disease (childhood Batten disease)
CLN1 disease, also known as infantile neuronal ceroid lipofuscinosis or infantile Batten disease, is a rare lysosomal overload disease that progresses rapidly without approved treatment. It primarily affects the central nervous system and usually manifests in the first year of life with visual impairment that can progress to blindness, progressive motor and cognitive decline, seizures and ultimately premature death. The underlying cause of the disorder is a defect in the PPT1 gene which encodes the enzyme of the same name, resulting in the accumulation of lipopigments in the cells, resulting in neuroinflammation and neurodegeneration. Some patients with CLN1 disease develop symptoms later in childhood or in adulthood; these variants are called late-infant, juvenile, or adult CLN1.

About ABO-202
ABO-202 is a new single-use gene therapy for CLN1 disease, a rare lysosomal overload disease with rapid progression without approved therapy. ABO-202 provides a functional copy of the PPT1 gene to treat the underlying gene and enzyme deficiency which results in abnormal accumulation of lipopigments and leads to neuroinflammation and neurodegeneration.

About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company that develops gene and cell therapies for serious illnesses. Abeona’s clinical programs include EB-101, its autologous gene-corrected cell therapy for dystrophic recessive epidermolysis bullosa in phase 3 of development, as well as ABO-102 and ABO-101, new gene therapies based on AAV for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in phase 1/2 of development. The Company’s AAV-based gene therapy portfolio also includes ABO-201 for CLN3 disease. Abeona’s next-generation AIM â„¢ capsids have shown the potential to improve tropism profiles for a variety of devastating diseases. Abeona’s fully functional gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal phase 3 VIITAL â„¢ study and is capable of clinically and commercially producing AAV-based gene therapies. For more information visit www.abeonatherapeutics.com.

About Taysha gene therapies
Taysha Gene Therapies is a patient-centered gene therapy company with a mission to eradicate monogenic CNS disease. We are focused on the development and commercialization of AAV-based gene therapies for the treatment of monogenic CNS diseases in rare and large patient populations. We were founded in partnership with the University of Texas Southwestern Medical Center, or UT Southwestern, to develop and commercialize transformative gene therapy treatments. In collaboration with UT Southwestern, we are developing a deep and sustainable product portfolio of 18 gene therapy product candidates, with exclusive options to acquire four additional development programs. By combining our leadership team’s proven track record in the development and commercialization of gene therapy drugs with UT Southwestern’s world-class gene therapy research capabilities, we believe we have created a powerful engine to develop transformative therapies. to dramatically improve the lives of patients. More information is available at www.tayshagtx.com.

Forward-looking statements
This press release contains certain forward-looking statements 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 which involve risks and uncertainties. . These statements include statements about the Company’s clinical trials and its product and product candidates, future regulatory interactions with regulatory authorities, and the Company’s goals and objectives. We have attempted to identify forward-looking statements by words such as “may”, “will”, “believe”, “estimate”, “expect” and similar expressions (as well as other words or expressions referring to future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by these forward-looking statements due to various important factors, many risks and uncertainties, including, but not limited to, the potential impacts of the COVID-19 pandemic on our business, our 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 bodies, the impact competition, the ability to license any technology that may be required to bring our products to market, the ability to obtain or obtain necessary regulatory approvals, the impact of changes in financial markets and economic conditions global, 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 Security and Exchange Commission. The Company does not undertake to revise any 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 if required by federal securities laws. .

Abeona Therapeutics contacts:

Investors:
Greg Gin
Vice-President, Investor Relations
Therapeutic Abeona
+1 (646) 813-4709
[email protected]

Media:
Scott Santiamo
Director, Corporate Communications
Therapeutic Abeona
+1 (718) 344-5843
[email protected]

Contacts of Taysha Gene Therapies:

Society:
Niren Shah, PharmD, MBA
Taysha’s gene therapies
[email protected]

Media:
Carolyn hawley
Canale Communications
[email protected]

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