Abeona Therapeutics庐 Announces Option Exercise by Beacon Therapeutics for Novel AAV204 Capsid for Ophthalmology Gene Therapy
Abeona Therapeutics (Nasdaq: ABEO) announced that Beacon Therapeutics has exercised its option to license Abeona's patented AAV204 capsid for potential gene therapies targeting retinal diseases. The worldwide, non-exclusive license follows their July 2024 evaluation agreement.
The AAV204 capsid, from the AIM鈩� capsid library licensed from the University of North Carolina at Chapel Hill, has demonstrated high macular and optic nerve transduction levels after para-retinal administration and can facilitate transduction of both inner and outer retina after intravitreal administration in mice and non-human primates.
Under the agreement, Beacon can use AAV204 for up to five gene or ophthalmology disease targets. Abeona will receive an undisclosed upfront license payment, potential development, regulatory, and sales milestone payments, and tiered royalties on worldwide net sales of licensed products incorporating AAV204.
Abeona Therapeutics (Nasdaq: ABEO) ha annunciato che Beacon Therapeutics ha esercitato l'opzione per ottenere la licenza del capside AAV204 brevettato di Abeona, destinato a potenziali terapie geniche per malattie retiniche. La licenza mondiale e non esclusiva segue l'accordo di valutazione siglato a luglio 2024.
Il capside AAV204, proveniente dalla libreria AIM鈩� concessa in licenza dall'Universit脿 della Carolina del Nord a Chapel Hill, ha dimostrato elevati livelli di trasduzione della macula e del nervo ottico dopo somministrazione para-retinica e pu貌 favorire la trasduzione sia della retina interna che esterna dopo somministrazione intravitreale in topi e primati non umani.
Secondo l'accordo, Beacon potr脿 utilizzare AAV204 per un massimo di cinque obiettivi genici o malattie oftalmologiche. Abeona ricever脿 un pagamento anticipato per la licenza, non divulgato, oltre a potenziali pagamenti per sviluppo, regolamentazione e traguardi di vendita, oltre a royalties a scaglioni sulle vendite nette mondiali dei prodotti concessi in licenza che incorporano AAV204.
Abeona Therapeutics (Nasdaq: ABEO) anunci贸 que Beacon Therapeutics ha ejercido su opci贸n para licenciar el caparaz贸n AAV204 patentado de Abeona para posibles terapias g茅nicas dirigidas a enfermedades retinianas. La licencia mundial y no exclusiva sigue al acuerdo de evaluaci贸n firmado en julio de 2024.
El caparaz贸n AAV204, proveniente de la biblioteca AIM鈩� licenciada por la Universidad de Carolina del Norte en Chapel Hill, ha demostrado altos niveles de transducci贸n en la m谩cula y el nervio 贸ptico tras administraci贸n para-retiniana y puede facilitar la transducci贸n tanto de la retina interna como externa despu茅s de la administraci贸n intrav铆trea en ratones y primates no humanos.
Bajo el acuerdo, Beacon puede usar AAV204 para hasta cinco objetivos g茅nicos o enfermedades oftalmol贸gicas. Abeona recibir谩 un pago inicial por la licencia, no divulgado, adem谩s de posibles pagos por desarrollo, regulatorios y por hitos de ventas, as铆 como regal铆as escalonadas sobre las ventas netas mundiales de productos licenciados que incorporen AAV204.
Abeona Therapeutics (雮橃姢雼�: ABEO)電� Beacon Therapeutics臧� 毵濍 歆堩櫂鞚� 氇╉憸搿� 頃橂姅 鞛犾灛鞝� 鞙犾爠鞛� 旃橂毳� 鞙勴暣 Abeona鞚� 韸鬼棃氚涭潃 AAV204 旌§嫓霌� 霛检澊靹犾姢 鞓奠厴鞚� 頄夓偓頄堧嫟瓿� 氚滍憸頄堨姷雼堧嫟. 鞝� 靹戈硠 牍勲弲鞝� 霛检澊靹犾姢電� 2024雲� 7鞗� 韽夑皜 瓿勳暯鞐� 霐半ジ 瓴冹瀰雼堧嫟.
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瓿勳暯鞐� 霐半澕 Beacon鞚� 斓滊寑 雼れ劘 臧滌潣 鞙犾爠鞛� 霕愲姅 鞎堦臣 歆堩櫂 韮�旯�鞐� 雽�頃� AAV204毳� 靷毄頃� 靾� 鞛堨姷雼堧嫟. Abeona電� 瓿店皽霅橃 鞎婌潃 靹犽秷 霛检澊靹犾姢 牍勳毄瓿� 鞛犾灛鞝侅澑 臧滊皽, 攴滌牅, 韺愲Г 鞚挫爼響� 歆�旮夑笀 氚� AAV204毳� 韽暔頃橂姅 霛检澊靹犾姢 鞝滍拡鞚� 鞝� 靹戈硠 靾滊Г於滌棎 霐半ジ 雼硠氤� 搿滌棿韹半ゼ 氚涥矊 霅╇媹雼�.
Abeona Therapeutics (Nasdaq : ABEO) a annonc茅 que Beacon Therapeutics a exerc茅 son option de licence du capside AAV204 brevet茅 d'Abeona pour des th茅rapies g茅niques potentielles ciblant les maladies r茅tiniennes. Cette licence mondiale et non exclusive fait suite 脿 leur accord d'茅valuation de juillet 2024.
Le capside AAV204, issu de la biblioth猫que AIM鈩� licenci茅e par l'Universit茅 de Caroline du Nord 脿 Chapel Hill, a d茅montr茅 des niveaux 茅lev茅s de transduction de la macula et du nerf optique apr猫s administration para-r茅tinienne et peut faciliter la transduction de la r茅tine interne et externe apr猫s administration intravitr茅enne chez la souris et les primates non humains.
Selon l'accord, Beacon peut utiliser AAV204 pour un maximum de cinq cibles g茅n茅tiques ou maladies ophtalmologiques. Abeona recevra un paiement initial de licence non divulgu茅, ainsi que des paiements potentiels li茅s au d茅veloppement, 脿 la r茅glementation et aux 茅tapes de vente, ainsi que des redevances progressives sur les ventes nettes mondiales des produits licenci茅s incorporant AAV204.
Abeona Therapeutics (Nasdaq: ABEO) gab bekannt, dass Beacon Therapeutics seine Option zur Lizenzierung der patentierten AAV204-Kapsid von Abeona f眉r potenzielle Gentherapien zur Behandlung von Netzhauterkrankungen ausge眉bt hat. Die weltweite, nicht-exklusive Lizenz folgt auf ihre Bewertungsvereinbarung vom Juli 2024.
Das AAV204-Kapsid aus der AIM鈩�-Kapsidbibliothek, lizenziert von der University of North Carolina in Chapel Hill, hat hohe Transduktionsraten der Makula und des Sehnervs nach para-retinaler Verabreichung gezeigt und kann nach intravitrealer Verabreichung bei M盲usen und nicht-menschlichen Primaten sowohl die innere als auch die 盲u脽ere Netzhaut transduzieren.
Im Rahmen der Vereinbarung kann Beacon AAV204 f眉r bis zu f眉nf Gen- oder ophthalmologische Krankheitsziele verwenden. Abeona erh盲lt eine nicht offen gelegte Vorauszahlung f眉r die Lizenz sowie potenzielle Entwicklungs-, Zulassungs- und Verkaufsmeilensteinzahlungen und gestaffelte Lizenzgeb眉hren auf den weltweiten Nettoumsatz der lizenzierten Produkte mit AAV204.
- Non-exclusive licensing agreement allows Abeona to explore additional ophthalmic applications of AAV204
- Agreement includes upfront payment, milestone payments, and tiered royalties
- AAV204 demonstrates promising therapeutic potential in retinal disease treatment
- License covers up to five gene or ophthalmology disease targets
- None.
Insights
Abeona secures non-exclusive AAV204 capsid license deal with Beacon, generating immediate revenue and future milestone potential while retaining technology rights.
This licensing agreement represents a significant validation for Abeona's AAV204 capsid technology. The non-exclusive nature of the deal is particularly advantageous as it allows Abeona to maintain rights for exploring additional applications and potential partnerships beyond Beacon's five targets. This preserves considerable future value potential.
The structure includes three revenue components: an upfront payment, development/regulatory/commercial milestones, and tiered royalties on product sales. While financial terms remain undisclosed, ophthalmology gene therapy deals typically command substantial economics given the high unmet need and commercial potential in retinal diseases.
AAV204's differentiated mechanism - achieving high macular and optic nerve transduction via para-retinal administration while also facilitating inner/outer retina transduction through intravitreal delivery - addresses a critical challenge in ocular gene therapy. Current limitations in delivery methods and target tissue penetration have hampered development, making this technology potentially transformative.
The deal's significance extends beyond immediate financial benefits. It demonstrates external validation of Abeona's AIM鈩� capsid library, bolsters investor confidence in their technology platform, and creates potential for additional partnerships. By retaining non-exclusive rights, Abeona maintains maximum optionality for their technology while securing near-term cash flow - an ideal scenario for biotech companies balancing platform monetization with long-term value creation.
Abeona will receive a license payment and potential development, regulatory, and sales milestones, and royalties
CLEVELAND, July 01, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that Beacon Therapeutics, an ophthalmic gene therapy company and Syncona portfolio company, has exercised its option to license Abeona's patented AAV204 capsid for use in potential gene therapies for a range of prevalent and rare retinal diseases that result in blindness. This worldwide, non-exclusive license is pursuant to the agreement between Abeona and Beacon, announced in July 2024, to evaluate the therapeutic potential of AAV204.
AAV204, a novel AAV capsid from the AIM鈩� capsid library licensed by Abeona from the University of North Carolina at Chapel Hill, has been shown to achieve high macular and optic nerve transduction levels after para-retinal administration and has also been shown to facilitate transduction of both the inner and outer retina after intravitreal administration in mice and non-human primates.
鈥淏eacon鈥檚 option exercise further validates AAV204鈥檚 potential to enable targeted delivery of gene therapies in rare and prevalent ophthalmic diseases,鈥� said Dr. Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development at Abeona Therapeutics. 鈥淥ur non-exclusive agreement with Beacon enables us to fully explore the therapeutic value of AAV204 in additional ophthalmic diseases.鈥�
The exercise of this license option concludes Beacon鈥檚 initial evaluation of the AAV204 capsid for development and commercialization of gene therapies and gives Beacon the right to use AAV204 in connection with up to five gene or ophthalmology disease targets. Under the terms of the agreement, Abeona will receive an undisclosed upfront license payment with additional payments upon the achievement of certain development, regulatory, and sales milestones, along with tiered royalties on worldwide net sales for licensed products incorporating AAV204.
About the AIM鈩� capsid library
The AIM鈩� capsid library is a collection of novel AAV serotypes that target delivery of genetic payloads to key tissues implicated in devastating genetic diseases, including the central nervous system (including the retina), lungs, eye, muscle, liver and other tissues, with potentially improved tropism profiles. AIM鈩� vectors have shown the potential to evade the immune response generated by exposure to naturally-occurring AAV vectors in preclinical studies. AAV204 is covered by U.S. Patent Nos. 10,532,110 and 10,561,743.
About Abeona Therapeutics
Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona鈥檚 ZEVASKYN鈩� (prademagene zamikeracel) is the first and only autologous cell-based gene therapy for the treatment of wounds in adults and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). The Company鈥檚 fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, Ohio serves as the manufacturing site for ZEVASKYN commercial production. The Company鈥檚 development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona鈥檚 novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit .
ZEVASKYN鈩�, Abeona Assist鈩�, Abeona Therapeutics庐, and their related logos are trademarks of Abeona Therapeutics Inc.
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 鈥渕ay,鈥� 鈥渨ill,鈥� 鈥渂elieve,鈥� 鈥渁nticipate,鈥� 鈥渆xpect,鈥� 鈥渋ntend,鈥� 鈥減otential,鈥� and similar words and 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, our ability to commercialize ZEVASKYN, the therapeutic potential of ZEVASKYN, whether the unmet need and market opportunity for ZEVASKYN are consistent with the Company鈥檚 expectations, continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with and inspections from the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; and other risks disclosed in the Company鈥檚 most recent Annual Report on Form 10-K and subsequent 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.
Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics [email protected]
FAQ
What is the purpose of Abeona Therapeutics' AAV204 capsid license agreement with Beacon Therapeutics?
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