2021-01-19| R&D

Daiichi Sankyo Collaboration Marks ViGeneron’s Second Gene Therapy Deal in a Single Month

by Judy Ya-Hsuan Lin
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On January 18th, Munich, Germany-based gene therapy company, ViGeneron announced a research collaboration with Daiichi Sankyo on a novel therapeutic protein to address an undisclosed target in a highly prevalent ophthalmic disease. Under the agreement, Daiichi Sankyo will conduct the first stage research together with ViGeneron and will have the option to negotiate future collaboration agreements for the undisclosed therapeutic target.

“There is significant unmet medical need for a sustained therapy to treat eye diseases. ViGeneron’s innovative gene therapy expertise combined with Daiichi Sankyo’s ophthalmic knowledge creates the potential to develop a sustained novel gene therapy to overcome the current limitations in treating this highly prevalent ophthalmic disease,” commented Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron. “We look forward to working with Daiichi Sankyo to drive cutting-edge innovative science and to create an effective treatment for patients.”


ViGeneron’s Specialty

ViGeneron’s pipeline is based upon two proprietary adeno-associated virus (AAV) technology platforms. The collaboration with Daiichi Sankyo is focused on a novel engineered adeno-associated vector (vgAAV) from one of the platforms, the vgAAV gene therapy vector platform.

vgAAV allows superior transduction of target cells via intravitreal injection for efficient lateral spreading and minimizing the risk of collateral damage induced by conventional subretinal injection. The other is REVeRT vector platform which targets diseases caused by mutations in large genes (>5Kb).


vgAAV Vector Platform

The vgAAV gene therapy vector platform assists in figuring out a way to pack split genes into the vector, including full-length proteins produced by splicing mRNA. This enhances the flexibility of gene insertion and the technology itself applies to work for a wider range of cell types in the retina and other tissues like the central nervous system.

In addition, AAV-based vectors do not tend to incur a strong immune response and can accurately insert a DNA sequence into the host’s genome. Moreover, their behavior is more predictable unlike retroviruses, which are likely to insert DNA at random points and potentially cause cancer. Conversely, AAVs cannot carry large size genes and thus limit its application on gene editing technology.


Foundation & Prior Collaborations

Founded in 2017, ViGeneron is a spin-off from the Ludwig-Maximilians University in Munich. It is led by a seasoned team with in-depth experience in AAV vector technology and clinical ophthalmic gene therapy programs, including CEO and co-founder Dr. Caroline Man Xu and two scientific co-founders, Professor Dr. Martin Biel and Professor Dr. Stylianos Michalakis.

In 2019, ViGeneron expressed that its pipeline addresses ophthalmic diseases that desperately require new treatments because there were no currently approved treatment options, except for their two leading drugs with undisclosed indications.

The same year, it closed a series A financing round led by WuXi AppTec, a Chinese provider of R&D and manufacturing services and Sequoia Capital China, a venture capital firm based in Beijing. Yet, the company’s next generation gene therapy platforms were still overcoming the limitations of AAV gene therapies at that time.

Early in January this year, ViGeneron announced a global collaboration and licensing agreement with Biogen to optimize and validate in vitro therapeutic candidates for the undisclosed target as well as to develop and commercialize gene therapy products based on AAV vectors.

As per the agreement The companies will work together on the in vivo proof of concept (POC). In addition to an upfront payment and R&D funding, ViGeneron will also receive development, regulatory and commercial milestone payments, as well as tiered royalties on product sales arising from the collaboration.

By Judy Ya-Hsuan Lin

Related Article: Sarepta’s Stocks Drop 50% Following Middling Trial Data for Gene Therapy



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