Sanofi, SIRION Enter Exclusive AAV Agreement

SIRION and Sanofi

Sanofi has entered an exclusive licensing and collaboration agreement with SIRION Biotech to develop tissue-selective adeno-associated virus (AAV) vectors for next-generation gene therapies in broad therapeutic categories, according to a statement from SIRION, a German company focused on gene delivery technology.

“We are delighted to be collaborating with Sanofi,” Christian Thirion, PhD, CEO and Co‑founder of SIRION, said in a statement. “Sanofi is an ideal partner with decades-long experience in treating a multitude of disorders and exploring viral vectors as a therapeutic modality. AAV vectors with improved efficacy can enable quickly entering into clinical trials with efficient, safe low doses and scalable therapeutic candidates and can expand the impact of gene therapies.”

The therapies developed will be geared toward disorders affecting major organs, according to the release. The collaboration will focus on developing new and modified AAV capsids, with a focus on safety and improved specificity and gene delivery efficiency.

“We believe that through this exclusive partnership with Sanofi within these large disease fields, AAV-based gene therapies will provide novel treatment options to many millions of patients worldwide suffering from these diseases," Sabine Ott, senior vice president BD & Licensing at SIRION, said in a statement.

SIRION is focused on vector optimization and manufacturing, across various gene editing technologies, with a focus on enhanced vector designs and capsid optimization. To achieve these goals, in 2018, SIRION formed a preferred partnership agreement with Heidelberg University Hospital and Dirk Grimm, PhD, a pioneer in AAV therapy. “This preferred partnership agreement underpins our long-lasting fruitful collaboration with SIRION and accelerates our efforts to leverage our expertise in AAV capsid optimization toward the clinic for a variety of disorders," Grimm, professor for Viral Vector Technologies, Medical Faculty at University of Heidelberg, said at the start of the partnership.

In addition to optimization and discovery, SIRION has an established system for manufacturing all common AAV serotypes that follows good manufacturing practice standards. Moreover, the company notes that its manufacturing processes are prepared for scaling, to take agents from the research and development phase to the clinic.

“This partnership adds to our expanding toolbox of technologies in the important, emerging area of gene therapeutics,” Christian Mueller, PhD, global head of genomic medicine at Sanofi, said in a statement. “Leveraging our expertise in virus-based vaccine and viral vector manufacturing together with Prof. Grimm’s cutting-edge AAV capsid evolution technology and SIRION’s expertise and capabilities in AAV vector manufacturing will allow us to better address unmet medical needs and open new frontiers for drug discovery in genomic medicine.”

A number of other companies have entered non-exclusive licensing agreements with SIRION for its LentiBOOST transduction technology. The LentiBOOST technology focuses on the optimization of cell specific transduction with lentivirus vectors. These methods increase the permeability of the cell membrane using enhancers, to facilitate easier access to the nucleus. Early evidence has shown this to be a non-toxic, highly effective approach. In 2020, the company signed agreements with Mustang Bio and Beam Therapeutics and in early January it also entered an agreement with Cellectis.