New SmiLe company rAAVen Therapeutics has its first customer

SmiLe Incubator

rAAVen Therapeutics designs and validates specific viral vectors for pharmaceutical companies that develop gene therapies. Now, the company has signed an agreement with an American customer, Myrtelle Inc., a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, and will use its unique technology to develop a specific vector of a modified “adeno-associated virus” (AAV) for the customer.

Gene therapy, a modern form of treatment falling under the rubric “advanced therapy medicinal products” (ATMP), is progressing rapidly, despite the relative newness of the technology. Gene therapy is used for diseases caused by inherited or acquired defects in the genetic material within the body's cells. To date, about 30 drugs have been approved by pharmaceutical authorities, five of which use AAV. In cases where viral vectors are used for direct injection into patient organs, vectors of adeno-associated virus (AAV) are most commonly involved.

rAAVen Therapeutics uses its expertise in modifying viral vectors for treatment of a broad spectrum of diseases to design and produce new viral vectors to enable customers to deliver precision treatment of cells in various organs, such as the brain or the liver. The company has developed a novel technology and knowledge concerning the interactions between peptides and those cells that comprise the target for the treatment in question. Thanks to its unique screening methodology, the company is able to produce a higher proportion of functioning vectors than many of its competitors.

“Interest in AAV technology is strong – globally, around 15,000 research groups work with AAV in clinical applications. Many smaller companies prefer to collaborate with other small companies like ours, and last summer we signed an agreement with our first customer. We are thrilled with the support we are now receiving from SmiLe’s incubator program and for access to SmiLe’s network and laboratories, including its brand-new virus lab. Since we are a startup company, we would never have been able to do this on our own,” says Marcus Davidsson, founder and chief operating officer of rAAVen Therapeutics.

“It’s extremely exciting to welcome yet another company with innovative technology pertaining to the field of advanced therapy medicinal products (ATMP), one of the most important therapeutic areas of the future. The company uses our well-equipped laboratories, which we regularly upgrade and which all of the companies may jointly use,” says Ebba Fåhraeus, CEO of SmiLe Incubator.


About rAAVen Therapeutics: We want to help pave the way for next generation of gene therapies, and have the expertise and commitment to generate novel AAV capsids that can be used to tackle a wide array of disorders. Using state of the art methodologies within cloning, virus variant production and sequencing, we build novel viral vectors for precision targeting of defined cell populations or with desired properties.

About Myrtelle: Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative and genetic hearing loss diseases. For more information, please visit the Company’s website at: