Gene therapy upstart bags a $22M A round for neurodegenerative work

A rendering of Lysogene's SAF-301 gene therapy viral vector--Courtesy of Lysogene

You can add another upstart to the swelling list of gene therapy biotechs hitting the industry radar around the world. Sofinnova stepped in to lead the $22 million A round for Paris-based Lysogene, which is already in the clinic with a new therapy for rare cases of Sanfilippo syndrome, with plans to branch out into other neurodegenerative conditions.

Lysogene CEO Karen Aiach

Lysogene got started 5 years ago with some seed money and says that last year it successfully tested its lead gene therapy, SAF-301, in a Phase I/II study for the lethal Sanfilippo syndrome, using a surgically inserted viral vector to introduce a functional SGHS gene to replace a dysfunctional one with the aim of slowing or stopping the disease. Sanfilippo is a lysosomal storage disorder, a rare genetic disease that afflicts about one out of every 100,000 children. And it has attracted the attention of a variety of drug developers.

The biotech was founded by Karen Aiach, an Arthur Andersen vet and financial consultant. Co-founder Olivier Danos, a research executive at Kadmon, the biotech Sam Waksal set up after getting out of prison, is the chief scientific adviser at Lysogene. Danos is the former director of the Gene Therapy Consortium of University College of London.

Gene therapy has had its ups and dramatic downs over the years, but recently it's mostly been on the upswing. Bluebird bio ($BLUE) is one of several gene therapy specialists in the field and heavyweight biotech Regeneron ($REGN) just days ago signed up for a high-profile collaboration with Avalanche Biotechnologies. UniQure ($QURE), which won an approval for the world's first gene therapy, is also focused on Sanfilippo syndrome. Newcomers in the gene therapy field also include Spark Therapeutics and GenSight, another Paris-based startup. Investors clearly feel that much of the risk has been taken out of the equation, angling to get in early on the new ground floor for an older technology.   

Sofinnova got this party started with its seed money and Bpifrance, through its InnoBio fund, and Novo Seeds joined in on the A round. The three funds will be represented on the board by Rafaèle Tordjman, Chahra Louafi and Henrijette Richter.

"Today, we are in a strong position to successfully face our challenges," says Aiach in a statement. "The company  is preparing the pivotal clinical study for its lead product and quickly plans on developing a second product addressing another genetic pathology involving the central nervous system, a field where patients' and families' needs still remain highly unfulfilled."

- here's the release