After spending some time in Sanofis pipeline, a gene therapy for a genetic eye disease that causes blindness is now back in the hands of its creators at Atsena Therapeutics. And on Wednesday, the North Carolina-based startup landed a $55 million Series A to push it through a Phase I/II trial.

Atsenas founders, power couple Shannon and Sanford Boye, did some early work on the gene therapy at the University of Florida in the early 2000s, before it was licensed by Sanofi. The biotech launched this year with $8 million in funding, then licensed the candidate from Sanofi for an undisclosed amount in July.

Its kind of like a homecoming for them, Atsena CEO Patrick Ritschel said of Shannon and Sanford Boye, who now serve as CSO and chief technical officer, respectively.

The lead candidate is designed to treat GUCY2D-associated Leber congenital amaurosis, or LCA1 a disease that affects the retina and is a leading cause of blindness in children. The drug uses an AAV capsid to deliver the target GUCY2D gene, the idea being that if you provide a replacement gene, it will encode the correct form of the protein.

This is a very good target for gene therapy. Its monogenic. It has a well-defined single gene characteristic. Importantly, the structure of the retina is well-preserved, so we think that the likelihood of success in gene therapy should also be quite high because the cells are there, theyre just not functional, Ritschel said.

The Phase I/II is enrolling 15 participants total over five cohorts, with completion of enrollment expected in late 2021 or early 2022. Then its a 52-week readout, Ritschel said, with a one-year follow-up. The Series A should carry the candidate to an efficacy readout, according to Atsena.

In addition to supporting that trial, the financing will also be used to build out the team, complete manufacturing development for Phase III, and advance the biotechs preclinical programs toward an IND. Atsena has a preclinical candidate for MYO7A-associated Usher syndrome (USH1B) that was also developed in the Boyes labs, and another mystery candidate that it has yet to release details on. And Ritschel is looking to add at least another 20 staffers by 2021.

Spark Therapeutics won approval for their own ophthalmic gene therapy back in 2017. The drug, Luxturna, treats patients with inherited retinal disease due to mutations in both copies of theRPE65 gene. In some cases, a defect in that gene results in LCA.

The field gained a Big Pharma player in October, when the Novartis Institutes for BioMedical Research bought out Cambridge, MA-based startup Vedere Bio, which Cyrus Mozayeni built around academic work from Ehud Isacoff and John Flannery at Cal-Berkeley to find a gene therapy approach to pan-genotypic vision restoration in patients with photoreceptor-based blindness. Novartis paid $150 million, with another $130 million on the table in milestones.

Were really excited, Ritschel said. I think that this is a company that has got a lot of programs, very solid science behind it. Its got great founders, strong support from the University of Florida where a lot of the technology originated from. And I think were looking forward to really building things out.

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After reeling back a gene therapy from Sanofi, a North Carolina upstart bags enough money to do something about it - Endpoints News