Flush With $63M, Durham Gene Therapy Startup Gets Ready To Scale

Shannon Boye with young patient
Co-founder Shannon Boye, Ph.D. (left) with young LCA patient and his mom.
-- Atsena Therapeutics photos

There’s a new biotech startup in town, and it’s already generating a lot of buzz.

Meet Atsena Therapeutics – a clinical-stage gene therapy company headquartered in Durham that’s on a mission to reverse and prevent blindness.

Shortly after launching last April, it paid French pharmaceutical company Sanofi for exclusive rights to a gene therapy targeting Leber congenital amaurosis (LCA), a leading cause of blindness in children.

That spurred investors to pour $55 million into Atsena -- just months after it raised $8.2 million from investors including Durham’s Hatteras Venture Partners.

CEO Patrick Ritschel
CEO Patrick Ritschel

“With the acquisition and the progress of our preclinical programs, the timing was right,” CEO Patrick Ritschel told the North Carolina Biotechnology Center.

Sofinnova Investments led the round, with Hatteras investing once again alongside new investors Abingworth and Lightstone Ventures. 

Ritschel, who previously led another Durham gene therapy company called StrideBio, said it’s now time to scale.

“We're looking to drive forward, build out our team and continue to grow. We feel confident that we will benefit patients.”

Targeting blindness in children

LCA is the most common cause of inherited blindness in children, impacting two to three per 100,000 newborns. 

Atsena’s therapy targets LCA1, one of the most frequent forms of LCA caused by mutations in the GUCY2D gene.

Atsena’s founders Shannon Boye, Ph.D., and Sanford Boye created the therapy at the University of Florida, from which Sanofi originally licensed it.

Sanford Boye
Co-founder Sanford Boye

Atsena has an ongoing Phase 1/2 clinical trial evaluating this gene therapy in LCA1 patients, and is currently enrolling patients in the second dosing cohort. 

Ritschel said funds would be used to advance the trials, “with the hopes to move those into the clinic in the next 18 to 24 months” -- as well as complete manufacturing development for Phase 3. 

In addition, the company will be expanding its team.

“We expect to add more than 20 positions this year across the company, including [research and development], regulatory, quality, and senior management,” Ritchel said.

New headquarters are also in the works.

The company is currently operating out of lab space in the Chesterfield building in downtown Durham. 

“We're down to a short list of a couple spots. We hope to have a lease signed and everything retrofitted and moved in probably in the third quarter.”

‘A gene therapy hub’

Atsena is joining the fast-growing community of cutting-edge gene- and cell-therapy companies setting up shop in the Research Triangle.

In recent months, companies like Taysha Gene Therapies and Beam Therapeutics have announced expansion projects in the region.

Meanwhile, Asklepios BioPharmaceutical (AskBio), a clinical-stage gene therapy firm based in RTP, was acquired by drug giant Bayer in a deal potentially worth $4 billion last October. 

“Clearly, this area has become a hub for gene therapy,” Ritchel said.

For Atsena, the main advantage comes down to one thing: talent.

“There a tremendous amount of talent and infrastructure in the area. With the number of folks moving to the area, you'll see that that talent base continue to grow,” he said. “My experience with StrideBio reinforced the belief that the RTP area is a great place to build a gene therapy company.”

Chantal Allam, NCBiotech Writer
Thu, 01/14/2021 - 16:04