Atsena Therapeutics

This new video from the National Institutes of Health spotlights the incredible scientific and human impact of vision research, featuring Atsena’s Founder and Chief Scientific Officer, Dr. Shannon Boye, whose foundational work is helping pave the way for #InheritedRetinalDisease #GeneTherapy treatments that were once unimaginable.   As we pursue our mission to develop life-changing genetic medicines, we’re proud to build on this legacy and work alongside organizations like the National Eye Institute (NEI) and Association for Research in Vision and Ophthalmology (ARVO), which share our commitment to improving the lives of people with inherited retinal diseases. 🔗 Watch here: https://lnkd.in/e-2wz5tJ

We’re excited to announce that adult dosing has been completed in Part B of the LIGHTHOUSE #ClinicalTrial evaluating our investigational ocular #GeneTherapy, ATSN-201, to treat the #InheritedRetinalDisease X-linked retinoschisis (#XLRS). Following favorable preliminary data, pediatric dosing is expected to begin in Q4 2025, with pivotal enrollment planned for Q1 2026 and a BLA submission anticipated in early 2028. This milestone reflects important progress toward our mission of developing life-changing ocular gene therapies to prevent or reverse blindness. 💙 Learn more: https://lnkd.in/efHcRyuF Visual description: The graphic features a professional headshot of Kenji Fujita, MD, Atsena’s Chief Medical Officer, alongside a quote about favorable preliminary safety data and early readouts from the ATSN-201 study in XLRS. 

⚾ Nothing beats a night at the ballpark with #TeamAtsena! From the office to the stands, we’re grateful for the chance to spend time together outside of work, cheer on the home team, and celebrate the people who make our mission possible. Here’s to teamwork—on and off the field. Visual description: A group of Atsena members is gathered together indoors in a baseball stadium suite with the field visible through the windows behind them.

We’re inspired by Maverick (Mav) Raulerson's powerful story featured by the Foundation Fighting Blindness—a moving testament to resilience and hope when living with the #InheritedRetinalDisease X-linked retinoschisis (#XLRS). Patients like Mav remind us why our work to advance life-changing ocular #GeneTherapies to reverse and prevent blindness is so important. Watch the video below to learn more about Mav’s journey.

August is Children’s Eye Health and Safety Month – a reminder that healthy vision is essential for children’s learning, safety and development.    According to the American Academy of Ophthalmology (AAO), vision problems are among the most common disabling conditions in childhood, yet many go undetected. Early diagnosis and treatment can make a difference, helping children succeed in school, stay safe during play and fully engage with the world around them. At Atsena, we’re advancing ocular #GeneTherapy treatments that address #InheritedRetinalDiseases that can cause blindness beginning in childhood, including Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1) and X-linked retinoschisis (#XLRS). Read more from the AAO on the importance of children’s eye screenings: https://lnkd.in/d-WNDHf Visual description: The image features a split design: on the left, a purple background with white bold text reads “August is Children’s Eye Health & Safety Month” above the Atsena logo. On the right, a young girl sits while an eye care professional performs an eye exam. 

Our CEO, Patrick Ritschel, recently participated in the Chardan 3rd Virtual Ophthalmology Summit, which focused on innovative approaches to treating rare ophthalmic diseases, including #InheritedRetinalDiseases. Patrick shared insights on Atsena’s novel AAV.SPR capsid, used in our investigational first- and best-in-class #ocular #GeneTherapy ATSN-201 for X-linked retinoschisis (#XLRS), and discussed the latest clinical progress on the ongoing development of ATSN-101, the first gene therapy being studied to treat Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). 🎥 Watch the conversation: https://lnkd.in/dF8RhFPq

We’re excited to see our Founder & CSO, Dr. Shannon Boye, featured as an Industry Insider in the July/August issue of Ophthalmology Management!    In the article, Shannon discusses the progress of our two investigational #GeneTherapy programs for #InheritedRetinalDiseases—first-in-class ATSN-101 for LCA1 and first- and best-in-class ATSN-201 for X-linked retinoschisis (#XLRS)—as well as broader trends shaping the ocular gene therapy landscape.   🔗 Read the full feature: https://lnkd.in/gm5CUJW6  

We’re pleased to share that the FDA has agreed to expand our ongoing LIGHTHOUSE study of ATSN-201, a first- and best-in-class #GeneTherapy for X-linked retinoschisis (#XLRS) into a continuous Phase 1 / 2/ 3 clinical trial, providing a clear pathway to approval without the need for an additional registrational study.   This alignment: ✅ Accelerates our timeline by ~1.5 years ✅ Builds on strong safety and efficacy data from Part A ✅ Positions ATSN-201 to be the first gene therapy—and first treatment— evaluated in a pivotal trial for XLRS   💡 Pivotal enrollment begins Q1 2026 📄 BLA submission anticipated in early 2028 Read more: https://lnkd.in/eU9T2XsC   We’re grateful for the FDA’s guidance as we advance toward delivering a potential one-time treatment for this #InheritedRetinalDisease. Visual description: This image features a quote from Patrick Ritschel, Atsena’s CEO, on the right side over a purple-to-blue gradient background with a dotted pattern. The quote highlights the significance of an FDA agreement accelerating the development of ATSN-201, a gene therapy for XLRS, and its potential to become the first available treatment for the inherited retinal disease. On the left, there is a photo of Mr. Ritschel smiling with arms crossed, framed in a circular design with blue and orange accent arcs. The Atsena logo appears at the bottom right.  

As this week comes to a close, we want to express our support for individuals living with combined hearing and vision loss, known as #DeafBlindness. Deafblind Awareness Week is a time to recognize their experiences, the unique challenges they face, and the tailored support they deserve.   At Atsena, our mission to restore sight through gene therapy is deeply rooted in improving quality of life for people affected by #InheritedRetinalDiseases, including those in the deafblind community. We stand with advocates, families, and individuals working to build a more inclusive, accessible world.   Learn more about deafblindness from @Helen Keller Services: https://lnkd.in/e8ztHj9   Visual description: This image features a deep purple background with curved blue and orange accents in the corners. Centered prominently is the text "DeafBlind" in bold, dark purple on a white rounded rectangle, with "AWARENESS WEEK" written below in bright orange capital letters. The top left corner displays the Atsena logo in white.

Thank you, BIO 2025! Our CEO, Patrick Ritschel, joined a dynamic panel at the BIO International Convention in Boston to discuss the future of treating blinding eye diseases. Hosted as part of the Next Generation Biotherapeutics track, the panel explored the evolving landscape of therapies targeting the retina and cornea and keeping patients at the center of innovation.   Special thanks to moderator Jason Menzo of Foundation Fighting Blindness and the fellow panelists for an insightful and engaging discussion.   We’re proud to be part of this community advancing meaningful change for those affected by #InheritedRetinalDiseases.   Visual description:   The first photo captures the live panel discussion with the six men at the BIO International Convention, including Atsena CEO Patrick Ritschel (seated on the far right), in front of a backdrop that reads “BIO International Convention.”   The second photo was taken at the BIO International Convention and features the BIO panel group, including Atsena CEO Patrick Ritschel on the far right of the picture.