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Atsena Therapeutics to Present at May Scientific Conferences

DURHAM, NC, April 29, 2025 – Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced it will present at the following scientific conferences in May:

Retinal Therapeutics Innovation Summit 2025 – Salt Lake City, UT
Session: Retinal Genetic Augmentation
Date and Time: Friday, May 2, 2025, 3:30-3:45 p.m. MT
Presentation Title: Advancements in IRD Gene Therapy: From the Bench to the Clinic
Presenter: Kenji Fujita, MD, Chief Medical Officer, Atsena Therapeutics

ARVO 2025 Symposium – Salt Lake City, UT
Session: Innovating Breakthroughs in Ocular Gene Therapy
Presentation Date and Time: Thursday, May 8, 2025, 8:51-9:09 a.m. MT
Presentation Title: Interim safety and efficacy data in XLRS patients validates the use of a novel, laterally spreading capsid for the treatment of retinal disease
Presenter: Lesley Everett, MD, PhD, Assistant Professor, Ophthalmology, Oregon Health & Science University

Poster Session: Retinal Function Testing and Advanced Therapies
Presentation Date and Time: Thursday, May 8, 2025, 2:00-3:45 p.m. MT
Posterboard Number: A0499
Poster Title: Quantitative Comparison of Two Physical Mobility Tests as Functions of FST and BCVA to Evaluate Therapeutic Benefit in LCA1 Patients Treated with Unilateral Gene Therapy
Presenter: Alexandra Viviann Garafalo, Research Project Manager, Perelman School of Medicine, University of Pennsylvania

American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting – New Orleans and Virtual
Session: Gene Therapy Trials – In-Vivo Gene Therapy Modification
Date and Time: Friday, May 16th, 2025, 5:15 p.m. ET
Presentation Title: Safety and Efficacy of ATSN-201 Dose Escalation in Patients with X-linked Retinoschisis (XLRS)
Presenter: Shannon Boye, PhD, Founder and Chief Scientific Officer, Atsena Therapeutics

3rd Annual Retinal Imaging Biomarkers & Endpoints Summit – Boston, MA
Session: Delving into Commercial Trends, Trial Insights, and Therapeutic Applications to Spearhead Development
Date and Time: Thursday, May 29, 2025, 2:30 p.m. ET
Presentation Title: Delving Into Emerging Insights for Trial Endpoints: Analyzing Correlations Between FST & MLMT
Presenter: Kenji Fujita, MD, Chief Medical Officer, Atsena Therapeutics

About X-linked Retinoschisis (XLRS)
XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments.

About AAV.SPR
AAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and has a favorable safety profile relative to benchmark capsids. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/.

About Atsena Therapeutics
Atsena Therapeutics (“Atsena”) is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena’s first-in-class, investigational gene therapy for Leber congenital amaurosis type 1 (LCA1) has completed a Phase 1 / 2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8). Atsena is advancing ATSN-101 toward the initiation of a global pivotal trial as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/.

Media Contact:
Gina Mangiaracina
6 Degrees
(917) 797-7904
[email protected]

Business Contact:
[email protected]

Atsena Therapeutics Granted U.S. FDA Regenerative Medicine Advanced Therapy Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis

Marks fourth FDA designation for ATSN-201, which has also received Fast Track, Rare Pediatric Disease and Orphan Drug Designations

DURHAM, NC, April 15, 2025 – Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.

“We’re honored that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-201, further underscoring its potential to address the urgent, unmet need in XLRS—a rare inherited retinal disease with no approved treatments,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “This regulatory momentum, coupled with the recent close of our oversubscribed $150 million Series C financing, reinforces our commitment to advancing meaningful gene therapies that have the potential to improve vision and quality of life for individuals living with XLRS and other inherited retinal diseases.”

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for that disease or condition. RMAT designation provides sponsors with intensive FDA guidance on efficient drug development, including the ability to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA), and other opportunities to expedite development and review.
Currently, there are no approved treatments for XLRS, which is typically diagnosed in early childhood and affects approximately 30,000 males in the U.S. and the EU. The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, a Phase I/II, dose-escalation and dose-expansion clinical trial in male patients ages six and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860).

About X-linked Retinoschisis (XLRS)
XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments.

About AAV.SPR
AAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and has a favorable safety profile relative to benchmark capsids. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/.

About Atsena Therapeutics
Atsena Therapeutics (“Atsena”) is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena’s first-in-class, investigational gene therapy for Leber congenital amaurosis type 1 (LCA1) has completed a Phase 1 / 2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8). Atsena is advancing ATSN-101 toward the initiation of a global pivotal trial as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/.

Media Contact:
Gina Mangiaracina
6 Degrees
(917) 797-7904
[email protected]

Business Contact:
[email protected]

Atsena Therapeutics Announces Oversubscribed $150 Million Series C Financing to Further Advance Ocular Gene Therapy Programs

Financing led by new investor Bain Capital with participation from new investor Wellington Management and all existing investors

Proceeds to support advancement of ATSN-201 through potential approval and launch as well as preclinical programs to treat inherited retinal diseases

Norbert Riedel, PhD, will join Atsena’s Board of Directors

DURHAM, NC, April 2, 2025 – Atsena Therapeutics (“Atsena” or “the Company”), a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the successful close of an oversubscribed $150 million Series C financing. The financing was led by Bain Capital’s Life Sciences team, with participation from an additional new investor, Wellington Management. All the Company’s existing investors also participated in the round, including Lightstone Ventures, Sofinnova Investments, Abingworth, Foundation Fighting Blindness, Hatteras Venture Partners, Osage University Partners, and the Manning Family Foundation.

Proceeds from the financing will be used to advance Atsena’s lead program, ATSN-201, for the treatment of X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. The proceeds will also support Atsena’s preclinical pipeline of first-in-class therapies and expand the use of Atsena’s novel spreading AAV.SPR capsid.

“Closing our Series C marks a pivotal moment for Atsena as we advance our transformative ocular gene therapies and fuel our next phase of growth, innovation, and clinical progress,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “It follows a productive 12 months of key achievements including securing a partner to advance ATSN-101 to a global pivotal trial for Leber Congenital Amaurosis type 1 (LCA1) and initiating Part B of the ATSN-201 LIGHTHOUSE study for XLRS. We’re grateful for the support of our investors and partners who share our vision for the future of leveraging genetic medicine to reverse or prevent blindness.”

To date, Atsena’s clinical portfolio has received multiple designations by the U.S. Food and Drug Administration (FDA). ATSN-101, for the treatment of LCA1, has received Rare Pediatric Disease designation, Orphan Drug Designation, and Regenerative Medicine Advanced Therapy designation. ATSN-201 has been granted Fast Track, Rare Pediatric Disease, and Orphan Drug Designations. Updated data from the ongoing LIGHTHOUSE Phase I/II clinical trial evaluating ATSN-201 is anticipated later this year.

“We believe Atsena has a unique opportunity to deliver meaningful impact for patients with inherited retinal diseases on the basis of novel science and impressive clinical data generated to date,” said Amir Zamani, a Partner at Bain Capital. “We look forward to supporting Patrick and his strong team as they look to unlock the next phase of Atsena’s growth and innovation while thoughtfully advancing potentially groundbreaking therapies toward patients in need.”

In conjunction with the financing, Norbert Riedel, PhD, a seasoned scientist and biopharmaceutical executive, will join Atsena’s Board of Directors.

Wedbush & Co. served as exclusive placement agent to Atsena for the Series C financing, Cooley LLP acted as its legal advisor.

About Atsena Therapeutics
Atsena Therapeutics (“Atsena”) is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena’s first-in-class, investigational gene therapy for Leber congenital amaurosis type 1 (LCA1) has completed a Phase 1 / 2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8). Atsena is advancing ATSN-101 toward the initiation of a global pivotal trial as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/.

Media Contact:
Gina Mangiaracina
6 Degrees
(917) 797-7904
[email protected]

Business Contact:
[email protected]

Atsena Therapeutics Granted U.S. FDA Fast Track Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis

Marks third FDA designation for ATSN-201, which has also received Rare Pediatric Disease Designation and Orphan Drug Designation

DURHAM, NC, March 12, 2025 – Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.

“We are pleased that the FDA has granted Fast Track designation to ATSN-201, reinforcing its potential to address the significant unmet need in XLRS, a rare inherited retinal disease with no approved treatments,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “This designation, along with the previously granted Orphan Drug and Rare Pediatric Disease designations, marks an important milestone in advancing the development of ATSN-201. The Atsena team remains dedicated to developing transformative gene therapies and improving the quality of life of individuals suffering from XLRS and other inherited retinal diseases.”

Fast Track designation is granted to treatments intended to address serious or life-threatening diseases that have demonstrated the potential to meet an unmet medical need. Treatments that receive Fast Track designation may benefit from more frequent interactions with the FDA throughout drug development. In addition, the Fast Track program allows for Priority Review, if relevant criteria are met.

Currently, there are no approved treatments for XLRS, which is typically diagnosed in early childhood and affects approximately 30,000 males in the U.S. and the EU. The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, a Phase I/II, dose-escalation and dose-expansion clinical trial in male patients ages six and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860).

About X-linked Retinoschisis (XLRS)
XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments.

About AAV.SPR
AAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and has a favorable safety profile relative to benchmark capsids. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/.

About Atsena Therapeutics
Atsena Therapeutics (“Atsena”) is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena’s first-in-class, investigational gene therapy for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children, has completed a Phase 1 / 2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8). Atsena is advancing ATSN-101 toward the initiation of a global pivotal trial as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/.

Media Contact:
Gina Mangiaracina
6 Degrees
(917) 797-7904
[email protected]

Business Contact:
[email protected]

Atsena Therapeutics to Present ATSN-201 Safety and Efficacy Data at The Macula Society’s 48th Annual Meeting

 

DURHAM, NC, February 6, 2025 – Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that safety and efficacy data on ATSN-201 for the treatment of X-linked retinoschisis (XLRS) will be presented during a panel discussion at The Macula Society’s 48^th Annual Meeting taking place February 12-15, 2025, in Charlotte Harbor, Florida. ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.

Presentation details are as follows:

Session: Inherited Retinal Dystrophy I: Trials

Date and Time: Thursday, February 13, 2025, 7:00-7:45 a.m. ET

Presentation Title: Interim Safety and Efficacy of ATSN-201 Dose Escalation Study in Patients With X-linked Retinoschisis (XLRS)

Presentation Time: 7:25 a.m. ET

Presenter: Christine Nichols Kay, MD, Clinical Ophthalmology Advisor, Atsena Therapeutics

The LIGHTHOUSE study is a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial evaluating the safety and tolerability of ATSN-201 in male patients ages six and older with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860). ATSN-201 has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration.

 About X-linked Retinoschisis (XLRS)

XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments.

 About AAV.SPR

AAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in stark contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and does not cause inflammation. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/.

About Atsena Therapeutics
Atsena Therapeutics (“Atsena”) is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena’s first-in-class, investigational gene therapy for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children, has completed a Phase 1 / 2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8). Atsena is advancing ATSN-101 toward the initiation of a global pivotal trial as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/.

 

Media Contact:
Gina Mangiaracina

6 Degrees
(917) 797-7904

[email protected]

Business Contact:
[email protected]

Atsena Therapeutics Initiates Part B of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis

Part B of the LIGHTHOUSE study will enroll adults and children

 DURHAM, NC, January 8, 2025 – Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the initiation of Part B of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.

“We are pleased to initiate Part B of the LIGHTHOUSE study of ATSN-201 following successful enrollment and dosing of Part A. The functional and structural improvements seen in Part A validate our novel AAV.SPR spreading capsid,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “Importantly, Part B will enroll both adult and pediatric participants. The Rare Pediatric Disease designation granted for ATSN-201 highlights the unmet need for a treatment, and we remain focused on advancing a therapeutic option for these patients.”

Part A of the study evaluated the safety and tolerability of three different doses of ATSN-201 administered through subretinal injection. After reviewing early safety data from Part A, the Data Monitoring Committee has recommended proceeding to Part B using 1.0E11 vg/mL, a concentration that offered the optimal balance of tolerability and efficacy based on preliminary clinical results. Part B is a multicenter clinical trial that will evaluate nine additional adults and three pediatric patients with XLRS. The adult cohort will be broken down into three arms, low volume, high volume and control. Patients in the control arm will be observed off-therapy for one year and then have the option to receive treatment. The pediatric cohort will be dosed after evaluating preliminary data from the adult cohort in Part B. This part of the study will continue to assess safety and efficacy, including microperimetry, visual acuity and macular structure.

“A major benefit of ATSN-201 is that it does not need to be precisely placed underneath a specific retinal region. This gives surgeons more discretion regarding bleb placement during subretinal surgery and allows for safe delivery of the healthy gene to the critical portion of the retina,” said Kenji Fujita, MD, Atsena’s Chief Medical Officer. “By testing different volumes in Part B, we will better understand the optimal administration of ATSN-201 to achieve the broadest effect.”

Currently, there are no approved treatments for XLRS which is typically diagnosed in early childhood and primarily affects males. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and ultimately leads to blindness. Approximately 30,000 males in the U.S. and EU have this inherited retinal disease.

The LIGHTHOUSE study is a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial evaluating the safety and tolerability of ATSN-201 in male patients ages six and older with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860). ATSN-201 has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration.

About X-linked Retinoschisis (XLRS)

XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments.

 About AAV.SPR

AAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in stark contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and does not cause inflammation. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/.

About Atsena Therapeutics
Atsena Therapeutics (“Atsena”) is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena’s first-in-class, investigational gene therapy for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children, has completed a Phase 1 / 2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8). Atsena is advancing ATSN-101 toward the initiation of a global pivotal trial as part of an exclusive, strategic collaboration. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/.

 

Media Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
[email protected]

Business Contact:
[email protected]

Atsena Therapeutics Announces Dosing Completed in Part A of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis

 

DURHAM, NC, December 16, 2024 – Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced dosing has been completed in Part A of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.

“Nine adults have been treated in Part A of the study in which three dose levels of ATSN-201 are being evaluated for safety and efficacy. We have seen both structural and functional benefits in patients treated at all dose levels. Additionally, no serious adverse events related to treatment have been reported,” said Kenji Fujita, MD, Atsena’s Chief Medical Officer. “Collecting data from Part A of the trial is an important breakthrough for Atsena because it validates the use of our novel capsid to effectively treat inherited retinal disease and it informs the safest and most effective path forward for Part B. We look forward to providing further updates from those patients early next year.”

Currently, there are no approved treatments for XLRS which is typically diagnosed in early childhood and primarily affects males. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and ultimately leads to blindness. Approximately 30,000 males in the U.S. and EU have this inherited retinal disease.

“We are thrilled that dosing is complete in Part A of the LIGHTHOUSE study for ATSN-201 and that we’ve begun hearing positive anecdotes from those involved. This marks a significant milestone for patients with XLRS as we push to bring a therapeutic option to individuals that otherwise have no approved treatment,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “It also represents potential for individuals living with other inherited retinal diseases that could benefit from our novel capsid. We look forward to the insights gained from this trial as we pioneer new approaches to ocular gene therapy.”

The LIGHTHOUSE study is a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial evaluating the safety and tolerability of ATSN-201 in male patients ages six and older with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860). ATSN-201 has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration.

About X-linked Retinoschisis (XLRS)

XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and, ultimately, blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments.

 About AAV.SPR

AAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in stark contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and does not cause inflammation. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/.

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena’s program for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children, has completed a Phase 1 / 2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8) and is moving towards initiation of a pivotal trial. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit  https://atsenatx.com/.

 

Media Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
[email protected]

Business Contact:
[email protected]

Nippon Shinyaku and Atsena Therapeutics Enter into an Exclusive Strategic Collaboration for ATSN-101 in the U.S. and Japan

 

KYOTO, Japan and Durham, North Carolina, USA, November 13, 2024 – Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto; President, Toru Nakai) and Atsena Therapeutics, Inc. (Atsena; Headquarters: Durham, North Carolina, USA, Chief Executive Officer (CEO): Patrick Ritschel) have entered into an exclusive license agreement for the commercialization of ATSN-101 in the territory of the U.S. and for the development and commercialization of ATSN-101 in the territory of Japan for advancing Atsena’s first- in-class, investigational gene therapy ATSN-101 for Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1).

Under the terms of the licensing agreement, Nippon Shinyaku will receive exclusive commercial rights in the U.S. and Japan, and Atsena will retain commercial rights in the rest of the world. ATSN-101 will be marketed by NS Pharma, Inc. (New Jersey, USA, President: Yukiteru Sugiyama), a wholly owned subsidiary of Nippon Shinyaku in the U.S.

Atsena will receive an upfront payment, additional milestone payments, downstream royalties based on sales and will be reimbursed as it continues development work on ATSN-101, including an anticipated global pivotal trial.

ATSN-101 is a first-in-class, investigational gene therapy for the treatment of LCA1. Atsena has received Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for ATSN-101. In the event Atsena receives a Rare Pediatric Disease Priority Review Voucher (PRV) in connection with the approval of the Biologic License Application for ATSN-101, Atsena shall own and retain all rights, title and interest in such PRV.

“ATSN-101 provides a potential, innovative treatment in an area where no approved solutions currently exist,” said Nippon Shinyaku President Toru Nakai. “We are excited by the opportunity of this novel ocular gene therapy and our collaboration with Atsena and its groundbreaking science.”

“This collaboration creates a path to accelerate the development of ATSN-101 and validates Atsena’s pioneering technology and development capabilities. We anticipate this will be the first of many ocular gene therapy treatments from our clinical portfolio to come,” said Patrick Ritschel, CEO of Atsena Therapeutics. “We look forward to working with Nippon Shinyaku as we advance ATSN-101 into a pivotal trial and potential approval to provide an innovative solution to patients and families affected by LCA1 around the world.”

About GUCY2D-associated Leber congenital amaurosis (LCA1)
LCA1 is a monogenic eye disease that disrupts the function of the retina. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. LCA1 is one of the most common forms of LCA and there are no approved treatments for it.

About ATSN-101
ATSN-101 is an investigational, subretinal AAV5 gene therapy being evaluated in an ongoing Phase I/II clinical trial for LCA1. At 12 months post-treatment, ATSN-101 has demonstrated durable, clinically meaningful improvements in vision at the high dose and is well-tolerated. Results from this trial were recently published in The Lancet.

About Rare Pediatric Disease Designation
The Rare Pediatric Disease Designation is granted by the FDA for promoting the development of new drugs for rare diseases that occur in the U.S. before the age of 18 and affect fewer than 200,000 people. With this designation, the product will be eligible to receive a Priority Review Voucher upon approval that could be used to advance another program.

About Regenerative Medicine Advanced Therapy Designation
The Regenerative Medicine Advanced Therapy (RMAT) Designation is an FDA-designated system for advanced regenerative medicine therapies targeted at developing serious diseases that have shown certain effect in clinical trials based on the 21st Century Cures Act in the U.S. Companies with RMAT designations are given the opportunity for priority review and accelerated approval for the product.

About Orphan Drug Designation
The Orphan Drug Designation is provided for orphan drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. With orphan designation, the FDA grants a seven-year market exclusivity for the product and provides certain incentives such as tax credits towards the cost of development, upon approval.

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. In an ongoing Phase I/II clinical trial, the company is evaluating ATSN-101 for LCA1, one of the most common causes of blindness in children. In another ongoing clinical trial, the company is evaluating ATSN-201 for X-linked retinoschisis (XLRS), a genetic condition affecting boys and men that is typically diagnosed in childhood. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/.

About Nippon Shinyaku
Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness.

Please visit our website (https://www.nippon-shinyaku.co.jp/english/) for products or detailed information.

Atsena contacts:
Media: [email protected]
Business: [email protected]

Nippon Shinyaku contact:
[email protected]

Atsena Therapeutics to Present ATSN-201 Safety and Efficacy Data at the American Academy of Ophthalmology 2024 Annual Meeting

 

DURHAM, NC, October 10, 2024 – Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that safety and efficacy data on ATSN-201 for the treatment of X-linked retinoschisis (XLRS) will be presented during a panel discussion at the American Academy of Ophthalmology (AAO) 2024 Annual Meeting taking place October 18-21, 2024, in Chicago. ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.

Presentation details are as follows:

 

Session: First-time Results of Clinical Trials

Date and Time: Friday, October 18, 2024, 4:54 – 5:33 p.m. CDT

Presentation Title: Interim Safety and Efficacy of ATSN-201 Dose Escalation Study in Patients With X-linked Retinoschisis (XLRS)

Presentation Time: 5:08 p.m. CDT

Presenter: Christine Nichols Kay, MD, Clinical Ophthalmology Advisor, Atsena Therapeutics

 

The safety and tolerability of ATSN-201 is being evaluated in the LIGHTHOUSE study, a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial in male patients ages six and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860). ATSN-201 has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration.

 

About X-linked Retinoschisis (XLRS)

XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments.

 

About AAV.SPR

AAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection bleb margins to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond bleb margins. This is in stark contrast to benchmark AAV vectors, which remain confined to those original margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and does not cause inflammation. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/.

 

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition affecting boys and men that is typically diagnosed in childhood and leads to blindness later in life. Another ongoing Phase I/II clinical trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit  https://atsenatx.com/.

 

Media Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
[email protected]

Business Contact:
[email protected]

Atsena Therapeutics to Present at Chardan’s 8th Annual Genetic Medicines Conference

 

DURHAM, NC, September 24, 2024 – Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that it will present at Chardan’s 8^th Annual Genetic Medicines Conference taking place September 30 – October 1, 2024, in New York City. Chief Executive Officer Patrick Ritschel will deliver a company presentation on Tuesday, October 1, 2024, at 11:30 a.m. ET. Atsena Chief Medical Officer, Kenji Fujita, MD, and Mr. Ritschel will also participate in one-on-one meetings with potential investors during the conference.

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a progressive genetic condition affecting boys and men that is typically diagnosed in childhood. Another ongoing Phase I/II clinical trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit  https://atsenatx.com/.

 

Media Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
[email protected]

Business Contact:
[email protected]