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Atsena Therapeutics Announces Positive 12-month Safety and Efficacy Data from Ongoing Phase I/II Clinical Trial of ATSN-101 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1)

• ATSN-101 continues to demonstrate clinically meaningful improvements in vision at the highest dose and is well-tolerated 12 months post-treatment
• Data accepted for presentation at 47th Annual Macula Society Meeting

DURHAM, NC, December 4, 2023 – 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 positive 12-month safety and efficacy data from the ongoing Phase I/II trial of ATSN-101, the company’s investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1). At 12 months post-treatment, ATSN-101 has conferred clinically meaningful improvements in vision at the highest dose with no serious treatment-emergent adverse events.

“We continue to be encouraged by the data emerging from our Phase I/II trial of ATSN-101 in patients with GUCY2D-associated LCA1,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “The durability of clinically meaningful visual improvements in the absence of serious treatment-related adverse events at the 12-month mark underscore the safety, tolerability, and efficacy of our subretinal gene therapy. We believe the 12-month findings provide solid proof of concept that ATSN-101 will exceed the requirements set by the U.S. Food and Drug Administration for ultimate approval. We are exploring partnering and out-licensing options to advance ATSN-101 into a pivotal trial.”

In the Phase I/II trial, 15 patients ages 12 to 76 received unilateral subretinal injections of ATSN-101. Three adult cohorts (N=3 each) were treated with three ascending doses. Subsequently, one adult and one pediatric cohort (N=3 each) were treated at the high dose. In total, 9 patients received the high dose.

At 12 months, there were no serious treatment-emergent adverse events. Ocular inflammation was mild and reversible with steroid treatment. For high-dose patients, the mean (SE) change from baseline in dark-adapted full-field stimulus testing (FST) (white stimulus) was greater in treated eyes compared with untreated eyes at all six follow-up visits, and some patients exhibited over 10,000-fold improvements in retinal sensitivity. Of the six high-dose patients who were tested with multi-luminance mobility testing (MLMT), three improved by ≥2 levels compared to baseline (when available) or to the untreated eye, and all three demonstrated a maximum score of 6 in the treated eye. At 12 months, patients receiving the high-dose demonstrated a statistically significant improvement in best-corrected visual acuity (BCVA).

“The noteworthy improvements in key visual parameters demonstrate the potential of ATSN-101 to make a meaningful impact on the lives of patients affected by GUCY2D-associated LCA1,” said Paul Yang, MD, PhD, Associate Professor of Ophthalmology at OHSU School of Medicine. “As LCA1 causes early and severe vision impairment or blindness and there are no approved treatments, ATSN-101 has the potential to fill a significant unmet need within the LCA community.”

The 12-month data have been accepted for presentation at the 47th Annual Macula Society Meeting, which will be held February 7-10, 2024, in Palm Springs, CA.

The U.S. Food and Drug Administration (FDA) recently granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101. Atsena has also received orphan drug designation from the FDA for ATSN-101 for the treatment of LCA1.

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. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1.

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 12 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

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

Business Contact:
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RMAT designation recognizes the potential of ATSN-101 to address unmet medical needs for patients with LCA1

ATSN-101 has demonstrated clinically meaningful improvements in vision at the highest dose with no drug-related serious adverse events 6 months post-treatment in ongoing Phase I/II clinical trial

DURHAM, NC, November 14, 2023 – 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 U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101, the company’s lead investigational gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). RMAT designation was granted based on positive 6-month efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101.

“Receiving RMAT designation from the FDA for ATSN-101 marks a significant regulatory milestone for Atsena, validating the potential of our subretinal gene therapy to improve vision and make a meaningful difference in the lives of patients with LCA1,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics. “As we continue to explore options to advance ATSN-101 into a pivotal clinical trial, we look forward to reporting 12-month data from our ongoing Phase I/II trial by the end of this year.”

“There are no approved treatments for LCA1, an inherited retinal disease that results in early and profound vision impairment or blindness,” said Jason Menzo, Chief Executive Officer of Foundation Fighting Blindness. “RMAT designation is encouraging recognition of the potential of ATSN-101 to be an important treatment and provides hope to children and adults affected by LCA1.”

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.

Atsena has also received orphan drug designation from the FDA for ATSN-101 for the treatment of LCA1.

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. GUCY2D-LCA is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1.

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 6 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

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

Business Contact:
[email protected]

The LIGHTHOUSE study evaluating the safety and tolerability of investigational gene therapy ATSN-201 is enrolling male patients ages 6-64 with RS1-associated XLRS

ATSN-201 leverages novel spreading capsid to overcome challenges associated with intravitreally delivered AAVs in the treatment of XLRS

DURHAM, NC, August 28, 2023 – 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 first patient has been dosed in its Phase I/II clinical trial, the LIGHTHOUSE study, evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201 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.

“Dosing the first patient in the LIGHTHOUSE study marks a significant milestone for Atsena and the XLRS community,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “We are excited to be utilizing AAV.SPR in the clinic, as it has the potential to revolutionize the treatment of XLRS, as well as other inherited retinal disorders. Spreading laterally beyond the subretinal injection site, AAV.SPR facilitates the safe delivery of RS1 to photoreceptors in the central retina/fovea. We look forward to advancing the LIGHTHOUSE study and the continued development of our novel gene therapies to reverse or prevent blindness.”

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 6-64 with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860).

“Considering the lack of available therapies for X-linked retinoschisis, this is very exciting news for the inherited retinal disease community,” said Mark Pennesi, MD, PhD, Professor in Ophthalmology and Chief of the Paul H. Casey Ophthalmic Genetics Division Molecular and Medical Genetics, School of Medicine at Oregon Health & Science University (OHSU). “While attempts to deliver gene therapy through intravitreal routes faced challenges, subretinal treatment utilizing spreading AAV vectors has the potential to be the breakthrough we need to achieve efficacy.”

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 the layers of the retina, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss. 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 spreading 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 vector AAV5, which remains 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://atsena.wpenginepowered.com/our-approach/laterally-spreading-aav/.

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 6 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

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

Business Contact:
[email protected]

ATSN-201 leverages AAV capsid that spreads laterally beyond subretinal injection site to facilitate safe delivery of RS1 to photoreceptors in the central retina/fovea

Initiation of Phase I/II clinical trial, known as The Lighthouse Study, anticipated in mid-2023

DURHAM, NC, May 1, 2023 – 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 U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for a Phase I/II clinical trial of ATSN-201 in patients with X-linked retinoschisis (XLRS). ATSN-201 leverages one of the company’s novel spreading capsids, AAV.SPR, to overcome the challenges associated with intravitreally delivered AAVs in the treatment of XLRS. 

“Intravitreally delivered AAVs have limitations, as they do not drive sufficient gene expression in photoreceptors to confer therapy and can lead to vision-compromising inflammation,” said Shannon Boye, PhD, Founder and Director of Atsena Therapeutics. “AAV.SPR is well-suited for use in XLRS as it can drive therapeutic levels of gene expression in photoreceptors while avoiding the surgical risks of foveal detachment, which is important because XLRS patients have fragile retinas due to the presence of schisis lesions. Building on decades of research, we’re excited to progress our novel gene therapy for patients with XLRS who currently lack an approved treatment option.”

“With the FDA’s clearance of the IND application for ATSN-201, we’re preparing to advance our first program utilizing AAV.SPR into the clinic for the treatment of XLRS in mid-2023,” said Kenji Fujita, Chief Medical Officer of Atsena Therapeutics. “We look forward to evaluating ATSN-201 and addressing the unmet need for a treatment to improve or restore vision in patients with XLRS.”

The Lighthouse Study, a Phase I/II, open-label, dose-escalation clinical trial, will evaluate subretinal injection of ATSN-201 in male patients ages 6-65 with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1.

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 the layers of the retina, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss. 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 spreading 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 vector AAV5, which remains 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://atsena.wpenginepowered.com/our-approach/laterally-spreading-aav/.

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1), one of the most common causes of blindness in children. Interim safety and efficacy data has demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 6 months post-treatment. The company’s additional pipeline of proprietary assets includes ATSN-201, an investigational gene therapy that leverages one of the company’s novel spreading capsids, AAV.SPR, for the treatment of X-linked retinoschisis (XLRS), and ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

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

Business Contact:
[email protected]

• ATSN-101 demonstrated clinically meaningful improvements in vision at the highest dose with no drug-related serious adverse events 6 months post treatment
• Data presented at ARVO 2023

DURHAM, NC, April 25, 2023 – 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 positive 6-month safety and efficacy data from the ongoing Phase I/II clinical trial of ATSN-101, the company’s investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1). The data were presented by Christine Nichols Kay, MD, Clinical Ophthalmology Advisor for Atsena, at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting. 

“We’re encouraged by the clinically meaningful improvements in vision ATSN-101 has demonstrated at the highest dose 6 months post treatment, as well as the favorable safety profile of our subretinal gene therapy,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “The latest data reinforce our confidence in the potential of ATSN-101 to improve vision in patients with GUCY2D-associated LCA1, which results in early and severe vision impairment or blindness and lacks an approved treatment. We look forward to reporting 12-month data later this year and are exploring options to advance ATSN-101 into a pivotal trial.”

In the Phase I/II trial, 15 patients, including three pediatric patients, received ATSN-101 via subretinal delivery. Three adult cohorts (N=3 each) were treated with three ascending doses. Six additional patients received the high dose in a dose expansion phase. Among patients who received the high-dose treatment (N=9), the mean (SE) change from baseline in retinal sensitivity by dark-adapted full-field stimulus testing (FST) was significantly greater in treated eyes compared with untreated eyes at Day 28 and all subsequent follow-up visits. Two high-dose patients demonstrated best corrected visual acuity (BCVA) improvement greater than 0.3 logMAR, and no treated eyes had a decrease in BCVA. Of the five high-dose patients who were tested with the MLMT mobility test, four patients demonstrated either a maximum MLMT score of 6 or a ≥2 level improvement, compared to baseline when available or to the untreated fellow eye, at one or more post-treatment visits.

To date, no drug-related serious adverse events have been reported and ocular inflammation has been infrequent, minimal, and reversible with steroid treatment.

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. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1.

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1), one of the most common causes of blindness in children. Interim safety and efficacy data has demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 6 months post-treatment. The company’s additional pipeline of proprietary assets includes ATSN-201, an investigational gene therapy that leverages one of the company’s novel spreading capsids, AAV.SPR, for the treatment of X-linked retinoschisis (XLRS), and ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

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

Business Contact:
[email protected]

DURHAM, NC, April 11, 2023 – 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 6-month safety and efficacy data from the ongoing Phase I/II clinical trial of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1) will be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting, which is being held April 23-27 in New Orleans, LA.

LCA1 is a monogenic eye disease that disrupts the function of the retina and results in early and severe vision impairment or blindness. According to previously reported interim data from the Phase I/II clinical trial, ATSN-101 has demonstrated clinically meaningful improvements in vision with no drug-related serious adverse events.

Details of the oral presentation are as follows:

Title: Six-month safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1)
Presentation Number: 1914
Session: Advances in gene therapy and gene editing for ocular diseases 1
Date / Time: Monday, April 24, 3:45 – 4:00 p.m. CT
Location: Ernest N. Morial Convention Center, R07-R08
Presenter: Christine Nichols Kay, MD, Atsena Therapeutics

In addition, Atsena is pleased to sponsor and participate in the Retinal Cell and Gene Therapy Innovation Summit 2023, jointly organized by the Foundation Fighting Blindness and the Oregon Health & Science University (OHSU) Casey Eye Institute, which is being held on Friday, April 21 in New Orleans, LA.

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating ATSN-101 for a form of LCA, one of the most common causes of blindness in children. Positive interim data presented at the American Academy of Ophthalmology 2022 Annual Meeting demonstrated that subretinal delivery of ATSN-101 was well tolerated and patients treated with the highest dose saw clinically meaningful improvements in vision. The company’s additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

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

Business Contact:
[email protected]

ATSN-101 has demonstrated clinically meaningful improvements in vision with no drug-related serious adverse events

DURHAM, NC, February 8, 2023 – 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 positive interim data from the ongoing Phase I/II clinical trial of ATSN-101 will be presented at the 46th Annual Macula Society Meeting, which is being held February 15-18 in Miami, FL. This is an encore presentation of data that were presented as a late-breaker at the American Academy of Ophthalmology 2022 Annual Meeting.

ATSN-101, Atsena’s lead investigational gene therapy product, is being evaluated in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). LCA1 is a monogenic eye disease that disrupts the function of the retina and results in early and severe vision impairment or blindness.

“We are pleased to have the opportunity to share the positive interim safety and efficacy data from our ongoing Phase I/II trial of ATSN-101 with the retinal disease community at the upcoming Macula Society Annual Meeting,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “Subretinal delivery of ATSN-101 was well tolerated and demonstrated clinically meaningful improvements in vision in patients treated with the highest dose. With no approved treatments available for patients with LCA1, we are honored to be at the forefront of research for this inherited retinal disease and look forward to reporting additional data from our Phase I/II trial at medical meetings later this year.”

Details of the presentation are as follows:

Title: Safety and Efficacy of ATSN-101 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1)
Section: Session III – Inherited Retinal Disorders
Date / Time: Wednesday, February 15, 6:16 p.m. EST
Location: Fontainebleau Miami Beach
Presenter: Christine Nichols Kay, MD, Atsena Therapeutics

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. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1.

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating ATSN-101 for a form of LCA, one of the most common causes of blindness in children. Positive interim data presented at the American Academy of Ophthalmology 2022 Annual Meeting demonstrated that subretinal delivery of ATSN-101 was well tolerated and patients treated with the highest dose saw clinically meaningful improvements in vision. The company’s additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

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

Business Contact:
[email protected]

DURHAM, NC, December 7, 2022 – 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 Patrick Ritschel, Chief Executive Officer, will present a company overview at the Emerging Life Science Company Showcase hosted by NewYorkBIO and NYSE. The presentation will take place at The New York Stock Exchange on Wednesday, December 14, 2022, at 4:15 p.m. EST.

For more information and to register as an in-person or virtual attendee, please click here.

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating ATSN-101 for LCA1, one of the most common causes of blindness in children. Positive interim data presented at the American Academy of Ophthalmology 2022 Annual Meeting demonstrated that subretinal delivery of ATSN-101 was well tolerated and patients treated with the highest dose saw clinically meaningful improvements in vision. The company’s additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

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

Business Contact:
[email protected]

Biotech leader brings extensive experience in finance, business development and corporate strategy

DURHAM, NC, November 7, 2022 – 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 appointment of Elisabeth (Lis) Leiderman, MD, MBA, as Chief Financial Officer and Chief Business Officer. Dr. Leiderman has more than 15 years of finance, business development and strategy experience in the life sciences industry, most recently with gene therapy companies. She currently serves as a member of the board of directors and Audit Committee Chair of bluebird bio.

“Lis’ unique blend of finance, business and medical expertise will be essential as we advance our pipeline of ocular gene therapies and prepare to launch a pivotal trial in GUCY2D-associated Leber congenital amaurosis (LCA1) following the positive results from our Phase I/II trial of ATSN-101,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics. “We’re delighted to welcome Lis to our leadership team.”

Dr. Leiderman has extensive experience with financings, initial public offerings, mergers and acquisitions and licensing transactions. Prior to joining Atsena, Dr. Leiderman was Chief Financial Officer and Head of Corporate Development at Decibel Therapeutics, a clinical-stage biotechnology company developing gene therapeutics for restoration of hearing loss and balance disorders. At Decibel, she led the company’s Series D financing close and initial public offering. Previously, Dr. Leiderman was Chief Business Officer and Corporate Secretary at Complexa, Inc., and Senior Vice President, Head of Corporate Development at Fortress Biotech.

“I’m excited to be part of a talented team advancing novel technologies and life-changing gene therapies tailored to prevent or reverse blindness,” said Dr. Leiderman. “I look forward to contributing to Atsena’s growth and continued progress toward bringing important new therapies to people with inherited retinal diseases.”

Earlier in her career, Dr. Leiderman spent 10 years as a healthcare investment banker advising global corporate clients and their boards. She earned an MD from the American Medical Program at Tel Aviv University, an MBA from The Wharton School at the University of Pennsylvania and a BA from the University of Pennsylvania.

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating a potential therapy for LCA1, one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

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

Business Contact:
[email protected]

• ATSN-101 demonstrated clinically meaningful improvements in vision with no drug-related serious adverse events
• Data presented at the American Academy of Ophthalmology 2022 Annual Meeting

DURHAM, NC – Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, announced positive results from the Phase I/II clinical trial of ATSN-101, its lead investigational gene therapy product formerly known as SAR439483, for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1).

The data demonstrated that subretinal delivery of ATSN-101 was well tolerated and patients treated with the highest dose (1.0E11 vg/eye) saw clinically meaningful improvements in vision, as measured by full-field stimulus testing (FST) and multi-luminance mobility testing (MLMT), at more than one-month post treatment.

As of the July 25, 2022, data cut-off date, 15 patients, including three pediatric patients, were treated with ascending doses of ATSN-101. Patients treated with the highest dose (N=9) demonstrated a significantly larger mean change from baseline in retinal sensitivity and a trend toward a larger mean change in best-corrected visual acuity (BCVA) in treated eyes as compared with untreated eyes. In addition, three of four patients tested on MLMT demonstrated at least two-level improvement from baseline light levels. No drug-related serious adverse events were reported, and most treatment-emergent adverse events were mild and transient.

“Patients with LCA1 have profound visual impairment or blindness at birth, but their retinal structure remains intact, which indicates an opportunity to confer meaningful improvements following delivery of a subretinal gene therapy such as ATSN-101,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “We’re encouraged by these data that demonstrate ATSN-101 improved visual function while maintaining a favorable safety profile. We look forward to launching a pivotal trial for the evaluation of ATSN-101, which will lay the groundwork for successful registration and commercialization. We also look forward to advancing other promising programs in our gene therapy pipeline to reverse or prevent blindness for people with inherited retinal diseases.”

The data were presented on Saturday, Oct. 1, in a Late Breaking Developments session during the Retina Subspecialty Day at the American Academy of Ophthalmology Annual Meeting (AAO 2022) in Chicago, by Christine Nichols Kay, MD, Clinical Ophthalmology Advisor for Atsena.

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. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1.

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating a potential therapy for a form of LCA, one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

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

Business Contact:
[email protected]