Blog

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]