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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]

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• 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:
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View the AAO presentation.

• Data from Phase I/II trial of ATSN-101 in patients with GUCY2D-associated Leber congenital amaurosis (LCA1) to be presented

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, today announced that data from the Phase I/II clinical trial of ATSN-101 will be presented in a Late Breaking Developments session during the Retina Subspecialty Day at the American Academy of Ophthalmology Annual Meeting (AAO 2022), which is being held September 30 – October 3, 2022 in Chicago.

ATSN-101, Atsena’s lead investigational gene therapy product formerly known as SAR439483, 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.

Details of the presentation are as follows:

Title: Safety and Efficacy of SAR439483 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1)

Abstract Number: 30071742

Section: Section X: Late Breaking Developments, Part II

Date / Time: Saturday, October 1, 9:15 a.m. CST

Location: McCormick Place – Arie Crown

Presenter: Christine Nichols Kay, MD, Atsena Therapeutics

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]

• World-class facility supports the company’s growth, and discovery and development of gene therapies for inherited retinal diseases

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, today announced the grand opening of its new office and laboratory in the Alexandria Center^® for Advanced Technologies – Research Triangle. The occasion was celebrated during a ribbon-cutting ceremony yesterday.

The new state-of-the-art facility consists of approximately 20,000 square feet of laboratory and office space to accommodate research and corporate functions of Atsena’s rapidly expanding team. The company is strategically located on a dynamic campus in the Research Triangle, providing access to top talent and gene therapy expertise in the thriving innovation hub.

“Our new integrated office and lab space is an exciting indication of our progress toward bringing life-changing treatments to patients with inherited forms of blindness,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics. “We’re happy to call North Carolina’s Research Triangle our home as it connects us to a flourishing life sciences ecosystem and enables us to tap into leading talent as we continue to grow. At Atsena, we’re proud to have a team of individuals with deep expertise in ophthalmology, AAV gene therapy and CMC who are dedicated to improving the quality of life of patients with vision loss.”

For information about current job openings at Atsena, please visit https://atsena.wpenginepowered.com/careers/ and follow the company on LinkedIn.

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 https://atsena.wpenginepowered.com/.

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

Business Contact:
[email protected]

• Presentations to feature laterally spreading capsid for the treatment of X-linked retinoschisis and dual vector technology for the treatment of MYO7A-associated Usher syndrome

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, today announced that research on two of its next-generation adeno-associated virus (AAV) technologies will be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting, which is being held May 1-4 in Denver, and the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting, which is being held May 16-19 in Washington, D.C.

“The AAV technologies that power our pipeline are tailored to overcome the limitations of current gene therapies and address significant unmet needs in inherited retinal diseases,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena. “We look forward to sharing research with the ophthalmology and gene therapy communities on AAV.SPR, one of our novel spreading capsids that is being leveraged in IND-enabling studies of ATSN-201 for X-linked retinoschisis (XLRS), as well as our dual vector technology that is being utilized in our ATSN-301 preclinical program for MYO7A-associated Usher syndrome.”

Details of the presentations are as follows:

ARVO 2022 Annual Meeting

Title: Laterally Spreading AAV.SPR-hRS1 Vector for Treatment of XLRS
Abstract Number: A0341
Poster Number: 2825
Session: Developing Molecular Therapies for Inherited Ocular Disease
Date / Time: Tuesday, May 3, 1:00-3:00 p.m. MDT
Location: Poster Hall
Presenter: Linda B. Couto, PhD, Chief Scientific Officer, Atsena Therapeutics

ASGCT 25th Annual Meeting

Title: Laterally Spreading AAV.SPR-hRS1 Vector for Treatment of XLRS
Abstract Number: 296
Poster Number: M-177
Session: Ophthalmic and Auditory Diseases
Date / Time: Monday, May 16, 5:30-6:30 p.m. EDT
Location: Hall D
Presenter: Sanford L. Boye, Founder and Chief Technology Officer, Atsena Therapeutics

Title: Characterization of Optimized Dual AAV-MYO7A Vectors for the Treatment of Usher Syndrome (USH1B) in Myo7a-/- Mice and NHP
Abstract Number: 1207
Session: New Technologies for AAV Gene Therapy (oral presentation)
Date / Time: Thursday, May 19, 11:30 a.m. EDT
Location: Ballroom C
Presenter: Kaitlyn Calabro, PhD, University of Florida

Details of a workshop in which Atsena is participating at the ASGCT 25th Annual Meeting are as follows:

Title: Designing Preclinical Programs to Assess Efficacy and Safety for Novel Viral Vectors
Session: Viral Vector Development Workshop
Date / Time: Sunday, May 15, 9:00-9:20 a.m. EDT
Location: Room 202
Presenter: Eva Andres-Mateos, Senior Director Translational Research, Atsena Therapeutics

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]

• Subretinal delivery of RS1 using AAV capsid that spreads laterally beyond the injection site overcomes challenges associated with intravitreal delivery

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, today unveiled its preclinical gene therapy program for X-linked retinoschisis (XLRS), a monogenic disease caused by mutations in the RS1 gene. XLRS is characterized by schisis, or abnormal splitting of the layers of the retina, which causes impaired visual acuity that is not correctable and leads to progressive vision loss. XLRS primarily affects males and is typically diagnosed in early childhood.

“We are pleased to disclose our program for XLRS, an inherited retinal disease lacking approved treatments to improve or restore vision,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena. “Following a recent pre-IND meeting with the U.S. Food and Drug Administration, we are working expeditiously to advance our differentiated gene therapy candidate for XLRS toward the clinic.”

Atsena’s XLRS gene therapy program leverages one of the company’s novel AAV capsids, AAV.SPR, that spreads laterally beyond the subretinal injection site, enabling 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. More information about the study and additional figures are available at: https://www.atsenatx.com/our-approach/laterally-spreading-aav/

AAV.SPR exhibits enhanced lateral spread in subretinally injected macaques.

“Our novel spreading capsids may be the key to overcoming the challenges associated with intravitreally delivered AAVs in the treatment of XLRS, such as inefficient photoreceptor transduction and inflammation,” said Linda B. Couto, PhD, Chief Scientific Officer of Atsena. “We are excited to continue advancing the research and development of our XLRS program, as we believe our approach facilitates the safe and efficient treatment of the central retina in XLRS patients.”

“We have spent years designing novel AAV capsids for efficient retinal transduction via multiple delivery routes. Our work, as well as that of others, highlights that intravitreally delivered AAVs do not drive sufficient gene expression in photoreceptors to confer therapy, and can even lead to vision-compromising inflammation,” said Shannon Boye, PhD, Founder and Director of Atsena. “AAV.SPR was born out of a desire to drive therapeutic levels of gene expression in photoreceptors while avoiding the surgical risks of foveal detachment, and the immunological complications of intravitreal delivery. The ability to safely and efficiently target foveal cones and to transduce wider expanses of retinal tissue represents a major leap forward in our field.”

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, with an additional office in northern New Jersey. For more information, please visit atsenatx.com.

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

Business Contact:
[email protected]

• Dr. Couto strengthens leadership team with extensive AAV and ocular gene therapy expertise

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, today announced the appointment of Linda B. Couto, PhD, as Chief Scientific Officer (CSO). Dr. Couto brings more than 25 years of gene therapy experience to the Atsena leadership team. She has been a senior gene therapy consultant to the company and will assume the CSO responsibilities from Shannon Boye, PhD, Atsena’s co-founder who has been serving as acting CSO. “I’m thrilled to welcome Linda to the Atsena team and look forward to working with her to develop novel ocular gene therapies,” said Dr. Boye, who will remain on Atsena’s board of directors and continue to provide scientific and strategic counsel.

“Linda’s depth of knowledge about adeno-associated virus (AAV) vectors and experience with the first approved gene therapy for inherited retinal disease are extraordinary assets to Atsena,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena. “We’re delighted that Linda is taking on the role of Chief Scientific Officer and look forward to continuing to leverage her gene therapy expertise as we progress the development of novel treatments aimed at reversing or preventing blindness.”

Prior to Atsena, Dr. Couto was Head of Pharmacology and Toxicology and Ocular Research Lead for Spark Therapeutics. She has been responsible for the pre-clinical development of numerous recombinant AAV vectors, encompassing vector design, execution of proof-of-concept studies in animal models of disease, and Investigational New Drug (IND)-enabling pharmacology, biodistribution, and toxicology studies, across multiple therapeutic areas. Dr. Couto has authored numerous non-clinical modules to support regulatory applications for gene therapies at various stages of development from pre-IND to Biologics License Applications and Marketing Authorization Applications, including LUXTURNA^®.

Dr. Couto was also Associate Director of the Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia. Earlier in her career, she held positions of increasing responsibility at several gene therapy biotechnology companies including Avigen Therapeutics and Somatix Therapy Corporation. Dr. Couto holds a PhD from the Massachusetts Institute of Technology.

“I’m excited to contribute to the advancement of Atsena’s innovative AAV technology platform and pipeline of ocular gene therapy programs,” said Dr. Couto. “I share the Atsena team’s passion for developing treatments for inherited retinal diseases and believe we have the potential to significantly improve patients’ lives with the power of genetic medicine.”

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 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]

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, today announced the appointment of Jennifer Wellman to its board of directors. Ms. Wellman, a biotech executive with more than 20 years of gene therapy development experience, will serve as an independent director.

“Jen’s extensive U.S. and EU regulatory expertise and impressive track record in the clinical development of AAV-based gene therapies are valuable to Atsena as we progress cutting-edge gene therapies designed to overcome the hurdles presented by inherited retinal disease,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena. “We’re thrilled to welcome Jen to Atsena’s board of directors and look forward to leveraging her strategic insights.”

Ms. Wellman is the Chief Operating Officer of Akouos, Inc., a precision genetic medicine company. Prior to Akouos, Ms. Wellman was Head of Product Development Strategy at Spark Therapeutics, Inc., now a subsidiary of Roche Holding AG, where she was also a co-founder. At Spark, she led the regulatory and clinical development for LUXTURNA® from the pre-IND phase through the filings of the marketing applications to the U.S. Food and Drug Administration and European Medicines Agency. Previously, Ms. Wellman was the Director of Regulatory Affairs for the Center for Cellular and Molecular Therapeutics at Children’s Hospital of Philadelphia; there, she directed multiple AAV preclinical programs and clinical trials for inherited retinal diseases, as well as other therapeutic areas. Earlier in her career, she served as an Associate Scientist at Avigen, Inc. Ms. Wellman received her M.S. from the University of New Haven and B.S. in microbiology and immunology from Queens University (Canada).

“Atsena’s clinical program for LCA1 and novel AAV capsid technology position the company to deliver potential life-changing treatments uniquely suited to prevent or reverse blindness and vision loss,” said Ms. Wellman. “I’m delighted to join Atsena’s board and support the team in the development of therapies for individuals living with inherited retinal diseases.”

Benjamin Yerxa, PhD, Chief Executive Officer of Foundation Fighting Blindness, has moved from Atsena’s board of directors to its scientific advisory board.

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 one of the most common causes of blindness and vision loss in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated viral (AAV) vector 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]

Phase I/II clinical trial is ongoing in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D

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, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for its investigational gene therapy product for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1), a genetic eye disease that affects the retina. The safety and efficacy of the gene therapy are being evaluated in a Phase I/II clinical trial, which is currently enrolling patients (ClinicalTrials.gov Identifier: NCT03920007).

“Receiving orphan drug designation from the FDA is an important milestone for our LCA1 gene therapy clinical program,” said Kenji Fujita, MD, Chief Medical Officer of Atsena. “We look forward to the continued progression of our Phase I/II clinical trial as we seek to develop a new treatment for children and adults who have severe visual impairment or blindness due to GUCY2D-associated LCA1.”

The FDA may grant orphan drug designation to drugs and biologics intended to treat diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, such as tax credits for qualified clinical testing, exemptions from certain FDA application fees, and seven years of market exclusivity, if approved.

Atsena’s LCA1 program is based on more than 15 years of research conducted at the University of Florida. The company exclusively licensed the rights to the gene therapy from Sanofi, which originally licensed it from University of Florida.

About LCA1

Leber congenital amaurosis (LCA) is the most common cause of blindness in children. LCA1 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 inherited retinal disease.

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 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]