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• 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]

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• 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]

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• 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:
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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]

Dr. Fujita brings extensive clinical development and regulatory strategy experience

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 Kenji Fujita, MD, to the newly created position of Chief Medical Officer. An internist and cardiologist, Dr. Fujita brings more than 20 years of experience in academic medicine and the biopharmaceutical industry to the Atsena leadership team.

“We are delighted to welcome Kenji to Atsena as our Chief Medical Officer,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena. “His extensive experience leading clinical development programs and executing regulatory strategies will be invaluable as we continue to develop cutting-edge gene therapies with the goal of overcoming the unique hurdles of inherited retinal diseases to prevent or reverse blindness.”

Dr. Fujita’s experience spans preclinical to post-marketing research across a wide range of disease areas and therapeutic platforms. He has spearheaded five successful regulatory filings. Prior to joining Atsena, he was Vice President of Clinical Development at Alnylam Pharmaceuticals. There, he led the successful filing and registration of OXLUMO™, the first treatment for hyperoxaluria, in addition to leading clinical programs in hypertension and liver disease. Dr. Fujita has also held clinical development roles at Alexion Pharmaceuticals, where he led neurology and rare metabolic disease programs, and Merck, where he worked in cardiovascular clinical development and served as the Clinical Development Lead of the Emerging Markets Research and Development group.

Dr. Fujita currently serves as an Assistant Professor in Clinical Medicine at Robert Wood Johnson University Hospital. He holds an MD from Harvard Medical School and AB in Biochemical Sciences from Harvard College.

“I’m pleased to join Atsena as the company accelerates its Phase I/II LCA1 clinical trial and continues to advance two preclinical programs leveraging its AAV technology platform,” said Dr. Fujita. “Together with Atsena’s team deeply rooted in ocular gene therapy, I look forward to advancing life-changing treatments for patients with inherited retinal diseases.”

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:
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• Round was led by Sofinnova Investments with participation from Abingworth, Lightstone Ventures and all existing investors
• Company expands board of directors and plans to build out team

DURHAM, NC and BOSTON, MA – 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 it has closed an oversubscribed $55 million Series A financing led by Sofinnova Investments with participation from additional new investors Abingworth and Lightstone Ventures. Founding investors Hatteras Venture Partners and the Foundation Fighting Blindness’ RD Fund, along with existing investors Osage University Partners, University of Florida, and Manning Family Foundation, also participated in the round. Sarah Bhagat, PhD, Partner at Sofinnova, Jackie Grant, PhD, Principal at Abingworth, and Jason Lettmann, General Partner at Lightstone, will join Atsena’s board of directors.

Proceeds will be used to advance Atsena’s ongoing Phase I/II clinical trial evaluating a gene therapy for patients with GUCY2D-associated Leber congenital amaurosis (LCA1), one of the most common causes of blindness in children, as well as complete manufacturing development for Phase 3. In addition, the funds will enable Atsena to expand its team to support the research and development of novel gene therapies, including the progression of two existing preclinical programs in inherited retinal diseases toward the clinic and advancement of the company’s innovative adeno-associated virus (AAV) technology platform.

“We are grateful for the support of our new and existing investors and are encouraged by their enthusiasm for the potential of our technology to overcome the unique hurdles of inherited retinal diseases to prevent or reverse blindness,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena. “The Series A financing provides financial runway to reach the key inflection point of reading out efficacy data from our LCA1 clinical trial. While we continue expeditiously advancing this trial and our preclinical programs, we are excited to accelerate our growth as a leading ophthalmic gene therapy company.”

The Phase I/II LCA1 clinical trial is currently enrolling patients in the second dosing cohort. Atsena exclusively licensed the rights to the gene therapy from Sanofi, which originally licensed it from University of Florida. 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.

“We believe Atsena’s foundation in ocular gene therapy and potentially game-changing novel AAV vectors position the company to become a partner of choice,” said Dr. Bhagat. “Sofinnova is delighted to support Atsena and we look forward to helping the team further its mission to develop life-changing gene therapies for patients with inherited retinal diseases.”

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, Atsena has a licensing, research and manufacturing collaboration with the University of Florida and has offices in Boston, MA and North Carolina’s Research Triangle, environments rich in gene therapy expertise. For more information, please visit atsenatx.com.

About Sofinnova Investments

Since our founding in 1974, Sofinnova has been active in life science investing. We are a clinical-stage biopharmaceutical investment firm with approximately $2.3B in assets under management and committed capital. We invest in both private and public equity of therapeutics-focused companies. Our goal is to actively partner with entrepreneurs in both the U.S. and Europe, across all stages of company formation. From drug development and navigating the regulatory process to company building and IPO, we strive to be collaborative, meaningful board members, and excellent partners at every level. We seek to build world class companies that aspire to dramatically improve the current state of medical care today and ultimately, the lives of patients. Sofinnova has expertise investing in gene therapy companies, including investments in Spark, which developed the first approved gene therapy, Akouos, and Audentes, and Xylocor. For more information, please visit www.sofinnova.com.

About Abingworth

Abingworth is a leading transatlantic life sciences investment firm. Abingworth helps transform cutting-edge science into novel medicines by providing capital and expertise to top caliber management teams building world-class companies. Since 1973, Abingworth has invested in approximately 168 life science companies, leading to more than 44 M&A/exits and close to 70 IPOs. Our therapeutic focused investments fall into 3 categories: seed and early-stage, development stage, and clinical co-development. Abingworth supports its portfolio companies with a team of experienced professionals at offices in London, Menlo Park (California) and Boston. For more information, visit abingworth.com.

About Lightstone Ventures

Lightstone Ventures is a leading venture capital firm investing in therapeutic-oriented companies across the life science spectrum, from breakthrough medical devices to novel drugs and biopharmaceuticals. Founded in 2012, Lightstone has been part of many successful new ventures from inception through commercialization and plays a critical role guiding and building successful healthcare companies. With a proven strategy and global footprint, the Lightstone team has been involved in several of the largest venture-backed life science exits over the last decade including: ALX Oncology, Acceleron, Ardian, Calithera, Claret Medical, Disarm, MicroVention, Nimbus, Plexxikon, Portola, Promedior, Proteolix, Ra Pharma, Tizona, Twelve and Zeltiq. For more information, visit https://www.lightstonevc.com.

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

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