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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.

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Tony Plohoros
6 Degrees
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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:
[email protected]

• 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
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• Therapy currently being evaluated in Phase I/II clinical study
• Company formed with $8.15 million Series 1; led by founding investors Hatteras Venture Partners and the Foundation Fighting Blindness

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 it has acquired exclusive rights to a gene therapy targeting GUCY2D-associated Leber congenital amaurosis (LCA1), a genetic eye disease that affects the retina and is a leading cause of blindness in children, from Sanofi, which originally licensed it from the University of Florida. The therapy was created in the laboratory of Atsena Founder and Chief Scientific Officer Shannon Boye, Ph.D., and Founder and Chief Technology Officer Sanford Boye, M.Sc., at the University of Florida.

“We are thrilled that our gene therapy for LCA1 is coming home to Atsena and that we will have the opportunity to further its development,” said Shannon Boye. “Atsena was founded to advance treatments for inherited retinal diseases and believes in centering patients’ perspectives and needs in all we do. We are honored to continue to work with LCA1 patients and their families as we strive to treat this debilitating disease.”

LCA is the most common cause of blindness in children, impacting two to three per 100,000. 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.

Atsena has an ongoing Phase I/II clinical trial evaluating this gene therapy in LCA1 patients. The second cohort in the trial is expected to be dosed in the fall of 2020.

“Atsena’s gene therapy has the potential to be a major advance in treating blindness in both children and adults affected by this inherited retinal disease,” said Benjamin Yerxa, Ph.D., Chief Executive Officer of the Foundation Fighting Blindness and Atsena board director. “The foundation was instrumental in supporting proof of concept studies in the founders’ labs over the last 15 years. Now, via investment in Atsena through our Retinal Degeneration (RD) Fund, we are excited to support this potential breakthrough treatment for LCA1.”

Atsena closed a Series 1 funding of $8.15 million in April 2020, led by founding investors Hatteras Venture Partners and the Foundation Fighting Blindness’ RD Fund with participation by Osage University Partners, PBM Capital and the University of Florida. Patrick Ritschel, M.B.A., co-founder and former President of gene therapy company StrideBio, serves as Atsena’s Chief Executive Officer.

“Atsena is pleased to have the support of an enthusiastic investor base that shares our dedication to bringing the life-changing power of genetic medicine to patients living with LCA1 and other forms of blindness,” said Ritschel. “We look forward to working closely with our investors and patients as we continue to grow, and expect to announce additional milestones later this year.”

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company, focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness. The company has an ongoing Phase I/II clinical trial 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 the hurdles presented by inherited retinal disease, and its approach is guided by the specific needs of each patient condition. Founded by pioneers in ocular gene therapy, Atsena has a licensing, research and manufacturing collaboration with the University of Florida and is headquartered 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]