Press Releases

Research Triangle Park, N.C. – July 11, 2022 – Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced that Ben Yerxa, Ph.D., president and CEO of Opus, will present at the Ophthalmology Innovation Source (OIS) Retina Innovation Summit 2022 on Wednesday, July 13, 2022, in New York.

Dr. Yerxa will deliver a company overview as part of the Innovation Showcase to discuss Opus’ unique model of combining validated science and commitment to patient need with wholly owned programs in inherited retinal diseases. Dr. Yerxa will also highlight Opus’ lead AAV-based gene therapy programs being developed to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA).

“We’re honored to be selected to participate in the OIS Innovation Showcase and highlight Opus’ patient-first, science-driven approach and pipeline of AAV-based gene therapy programs,” said Dr. Yerxa. “Opus’ mission is to build an engine to accelerate multiple rare genetic retinal disease therapeutics into the clinic and ultimately to the patients who need them. We look forward to providing an update on our LCA programs approaching clinical stage in the near-term, as well as our long-term approach to addressing additional genetic targets across the broad spectrum of inherited retinal diseases.”

The OIS Retina Innovation Summit brings together entrepreneurs, ophthalmic start-up companies, clinical thought leaders, industry executives and investment professionals for a day-long summit showcasing novel therapies in development for ophthalmic diseases and vision disorders. For more information, visit https://ois.net/ois-retina-innovation-summit-2022/.

About Opus Genetics
Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com.

Media Contact:
Heather Anderson

6 Degrees
919-827-5539
[email protected]

Industry veteran and experienced CEO will continue to lead the company as its AAV-based gene therapies for inherited retinal diseases approach the clinic

Research Triangle Park, N.C. – June 21, 2022 – Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced the appointment of Ben Yerxa, Ph.D., as Chief Executive Officer (CEO). Dr. Yerxa previously served as acting CEO of Opus, in addition to former roles as CEO of the Foundation Fighting Blindness and the Retinal Degeneration (RD) Fund, the venture arm of the Foundation focused on making venture philanthropy investments to further the Foundation’s mission of accelerating research for preventing, treating and curing blinding diseases.

Opus is the first spin-off company internally conceived and launched by the RD Fund, and combines unparalleled insight and commitment to patient need with wholly owned AAV-based gene therapy programs in inherited retinal diseases, while creating novel manufacturing scale and efficiencies tailored for producing rare disease therapeutics.

“I’m exceptionally proud of the progress we’ve made toward our mission during my nearly five years at the Foundation, including the launch of the RD Fund to further the Foundation’s mission through venture philanthropy,” said Dr. Yerxa. “Stepping into the permanent CEO role at Opus Genetics – a company that we created and launched through the RD Fund – enables me to be on the front line of bringing potentially life-changing treatments to the patients who so urgently need them.”

Dr. Yerxa continued, “More than 280 genes are known to cause inherited retinal diseases, and it’s estimated that over 180,000 patients are waiting for treatments for their individual genetic conditions. Opus is combining the best instruments of science with patient need to grow and advance our pipeline of AAV-based gene therapies for inherited retinal diseases, and I’m pleased to continue leading the Opus team in this important work.”

Dr. Yerxa has more than 25 years’ experience in biotechnology, nonprofit and drug development and in translating promising research discoveries into clinical milestones and commercial launches in the ophthalmology, pulmonary, rare disease, cardiovascular and HIV fields. He has been involved with the discovery and development of nine investigational new drugs, four Phase 3 clinical programs, two new drug applications and two drug approvals. Prior to joining the Foundation, Dr. Yerxa served as president and co-founder of Envisia Therapeutics, a company focused on developing novel ocular sustained delivery therapies for the front and back of the eye. He also has previously held founding and executive positions in several ophthalmology-based R&D organizations, including Liquidia Technologies, Clearside Biomedical, Parion Sciences and Inspire Pharmaceuticals.

“Opus is entering a pivotal time in its evolution as our first program, OPGx-001 to address mutations in the LCA5 gene, is expected to enter the clinic this year, bringing a potential treatment another step closer to patients in need,” said Jean Bennett, M.D., Ph.D., Opus scientific founder, board director and scientific advisory board member. “As a founder of the RD Fund and Opus, Ben is uniquely well-suited to realize Opus’ potential to tackle some of the most neglected forms of inherited blindness and make a real difference in the lives of patients.”

Dr. Yerxa holds 60 U.S. patents and is an inventor of DIQUAS™, an innovative treatment for dry eye approved in Japan. Dr. Yerxa earned his Ph.D. in organic chemistry from University of California, Irvine, and BA in chemistry from the University of California, San Diego.

About Opus Genetics
Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com.

Media Contact:
Heather Anderson

6 Degrees
919-827-5539
[email protected]

Biopharma clinical development veteran to propel Opus’ AAV-based gene therapies for inherited retinal diseases toward patients

Research Triangle Park, N.C. – May 11, 2022 – Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced the appointment of Jennifer Hunt as Chief Development Officer (CDO). In this role, Hunt will lead clinical development and regulatory affairs, responsible for progressing and expanding Opus’ AAV-based gene therapy pipeline which currently includes preclinical candidates OPGx-001, OPGx-002 and OPGx-003 to address forms of Leber congenital amaurosis (LCA) due to mutations of LCA5, RDH12 and NMNAT1 genes, respectively.

“We’re pleased to welcome Jennifer at this exciting time for Opus, as we build out the team that will be foundational to realizing our mission to efficiently advance therapies for inherited retinal diseases,” Ben Yerxa, Ph.D., CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, and acting CEO of Opus. “Jennifer’s deep clinical development background and experience overseeing trials in rare diseases and LCA will be instrumental as we progress our current programs toward the patients that need these therapies.”

Hunt brings over 25 years of drug development experience to Opus, with specific expertise in global clinical operations, product development and program management in biologics, small molecules, gene editing and gene therapy. Prior to joining Opus, she held key clinical and regulatory positions at several biopharmaceutical companies, including Genzyme, Voyager Therapeutics and Editas Medicine. While at Editas, she oversaw the development of CRISPR medicines across ophthalmology, hemoglobinopathies and oncology, including Editas’ gene therapy to restore vision loss in patients with LCA type 10, the first in-vivo CRISPR IND ever accepted by the U.S. Food and Drug Administration.

“I am inspired by Opus’ passionate commitment to patient need,” said Hunt. “Opus is in a strong position to advance multiple therapies for inherited retinal diseases. I’m looking forward to leveraging my experience to tackle some of the most neglected forms of inherited blindness and make a difference in the lives of patients.”

Hunt earned a B.S. in Biology from State University of New York College of Environmental Science and Forestry at Syracuse and a M.S. in Management from Lesley University.

About Opus Genetics

Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com.

Media Contact:
Heather Anderson
6 Degrees
919-827-5539
[email protected]

Retinal regions of preserved photoreceptors identified as targets for subretinal delivery of AAV8-based gene therapy to address mutations in genes that cause forms of Leber congenital amaurosis

Subretinal injection well tolerated in preclinical dose-ranging studies

Data presented at Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2022 

Research Triangle Park, N.C. – May 4, 2022 – Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced promising new preclinical data from studies evaluating the potential of its gene therapies OPGx-001 and OPGx-002 to address forms of Leber congenital amaurosis (LCA), a group of rare inherited retinal diseases characterized by photoreceptor degeneration, due to mutations of LCA5 or RDH12 genes, respectively.

In preparation for IND-enabling trials of OPGx-001 and OPGx-002, studies were conducted to determine eligibility, therapeutic window, and possible outcome measures for gene therapy for LCA5 and RDH12 inherited retinal diseases. In addition, safety evaluations for the subretinal delivery of an AAV8 vector containing LCA5 or RDH12 were performed in non-human primates (NHP).

The data demonstrated that despite severe retinal dysfunction, LCA patients exhibited detectable photoreceptor regions that may be targets for gene augmentation, identified in the central and midperipheral retina of LCA5-LCA patients and in the pericentral and peripapillary retina of RDH12-LCA patients. In two dose-ranging studies in NHPs, subretinal delivery of OPGx-001 and OPGx-002 was well tolerated, with mild inflammatory changes observed at the higher dose. The data support the therapeutic potential and tolerability of gene augmentation to address LCA5-LCA and RDH12-LCA and provide guidance for formal preclinical toxicology studies and future human clinical trials.

“Patients with Leber congenital amaurosis due to mutations of the LCA5 or RDH12 genes experience rapid retinal degeneration, resulting in vision loss in early childhood,” said Ash Jayagopal, Ph.D., Chief Scientific Officer of Opus. “The detection of preserved photoreceptors in LCA patients signals a therapeutic opportunity to target the mutation and potentially restore structure and function through gene augmentation. In addition, the encouraging dose-ranging results in the primate model suggest subretinal delivery of Opus’ AAV8-based gene therapies are safe and inform the doses to be used in our toxicology studies, a key step on our path toward the clinic for OPGx-001 and OPGx-002.”

The data were presented today at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2022 in Denver, Colo., by Jean Bennett, M.D., Ph.D., Scientific Co-founder, Opus Genetics, and University of Pennsylvania Perelman School of Medicine; and Tomas Aleman, M.D., University of Pennsylvania Perelman School of Medicine.

Also at ARVO 2022, Dr. Jayagopal was awarded the title of ARVO Fellow, an honor established to recognize current ARVO members for their individual accomplishments, leadership and contributions to the ARVO Association.

About Opus Genetics

Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com.

Media Contact:
Heather Anderson
6 Degrees
919-827-5539
[email protected]

Data support the continued development of OPGx-001 and OPGx-002 to address mutations in genes causing forms of Leber congenital amaurosis

Raleigh, N.C. – April 28, 2022 – Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced that data from two preclinical studies evaluating the potential of its gene therapies OPGx-001 and OPGx-002 to address forms of Leber congenital amaurosis (LCA) due to mutations of LCA5 or RDH12 genes, respectively, will be presented in poster sessions at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2022, being held May 1-4, 2022, virtually and in Denver, Colo.

Details of the presentations are as follows:

Title: Preparation for a Gene Therapy Trial for LCA5-Associated Retinal Degenerations: Treatment Potential in Patients and Dose-ranging Studies in Non-human Primates

Session: Novel Animal Models and Neuroprotection in the Retina

Location: Virtual webcast

Date / time: Wednesday, May 4, 2022, 12:30-2:30 p.m. MDT

Presenter: Jean Bennett, M.D., Ph.D., Scientific Co-founder, Opus Genetics; Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine

 

Title: Preparation for a Gene Augmentation Trial for RDH12-Associated Retinal Degenerations

Session: Retinal Degenerations, Gene Therapy, Transplantation, and Prostheses

Location: F0299, Denver Convention Center

Date / time: Wednesday, May 4, 2022, 3-5 p.m. MDT

Presenter: Tomas Aleman, M.D., Center for Advanced Retinal and Ocular Therapeutics (CAROT), Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine

The ARVO Annual Meeting is the premiere gathering for eye and vision scientists to share the latest research findings and collaborate on innovative solutions. For more information, visit https://www.arvo.org/annual-meeting/.

In addition, Ash Jayagopal, Ph.D., Chief Scientific Officer of Opus Genetics, will present a company overview, titled “Opus Genetics, a patient-centric biotech committed to developing ultrarare IRD therapies,” at the Foundation Fighting Blindness’ 7th Annual Retinal Cell and Gene Therapy Innovation Summit on Friday, April 29, 2022, at 10:20 a.m. MDT, in Denver, Colo. The annual summit, held prior to the ARVO Annual Meeting, features presentations by leading retinal disease experts on potential gene and stem-cell therapies and how best to deliver them to patients. For more information, visit https://www.fightingblindness.org/events/innovation-summit-2022-372.

About Opus Genetics

Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies. Based in Raleigh, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com.

Media Contact:
Heather Anderson
919-827-5539

[email protected]

 Opus’ first-in-human trials to begin in 2022

Raleigh, N.C. – April 11, 2022 – Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced a strategic manufacturing services agreement with National Resilience, Inc. (Resilience), a technology-focused biomanufacturing company dedicated to broadening access to complex medicines, to support the development and manufacturing of Opus’ pipeline.

Resilience will provide process and analytical development, quality control testing, and GMP manufacturing services for IND-enabling toxicology and first-in-human material for Opus’ adeno-associated viral (AAV) vector-based gene therapies for inherited retinal diseases (IRD) for use in both preclinical studies, and future clinical trials in the U.S. The work will be conducted at Resilience’s facilities in Waltham, Mass., and Research Triangle Park, N.C.

“Resilience embraces our innovative model to create a clinical manufacturing infrastructure that’s scaled to address rare inherited retinal diseases, and we are pleased to enter into this strategic collaboration,” said Ben Yerxa, Ph.D., CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, and acting CEO of Opus. “Leveraging Resilience’s expertise puts Opus in the best position to efficiently advance our AAV-based gene therapies into the clinic and toward the patients who need them.”

“Helping Opus advance their gene therapies for inherited retinal diseases to the clinic will hopefully lead to new treatments for underserved patients,” said Rahul Singhvi, ScD, Chief Executive Officer of Resilience. “We’re excited to partner with Opus on process development and manufacturing at this pivotal time for the company.”

Opus’s lead program, OPGx-001, is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein. Opus anticipates filing an IND and entering first-in-human trials with OPGx-001 this year. Opus’ earlier stage programs include OPGx-002, to restore protein expression and halting functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene, and OPGx-003, a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 (NMNAT1) gene.

About Opus Genetics

Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies. Based in Raleigh, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com.

Media Contact:
Heather Anderson
919-827-5539
[email protected]

20-year biopharma industry veteran to lead manufacturing for Opus’ AAV-based gene therapies for inherited retinal diseases

RALEIGH, N.C., Feb. 15, 2022 (GLOBE NEWSWIRE) — Opus Genetics, a patient-focused gene therapy company developing treatments for orphan inherited retinal diseases, today announced the appointment of Brian Leising as Vice President of Manufacturing.
Mr. Leising has over 20 years of gene therapy, vaccine and biopharmaceutical experience in manufacturing, quality, facility startup and process development roles across a variety of products in both the clinical and commercial stages. In his role, Mr. Leising will lead Opus’ efforts with key partners to manufacture IND-enabling GLP toxicology and first-in-human material to support the continued advancement of Opus’ portfolio toward clinical trials to address orphan inherited retinal diseases. Opus anticipates filing an IND and entering first-in-human trials with lead program OPGx-001 in LCA5 this year.

“Opus is building an engine to solve as many degenerative retinal diseases as possible, and manufacturing is a lynchpin of our success in efficiently advancing new therapies for rare inherited retinal diseases into the clinic,” said Ben Yerxa, Ph.D., CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, and acting CEO of Opus. “Brian will be instrumental in the conceptualization and development of our manufacturing approach and we’re glad to have him on board as we work toward IND and our first-in-human trials later this year.”

Mr. Leising previously served as Director of Manufacturing Quality at Novartis Gene Therapies (formerly AveXis). In this role, he was responsible for implementation and oversight of the site quality systems for AAV gene therapy for the Durham facility startup, validation and licensure for Zolgensma, as well as for multiple clinical molecules. Prior to Novartis Gene Therapies, Mr. Leising was Director of Clinical Gene Therapy Manufacturing at Pfizer’s former Bamboo facility overseeing Phase 1 manufacturing of their AAV-based gene therapies and future clinical manufacturing infrastructure. Mr. Leising holds an M.S. in biotechnology from the University of Maryland and a B.S. in biology from the Florida Institute of Technology.

“Opus has an incredible opportunity to move strong and validated science and well-characterized material into the clinic expeditiously,” said Mr. Leising. “I’m thrilled to join Opus to help drive the development of a scalable, strategic manufacturing infrastructure that can be leveraged for IND, clinical and commercial stages, for multiple products.”

About Opus Genetics
Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Raleigh, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com.

Media Contact:
Heather Anderson
919-827-5539
[email protected]

Program targets NMNAT1 gene based on the work of Opus scientific founder Dr. Eric Pierce

RALEIGH, N.C., Nov. 11, 2021 (GLOBE NEWSWIRE) — Opus Genetics, a patient-focused gene therapy company developing treatments for orphan inherited retinal diseases, today announced an agreement to license its third preclinical program to address mutations in the NMNAT1 gene, which cause a specific form of Leber congenital amaurosis (LCA), from Massachusetts Eye and Ear, the primary teaching hospital at Harvard Ophthalmology.

The new program, OPGx-003, is based on the work of Eric Pierce, M.D., Ph.D., Director of the Ocular Genomics Institute and William F. Chatlos Professor of Ophthalmology at Massachusetts Eye and Ear and Harvard Medical School, and scientific co-founder of Opus. OPGx-003 is a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 (NMNAT1) gene. Recent preclinical data have demonstrated the potential for this gene augmentation approach to achieve stable rescue of retinal structure and function. Opus expects to file an IND for OPGx-003 in the first half of 2023.

“We’re thrilled to collaborate with Dr. Pierce, who will bring his expertise in retinal gene therapy to researching a novel treatment of NMNAT1-associated retinal degeneration,” said Ash Jayagopal, Ph.D., Chief Scientific Officer of Opus Genetics. “Adding OPGx-003 to the Opus pipeline further underscores our commitment to bringing the required resources and expertise together to take promising science from the lab through the clinic and ultimately to patients who need it, and reinforces the importance of our innovative patient-focused model.”

“I’ve seen firsthand the need for new treatments for rare inherited retinal diseases,” said Dr. Pierce. “As someone who has devoted my life to research in this space, I believe strongly that Opus is an ideal company to advance this work to make a difference for patients in need.”

Opus’s lead program, OPGx-001, is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein. The company’s second program, OPGx-002, will focus on restoring protein expression and halting functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene (LCA13).

About Opus Genetics
Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Raleigh, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com.

Disclosures
Dr. Pierce holds equity in and serves as a consultant for Opus Genetics.

Media Contact:
Heather Anderson
919-827-5539
[email protected]