Press Releases

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
handerson@6degreespr.com

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
handerson@6degreespr.com

ALEIGH, N.C., Nov. 08, 2021 (GLOBE NEWSWIRE) — Opus Genetics, a patient-focused gene therapy company developing treatments for orphan inherited retinal diseases, today announced that Ben Yerxa, Ph.D., acting CEO of Opus and CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, will deliver a company presentation at Eyecelerator@AAO 2021, on Thursday, November 11, 2021, in New Orleans.

Dr. Yerxa’s Opus presentation will be part of the Retina Spotlight: Meet the Game-Changing Innovators With the Most Promising Strategies For Impacting Retina session, which includes a panel and a diverse line-up of five companies presenting innovations at all stages in a unique, interactive format.

“We’re thrilled to be invited to introduce Opus and highlight our innovative model at Eyecelerator,” said Dr. Yerxa. “Created by the Foundation’s RD Fund, Opus emerged from stealth mode in September singularly focused on developing and manufacturing orphaned AAV-based gene therapies to treat neglected, orphan inherited retinal diseases. We look forward to showcasing our unique, patient-guided approach at this premiere industry event.”

Eyecelerator@AAO 2021 is ophthalmology’s next-generation business conference that brings together leading ophthalmic companies and important new startups for fresh perspectives. The event was created in partnership between the American Academy of Ophthalmology (AAO) and the American Society of Cataract and Refractive Surgery (ASCRS) and occurs the day prior to the American Academy of Ophthalmology (AAO) 2021 Annual Meeting. For more information, visit www.eyecelerator.com.

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
handerson@6degreespr.com

Dr. Ash Jayagopal named Chief Scientific Officer
Mr. Joe Schachle appointed Chief Operating Officer

RALEIGH, N.C., Oct. 20, 2021 (GLOBE NEWSWIRE) — Opus Genetics, a patient-focused gene therapy company developing treatments for orphan inherited retinal diseases, today announced two key appointments to its founding executive team. Ash Jayagopal, Ph.D., has joined the company as Chief Scientific Officer (CSO) and Joe Schachle has joined as Chief Operating Officer (COO).

“We were thrilled to launch Opus last month to advance an AAV-based gene therapy portfolio to treat neglected, orphan inherited retinal diseases, and we welcome Ash and Joe’s leadership at this important and foundational time for the Company,” said Ben Yerxa, Ph.D., CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, and acting CEO of Opus. “Ash’s career discovering and developing therapies for ocular diseases makes him well-suited for the role of Opus CSO, where he will be instrumental in the advancement of our initial programs OPGx-001 and OPGx-002 for Leber congenital amaurosis. Additionally, Joe brings deep operational experience to his role as COO, specifically his expertise in strategic planning, business development and operations, which will be invaluable as we build our company and create novel orphan manufacturing scale and efficiencies.”

Dr. Jayagopal has more than 13 years of experience in drug development, drug delivery platforms and biomarker development for retinal diseases. Prior to joining Opus, Dr. Jayagopal served as the executive director of discovery medicine at Kodiak Sciences, where he led the drug discovery team and shaped the strategy for leveraging Kodiak’s biopolymer technology for delivery of large and small molecules in retinal diseases. Previously, Dr. Jayagopal was head of molecular pharmacology and biomarkers in ophthalmology at Roche, where he built and led a team of more than 25 scientists focused on the discovery and validation of biologics, small molecules, and gene therapies for ocular diseases, including inherited retinal diseases. Dr. Jayagopal has also served as an assistant professor in the departments of ophthalmology and visual sciences at the Vanderbilt Eye Institute, and the departments of molecular physiology and biophysics of Vanderbilt University Medical Center.

“Opus was formed to bring an unprecedented combination of resources, elite science and the expertise of pioneers in ocular gene therapy to bear and drive transformative treatments to patients,” said Dr. Jayagopal. “With its advanced pipeline – unique for a company at this stage – Opus has the potential to be clinical-stage in the near-term, and I look forward to working with the team to break new scientific ground in the pursuit of better treatments for inherited retinal diseases.”

Mr. Schachle brings more than 30 years of experience in life sciences to Opus, with specific expertise in strategic and operational planning, business development, marketing and sales, and business intelligence. Before joining Opus, Mr. Schachle served as vice president of Customer Experience Enablement and vice president of global commercial services and controlling at Grifols, where he led multiple commercial departments across business units, managed key cross-divisional initiatives and directed strategic brand planning process. Previously, Mr. Schachle was the COO for Parion Sciences, and part of the team that secured partners for the company’s lead programs, which exceeded $1 billion in deal value. Mr. Schachle has also served as chief commercial officer for Inspire Pharmaceuticals, where he oversaw multiple partnering deals and promoted three eye care brands, including Restasis®. In addition, he’s held multiple sales and marketing leadership positions at GlaxoSmithKline, where he managed several billion-dollar brands including Advair®, Imitrex®, Wellbutrin SR®, Epivir® / Retrovir® and Combivir®.

“Opus is built by and for patients to efficiently move validated science toward patients leveraging scalable, strategic manufacturing and processes,” said Mr. Schachle. “I look forward to leading the work to operationalize this first-of-its-kind model to address significant unmet need in the treatment of neglected, orphan inherited retinal diseases.”

Dr. Jayagopal holds a Ph.D. in biomedical engineering from Vanderbilt University and an MBA from the Kelley School of Business, Indiana University. Mr. Schachle holds a B.A. in business administration from James Madison University and an MBA from Old Dominion 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 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
handerson@6degreespr.com

Initial programs will focus on treatments for rare pediatric blinding conditions
Company formed to advance the work of scientific cofounders Dr. Jean Bennett, Junwei Sun and Dr. Eric Pierce

RALEIGH, N.C., Sept. 22, 2021 (GLOBE NEWSWIRE) — The Retinal Degeneration Fund (RD Fund), the venture arm of the Foundation Fighting Blindness aimed at rapidly driving research toward preventions, treatments and cures for the entire spectrum of retinal degenerative diseases, today announced the launch of Opus Genetics, a patient-focused gene therapy company efficiently developing therapies for orphan inherited retinal diseases. The $19 million in seed financing was led by the RD Fund with participation from the Manning Family Foundation and Bios Partners.

This is the first spin-out company internally conceived and launched by the RD Fund to further the Foundation’s mission. The initial seed funding will allow Opus to advance the preclinical research of its scientific founders, Jean Bennett, M.D., Ph.D., the F.M. Kirby Emeritus Professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania, and Junwei Sun, chief administrator of Penn’s Center for Advanced Retinal Ocular Therapeutics (CAROT), and Eric Pierce, M.D., Ph.D., William F. Chatlos Professor of Ophthalmology at Harvard Medical School and Massachusetts Eye and Ear. Dr. Bennett and Mr. Sun are also members of the Spark Therapeutics founding team.

“I’ve dedicated my career to the research and development of treatments for blinding diseases, and I’m eager to continue to build on this work with the RD Fund, an organization that understands the science and is deeply ingrained in the patient community,” said Dr. Bennett. “Founding Opus enables us to progress our first two programs in Leber congenital amaurosis while building an engine to move additional treatments toward the patients who need them.”

The company’s lead programs are licensed from the University of Pennsylvania and will focus on treatments to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA). LCA is a group of rare inherited retinal diseases that typically present in infancy and are characterized by degeneration of photoreceptors, the cells in the retina that make vision possible. Opus’s lead program, OPGx-001, is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein. LCA5 is one of the most severe forms of LCA, and affects approximately one in 1.7 million people. 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), which affects one in 288,000 people. Recent preclinical data have demonstrated the potential for both of these novel approaches to restore structure and function. Opus expects to file an IND for its OPGx-001 program in early 2022, and enter the clinic in mid-2022.

“Opus is a first-of-its-kind model for patient-focused therapeutic development. As the first company launched by the Foundation’s venture arm, RD Fund, Opus is uniquely positioned to bring experts, resources and patients together to efficiently advance ocular gene therapies for small groups of patients that to date have been neglected,” said Ben Yerxa, Ph.D., CEO of the Foundation and the RD Fund, and acting CEO of Opus. “We’re grateful for our fellow investors and supporters who share our commitment to realizing the promise of improving vision for people with devastating sight-limiting diseases, and look forward to building upon the pioneering work of Dr. Bennett, Mr. Sun and Dr. Pierce, and expanding our pipeline with more programs soon.”

In addition to Dr. Yerxa, the company is co-founded and managed by Rusty Kelley, Ph.D., Peter Ginsberg, and Jason Menzo, who also form the management team of the RD Fund. The Board for Opus is comprised of Drs. Yerxa, Kelley and Bennett.

“While potential treatments for these ultra-rare conditions have existed for years, families have been stuck in a holding pattern waiting on someone to deliver a feasible business model to bring them to market,” said Paul Manning, Manning Family Foundation. “We’re thrilled to be a part of the launch of Opus Genetics to establish a patient-first priority and build capabilities to tackle manufacturing obstacles and access to life-altering treatments for the people who need them most.”

For more information, visit www.opusgenetics.com.

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.

About The Retinal Degeneration Fund
The Retinal Degeneration Fund (RD Fund) is the venture arm of the Foundation Fighting Blindness, and a leading investor in the Inherited Retinal Disease space. It was established in 2018 to serve the Foundation’s mission to rapidly drive research toward preventions, treatments and cures for the entire spectrum of blinding retinal diseases—including retinitis pigmentosa, macular degeneration, and Usher syndrome. RD Fund focuses on mission-related investments in companies with projects nearing clinical testing.

About Foundation Fighting Blindness
Established in 1971, the Foundation Fighting Blindness is the world’s leading private funding source for retinal degenerative disease research. The Foundation has raised more than $750 million toward its mission of preventing, treating, and curing blindness caused by the entire spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt disease. Visit www.FightingBlindness.org for more information.

University of Pennsylvania Financial Disclosure
Dr. Jean Bennett and Junwei Sun are scientific collaborators, advisors and co-founders of Opus Genetics. As such, they hold an equity stake in the Company, and as inventors of the licensed technology may receive additional future financial benefits under licenses granted by Penn to Opus Genetics. Dr. Bennett’s laboratory at Penn receives sponsored research funding from Opus Genetics. The University of Pennsylvania also holds equity and licensing interests in Opus Genetics.

Media contacts:

For The Retinal Degeneration Fund:
Jason Menzo
317-489-7283
jmenzo@FightingBlindness.org

For Opus Genetics:
Heather Anderson
919-827-5539
handerson@6degreespr.com