Press Releases

Opus Genetics Receives FDA Clearance of IND Application for OPGx-001, a Gene Therapy Candidate Intended for the Treatment of Rare Inherited Retinal Disease LCA5

OPGx-001 is Opus’ first program to enter clinical evaluation and is designed to address vision loss due to mutations in the LCA5 gene, which causes one of the most severe forms of early-onset blinding disease Leber congenital amaurosis

Company anticipates initiating a Phase 1/2 clinical trial in early 2023 in the U.S.

 Research Triangle Park, N.C. – Dec. 1, 2022 – Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for a Phase 1/2, first-in-human clinical trial of OPGx-001 in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5). OPGx-001 is an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5gene to retinal photoreceptors. Currently, there are no approved treatments for individuals with LCA5-related vision loss.

“We founded Opus a little more than a year ago to quickly move promising potential treatments into the clinic for patients in need. This FDA clearance of our IND application for OPGx-001 for LCA5 marks a significant milestone for Opus, as our first program to enter the clinic,” said Ben Yerxa, Ph.D., Chief Executive Officer of Opus. “Preclinical studies in in vitro and in vivo models of LCA5 have provided support for the safety and efficacy of OPGx-001. We look forward to initiating our first-in-human trial of OPGx-001 in early 2023 and to continuing to build and advance our pipeline of gene therapies for unaddressed inherited retinal diseases in parallel.”

The Phase 1/2, open-label, dose-escalation trial will evaluate the subretinal delivery of OPGx-001 in nine adult patients with LCA5. The objective of the trial is to evaluate safety and potential benefit. Once safety in adults has been cleared, Opus plans to add a pediatric cohort.

For more information, visit clinicaltrials.gov (NCT05616793).

About OPGx-001

OPGx-001 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5 is an early-onset severe inherited retinal degeneration. Studies in LCA5 patients have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-001 utilizes an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to photoreceptors in the retina. Preclinical data, including animal and human iPSC models, have demonstrated preservation of retinal structure and visual function when OPGx-001 was administered prior to peak disease severity.

 About Opus Genetics

Opus Genetics is a clinical-stage gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’ 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.

Opus Media Contact:

Heather Anderson
6 Degrees
919-827-5539

handerson@6degreespr.com

Opus Genetics Leadership to Present at BTIG Ophthalmology Day

Research Triangle Park, N.C. – Nov. 22, 2022 – Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced that Ben Yerxa, Ph.D., Chief Executive Officer of Opus, will participate in a fireside chat during the virtual BTIG Ophthalmology Day on Tuesday, November 29, 2022, at 2:30 p.m. ET.

BTIG hosted events are intended for prospective and existing BTIG clients only. To listen to the live event, please contact your BTIG representative with interest. For more information on Opus’ upcoming events and presentations, click here.

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.

 

Opus Media Contact:
Heather Anderson
6 Degrees
919-827-5539

handerson@6degreespr.com

Opus Genetics Sponsors Uni-Rare Natural History Study for People with Inherited Retinal Diseases

Largest study to date to characterize natural history of rare retinal diseases

Research Triangle Park, N.C. – Nov. 14, 2022 – Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced the company will co-sponsor the Uni-Rare Study, a new natural history study for approximately 1,500 people with mutations in one of more than 300 rare genes associated with inherited retinal diseases (IRDs).

The two-part Uni-Rare Study is being launched to characterize the course of retinal degeneration and vision loss for people with these rare genetic mutations, as well as inform the design of future clinical trials and assist in trial recruitment. The study will be conducted by the Foundation Fighting Blindness Clinical Consortium, a global, 40-site network of clinical research centers prepared to launch IRD clinical trials and natural history studies, with the Jaeb Center for Health Research to serve as the coordinating center.

“Opus is building a strong portfolio of programs focused on treating genetic mutations that cause inherited retinal diseases. The natural history data collected as part of the Foundation’s Uni-Rare Study, a first of its kind in retinal diseases, will be critical to the future research and development of new therapies for inherited retinal diseases. Opus’ second program, OPGx-002, is focused on restoring protein expression and halting functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene, which is one of the genetic mutations that currently qualifies for the advanced portion of the Uni-Rare study,” said Jennifer Hunt, Chief Development Officer. “We’re proud to join with other leading ophthalmology organizations in supporting this important step forward to accelerate our understanding and development of new treatments for inherited retinal diseases.”

The Foundation Fighting Blindness is the world’s leading private funding source for retinal degenerative disease research. The Foundation has raised more than $891 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of blinding retinal diseases. For more information on the Uni-Rare Study, please read the Foundation’s press release or visit www.clinicaltrials.gov.

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

handerson@6degreespr.com

Opus Genetics Expands Board of Directors with Appointment of Global Ophthalmology Leader Dr. Adrienne Graves

Research Triangle Park, N.C. – Sept. 27, 2022 – Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced the appointment of Adrienne Graves, Ph.D., to its board of directors. Dr. Graves is the former CEO of Santen Inc., where she was responsible for growing the company’s global presence and advancing multiple ophthalmic products through development to approval and commercialization.

“On behalf of the Opus team and the board of directors, we welcome Adrienne to our board at this exciting time for the company as we progress our first three programs to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA) toward the clinic,” said Ben Yerxa, Ph.D., Chief Executive Officer of Opus. “Her nearly three decades of experience as a global executive and board member in the ophthalmology industry will be invaluable to Opus’ clinical and development efforts.”

Prior to Santen, she spent nine years at Alcon in roles of increasing responsibility, including director of International Ophthalmology, and establishing Alcon’s first Retinal Electrophysiology Laboratory. Dr. Graves also currently serves as board chair for the Foundation Fighting Blindness’ Retinal Degeneration (RD) Fund and Iveric Bio, and as a board director for Qlaris Bio, Nicox S.A., Surface Ophthalmics, Oxurion, TherOptix, and Greenbrook TMS, American Society of Cataract and Refractive Surgery (ASCRS) Foundation, Glaucoma Research Foundation, American Academy of Ophthalmology Foundation (Emeritus), Retina Global, Himalayan Cataract Project, and the Foundation Fighting Blindness. She earned her Ph.D. in Psychobiology/Neuroscience from the University of Michigan and completed a Postdoctoral Fellowship in Visual Neuroscience at the University of Paris.

“With its roots firmly in the patient community, Opus represents a new kind of company that combines unmatched scientific expertise and a scalable approach designed to bring transformative treatments to patients with inherited retinal diseases. The progress they’ve already made in their preclinical development programs is impressive,” said Dr. Graves. “I’ve dedicated my career to advancing the science and development of therapies to preserve and restore vision, and I look forward to bringing that experience to bear to support the Opus team in the work ahead.”

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
handerson@6degreespr.com

Opus Genetics Leadership to Present at 3rd Annual Gene Therapy for Ophthalmic Disorders Conference

Research Triangle Park, N.C. – September 12, 2022 – Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced that Chief Scientific Officer Ash Jayagopal, Ph.D., and Vice President of Clinical Development Erin O’Neil, M.D., will present at the 3rd Annual Gene Therapy for Ophthalmic Disorders meeting taking place September 13-16, 2022, in Boston, Mass.

Details for the presentations are as follows:

Title: Establishing a Sustainable Gene Therapy Pipeline for Inherited Retinal Diseases
Track: Day One, Track B (Clinical Stream)
Date and Time: September 14, 2022, 2:55 p.m. ET
Presenter: Dr. Ash Jayagopal, Opus Chief Scientific Officer

Title: Identifying the Right Endpoints for Your Therapy to Optimize Your Clinical Trials
Track: Day Two, Track B (Clinical Stream)
Date and Time: September 15, 2022, 11:30 a.m. ET
Presenter: Dr. Erin O’Neil, Opus Vice President of Clinical Development and Children’s Hospital of Philadelphia Physician

In addition, Opus leadership will be attending the PDA/FDA Joint Regulatory Conference taking place September 12-14, 2022, in Washington, D.C., and the American Academy of Ophthalmology Annual Meeting taking place September 30-October 3, 2022, in Chicago, Ill.

For more information on Opus’ upcoming events and presentations, click here.

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

handerson@6degreespr.com

Opus Genetics Hosts Inaugural Patient Advocacy Outreach Webinar on Inherited Retinal Diseases

Virtual event details Opus’ unique and transparent approach to building company centered on patient need, provides update on lead programs in Leber congenital amaurosis

Research Triangle Park, N.C. – Sept. 1, 2022  – Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced the release of the first patient advocacy outreach webinar featuring presentations by Opus leadership Ben Yerxa, Ph.D., Chief Executive Officer; Ash Jayagopal, Ph.D., Chief Scientific Officer; Joe Schachle, Chief Operating Officer; and Jennifer Hunt, Chief Development Officer. The recorded webinar, intended for inherited retinal disease (IRD) patients and their families, provides a corporate overview, program update and information on inherited retinal diseases.

“Opus Genetics is the first-of-its-kind company created by patients, for patients, and an integral component of our unique approach is building a company driven by transparency and trust,” said Dr. Yerxa. “Our commitment to patients extends beyond tackling some of the biggest unmet needs in inherited retinal diseases, to prioritizing patient voices throughout this journey. We are pleased to launch our first patient outreach webinar to share updates on our programs and increase understanding of the debilitating rare diseases that we are addressing. We are dedicated to continuing a dialogue with the patient community to share updates on our work through webinars, and other means, as we advance our pipeline of AAV-based gene therapies for blinding diseases.”

Opus currently has three lead programs being developed to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA). The webinar highlighted recent and upcoming milestones in each program, including:

  • LCA5: Opus is preparing an Investigational New Drug (IND) application to be filed with the U.S. Food and Drug Administration (FDA) by the end of 2022. The clinical trial will be completed at the University of Pennsylvania and Children’s Hospital of Philadelphia.
  • RDH12: Preclinical animal studies are ongoing. Opus anticipates filing an IND with the FDA in the second half of 2023.
  • NMNAT1: Initial preclinical animal studies showed signs of efficacy and additional preclinical animal studies are to be completed. Opus plans to request a pre-IND meeting with the FDA in 2022 to inform next steps and timing of a clinical trial.

Watch the webinar on Opus’ YouTube channel here:
https://www.youtube.com/watch?v=mtFe8ZuFXmM. Opus plans to offer patient webinars twice per year. For patient or family inquiries, please contact patientsupport@opusgtx.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 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

handerson@6degreespr.com

Opus Genetics Expands its Leadership Team

Vikram Arora, Ph.D., DABT, appointed Vice President of Non-Clinical Development

Erin O’Neil, M.D., appointed Vice President of Clinical Development

Sarah Tuller, J.D., RAC, appointed Vice President of Regulatory and Medical Writing

Research Triangle Park, N.C. – July 21, 2022 – Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced the appointments of Vikram Arora, Ph.D., DABT, as Vice President of Non-Clinical Development; Erin O’Neil, M.D., as Vice President of Clinical Development; and Sarah Tuller, J.D., RAC, as Vice President of Regulatory Affairs and Medical Writing. Dr. Arora will report to Opus Chief Scientific Officer Ash Jayagopal, Ph.D., and Drs. O’Neil and Tuller will report to Opus Chief Development Officer Jennifer Hunt.

“Vik, Erin and Sarah are all seasoned, accomplished leaders in their respective areas of expertise and we welcome them to our growing Opus Genetics team at this exciting time for the company,” said Ben Yerxa, Ph.D., CEO of Opus Genetics. “We envision a new, efficient model to transform the treatment of rare inherited retinal diseases, and these leaders are the tip of the spear for the important work to come as we prepare for our first program to enter the clinic and to continue to develop our pipeline.”

Dr. Arora brings over 20 years of research and development experience in pharmacology and toxicology spanning early drug discovery to IND-enabling non-clinical programs to support global regulatory submissions for commercial therapeutics including Xembify®, Prolastin-C® and HyperRAB®. Dr. Arora joins Opus following 11 years of leadership at Grifols where he led late-stage discovery research and subsequent development aspects of R&D projects, including pharmacology/toxicology, pharmacokinetics and viral safety for candidates in the pipeline. He also acted as the lead non-clinical R&D representative to regulatory agencies. Prior to Grifols, Dr. Arora served as Director of Pharmacology & Toxicology at Talecris Biotherapeutics, Principal Research Scientist at Bayer HealthCare, and Team Leader and Post-Doctoral Fellow at AVI BioPharma (now Sarepta Therapeutics) where he conducted preclinical studies leading to some of the earliest first-in-human trials of antisense-based gene therapies. Dr. Arora is a published investigator and patent holder in the areas of pharmacology, toxicology, genetics, drug metabolism and pharmacokinetics. He earned his Ph.D. in Pharmacology and Toxicology from the University of Nebraska Medical Center and a B.S. in Human Biology from the All India Institute of Medical Sciences.

“Vik’s deep pharmacology and toxicology experience will be invaluable in advancing Opus assets from inception to IND, and in spearheading the regulatory management of our preclinical programs,” said Dr. Jayagopal. “We’ve assembled an impressive cadre of talent on our scientific team, including recent additions of Director of Gene Therapy Scott Greenwald, Gene Therapy Senior Scientist Mayur Choudhary and Senior Scientific Fellow Krishna Fisher. With the appointment of Vik, we’ve rounded out the scientific foundation to bring forward first-in-class retinal gene therapies.”

Dr. O’Neil also currently serves as Attending Ophthalmologist at the Center for Inherited Retinal Degenerations and Ophthalmic Genetics at the Children’s Hospital of Philadelphia (CHOP), where she maintains a clinical affiliation. In this role, she oversees the treatment of pediatric patients with retinal degenerations and ocular manifestations of genetic disease. Dr. O’Neil also served as a principal investigator on a Foundation Fighting Blindness trial optimizing gene therapy for Choroideremia, for which she was awarded the Diana Davis Spencer Clinical Fellowship Award. Prior to her medical career, Dr. O’Neil served in business operations and marketing roles, including a role as Associate Product Marketing Manager at Google. Dr. O’Neil earned her M.D. from the University of Pennsylvania, a Postbaccalaureate Premedical Certificate from Bryn Mawr College and a B.A. with a Minor in Economics in International Relations from Pomona College.

Ms. Tuller has over 20 years of experience in regulatory strategy and operations, spanning numerous clinical and commercial-stage products across a wide range of therapeutic indications, including multiple orphan disease programs. She has led or been a part of numerous successful IND, NDA, MAA and BLA applications, including those of Portrazza® in metastatic, squamous NSCLC, Phoslyra® in ESRD, and Avonex Pen® in relapsing MS. Prior to joining Opus, she was Vice President RA at Disc Medicine, and held regulatory leadership positions at Astellas Pharma, where she supported the ophthalmology gene therapy, mitochondrial diseases, and regenerative (cell) medicinal product franchises, as well as prior appointments of increasing regulatory responsibility at companies including Biogen, ImClone, Fresenius, and Baxter. She earned her J.D. in IP law from the University of Dayton School of Law, holds a B.S. in Chemistry, and is RAC certified.

“I’m thrilled to welcome Erin and Sarah to the Opus development team,” said Ms. Hunt. “Opus’ goal is to maintain a steady clinical pace and file one IND per year. With her inherited retinal disease gene therapy clinical development and patient care experience, Erin is well suited to lead Opus’ clinical program development. Combined with Sarah’s background in clinical regulatory affairs and impressive track record of shepherding successful IND fillings, we have the development team leadership in place to expeditiously bring our programs through the clinic and to patients in need.”

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

handerson@6degreespr.com