Press Releases

$1M TRAP award to support preclinical safety study for gene therapy vector targeting rhodopsin- RHO-adRP

Additional project and operational funding to support MERTK gene therapy IND-enabling studies

RESEARCH TRIANGLE PARK, N.C., June 13, 2024 (GLOBE NEWSWIRE) — Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced it has received $1.7 million in project-based funding from the Foundation Fighting Blindness to help advance two preclinical candidate programs.

“Opus is immensely grateful for the generous support from the Foundation Fighting Blindness, which will catalyze our efforts in pioneering treatments for inherited retinal diseases. This $1.7 million funding infusion makes a significant impact to help accelerate the development of these two preclinical candidates,” said Ben Yerxa, Ph.D., chief executive officer of Opus. “Together, we strive toward a future where therapies to help treat patients with inherited retinal diseases are readily available.”

Opus received a $1M Translational Research Acceleration Program (TRAP) award to conduct a preclinical safety study for a gene therapy vector designed to target rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP). The safety study will be conducted in the established canine animal model at the University of Pennsylvania School of Veterinary Medicine (Penn Vet). The Company anticipates this is the last preclinical study required before the gene therapy will enter clinical trials. RHO-adRP is one of the most common IRDs, estimated to affect approximately one in 51,000 people, or more than 6,000 people, in the United States alone.

Additionally, Opus received approximately $700,000 in project-based and operational funding to support the preclinical development of a novel viral vector for treating retinitis pigmentosa due to mutations in the proto-oncogene tyrosine-protein kinase MER (MERTK) gene. The Company is collaborating with the Foundation to begin IND-enabling studies for a newly designed adeno-associated virus (AAV) viral vector to replace mutated MERTK genes in the retinal pigmented epithelial (RPE) cells of the retina. The initial funding will provide the resources for testing the vector in an established animal model of the disease and to conduct early safety assessments in larger animals. MERTK mutations cause a rod-cone dystrophy with early macular atrophy, and retinitis pigmentosa is the most common retinal phenotype.

“We’re excited to announce our funding commitment to Opus, a trailblazer in inherited retinal disease therapeutics and a company created based on our mission of ultimately curing blindness caused by retinal degenerative diseases. This investment highlights our steadfast dedication to hastening innovative solutions for those combating inherited retinal diseases,” said Jason Menzo, chief executive officer of the Foundation Fighting Blindness. “In collaboration with Opus, we’re propelling forward promising clinical candidate programs with the potential to revolutionize the lives of those affected by these challenging conditions.”

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 $816 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of blinding retinal diseases including: retinitis pigmentosa, macular degeneration, and Usher syndrome. Visit FightingBlindness.org for more information.

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, including a derisked LCA5 lead program currently in a Phase 1/2 clinical trial, 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:
Gina Mangiaracina
6 Degrees
[email protected]

ABU DHABI, UAE, June 5, 2024 /PRNewswire/ — During the BIO 2024 International Convention, the Department of Health – Abu Dhabi (DoH), the regulator of the healthcare sector in the Emirate, signed a Memorandum of Understanding (MoU) with Opus Genetics, a patient-first, clinical-stage gene therapy company. Through this collaboration, both entities will work together to accelerate the development of novel gene therapies for patients with rare inherited retinal diseases (IRDs) in the UAE.

IRDs are a major cause of vision loss worldwide, and affect approximately 5% of the Arabian Gulf population, severely impacting the lives of patients, their families, and communities. Leveraging the unique data access and capabilities of UAE’s world-leading Emirati Genome Programme and Opus Genetics’ expertise in clinical-stage gene therapy, the parties will explore the potential of conducting and expediting preclinical testing and clinical trials to benefit patients with IRDs.

In the presence of H.E. Mansoor Al Mansoori, Chairman of DoH, and Ben Yerxa, Ph.D., Chief Executive Officer (CEO) of Opus Genetics, the MoU was signed by Dr Asma Al Mannaei, the Executive Director of the Research and Innovation Centre at DoH and Dr. Yerxa.

Dr Asma Ibrahim Al Mannaei, said: “With our uniquely diverse population, established excellence in genomics, and a streamlined 30-day research approval process, Abu Dhabi is well suited to become a focal destination for gene therapy trials. Our collaboration with Opus Genetics reaffirms our commitment to changing lives by seeking and supporting ground-breaking research and the development of novel therapies. By leveraging the Emirate’s advanced

genomics expertise and data-enabled infrastructure, we seek to boost preclinical research and trials that will improve healthcare outcomes today and for generations to come. On our journey to becoming a global hub for health and life science, Abu Dhabi and the Department of Health will continue to act as a both an accelerator and a platform for innovation.”

Ben Yerxa, Ph.D., said: “We are thrilled to announce this collaboration with The Department of Health – Abu Dhabi, which marks a significant step forward in our mission to develop transformative gene therapies for patients with IRDs. Combining UAE’s Emirati Genome Programme with Opus’ expertise in clinical-stage gene therapy development will enable us to explore and expedite the development of novel gene therapies, potentially bringing much-needed treatments to patients with IRDs in Abu Dhabi and globally. We are committed to working together to accelerate preclinical testing and clinical trials, ultimately bringing hope and improved quality of life to those affected by these debilitating conditions.”

Led by the Department of Health – Abu Dhabi (DoH), a high-profile Abu Dhabi delegation headed by His Excellency Mansoor Ibrahim Al Mansoori, Chairman of the DoH, is visiting the United States of America (USA) between May 29^th,2024 and June 5^th,2024 to showcase the Emirate’s partnership opportunities and explore collaboration with leading organisations in Research and Development (R&D), manufacturing and innovation. Kicking-off in Philadelphia, delegates have met with existing and new partners to foster collaboration with leading education research institutions, governmental bodies and health-tech giants. The transnational mission has culminated in San Diego, coinciding with Abu Dhabi’s participation at BIO International Convention 2024 to exhibit the Emirate’s growth and development of its biotechnology industry. The participation has witnessed in-depth discussions, shared insights and expertise as well as explored collaborations in health-tech, life science and innovation.

Highlighting the rich diversity and expertise within Abu Dhabi’s ecosystem, the Delegation comprised of 20 key entities including the Department of Health – Abu Dhabi (DoH), Abu Dhabi Investment Office (ADIO), Abu Dhabi Executive Office (ADEO), G42, Masdar City, Mubadala Investment Company, Khalifa Economic Zones Abu Dhabi Group (KEZAD), Malaffi,

The Medical Office, Ambulatory Healthcare Services, PureHealth, One Health, M42, Technology Innovation Institute (TII), Burjeel Holdings, Khalifa University, Abu Dhabi Holding Company (ADQ), Abu Dhabi Stem Cell Centre (ADSCC) and Etihad Airways.

Photo – https://mma.prnewswire.com/media/2430307/DoH_and_Opus_Genetics.jpg

OPGx-LCA5 will advance to the next highest dose in mid-2024 based on positive safety and efficacy data

OPGx-LCA5 is well-tolerated and demonstrated clear signs of biological activity 

Research Triangle Park, N.C. – March 26, 2024 – Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced that the first cohort has completed dosing in its open-label, dose-escalation Phase 1/2 clinical trial evaluating the subretinal delivery of OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).

Based on positive safety and efficacy data from the first cohort of three adult patients, the Company will advance OPGx-LCA5 into the next highest dose. Opus anticipates initiating the next cohort mid-2024. There are also future plans to expand the study population to include subjects 13 years or older.

“In the first cohort, OPGx-LCA5 has been well-tolerated and demonstrated clear signs of biological activity through 90 days, warranting continued evaluation in the next highest dose,” said principal investigator Tomas S. Aleman, M.D., from the Center for Advanced Retinal and Ocular Therapeutics (CAROT), of the Scheie Eye Institute, Department of Ophthalmology of the Perelman School of Medicine, University of Pennsylvania. “Moreover, early anecdotal and VR challenge test feedback is encouraging and indicates that some of the patients, who have been nearly totally blind all their lives, are now able to see and identify objects for the first time.”

LCA5 is a form of an early-onset retinal degeneration that affects approximately one in 1.7 million people in the U.S. Currently, there are no approved treatments for individuals with LCA5-related vision loss.

“Based on these early clinical data, we’re excited for the potential of OPGx-LCA5 to transform the lives of patients affected by LCA5,” said Ben Yerxa, Ph.D., chief executive officer of Opus. “We’d like to thank the study participants and their families and the incredible efforts of the team at the University of Pennsylvania for reaching this clinical milestone, and we look forward to progressing the trial as we continue to dose escalate.”

For more information on the trial, visit clinicaltrials.gov (NCT05616793).

About OPGx-LCA5

OPGx-LCA5 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 dystrophy. 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-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in nine patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.

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, including a derisked LCA5 lead program currently in a Phase 1/2 clinical trial, 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:
Gina Mangiaracina
6 Degrees

[email protected]

 Data to be presented as part of gene augmentation therapy presentation 

Raleigh, N.C. – November 3, 2023 – Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced that preliminary data from a Phase 1/2 clinical trial evaluating the potential of its gene therapy to address Leber congenital amaurosis (LCA) due to mutations in LCA5 will be presented in a symposium at the American Academy of Ophthalmology annual conference held November 3-6, in San Francisco. The data will be presented as part of a broader presentation by Tomas S. Aleman, MD, the principal investigator of the trial, focused on gene augmentation therapies for the treatment of inherited retinal degenerations.

Details of the presentations are as follows:

Title: Gene Augmentation Therapy for Inherited Retinal Diseases
Session: The Future of Retinal Disease Pharmacological, Stem Cell and Gene Therapy Treatments
Location: In-Person Session SYM47, Moscone Center WEST3018, Live Broadcast, On Demand
Date / time: Sunday, November 5, 2023, 11:30 a.m. – 12:45 p.m. PST
Presenter: Dr. S Aleman, Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine

About OPGx-LCA5

OPGx-LCA5 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 dystrophy. 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-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. Preclinical data, including animal and human iPSC models, have demonstrated preservation of retinal structure and visual function when OPGx-LCA5 was administered prior to peak disease severity.

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:
Ben Yerxa
[email protected]

OPGx-LCA5 is designed to address vision loss due to Leber congenital amaurosis associated with mutations in the LCA5 gene, which causes one of the most severe early-onset retinal dystrophies

Company’s first product candidate from robust pipeline of gene therapies for inherited retinal diseases advances into clinic

Research Triangle Park, N.C. – September 7, 2023 – Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced that the first patient has been dosed in its Phase 1/2, first-in-human clinical trial of OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).

LCA5 is an early-onset retinal degeneration that affects approximately one in 1.7 million people in the U.S. Currently, there are no approved treatments for individuals with LCA5-related vision loss.

“Dosing our first patient establishes Opus as a clinical-stage company and is a point of progress in our mission to advance first-in-class gene therapies for inherited retinal diseases,” said Ben Yerxa, Ph.D., chief executive officer of Opus. “Despite the severe retinal dysfunction in patients with LCA5, preclinical data suggest an opportunity for therapeutic intervention, including retinal structural and functional restoration when OPGx-LCA5 was administered prior to peak disease severity. We look forward to progressing the trial of this potentially transformative therapy for patients affected by LCA5.”

The Phase 1/2, open-label, dose-escalation trial is evaluating the subretinal delivery of OPGx-LCA5 in nine adult patients with LCA5. The objective of the trial is to evaluate the safety and preliminary efficacy of OPGx-LCA5 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.

Once safety in adults has been established and confirmed by the FDA, Opus plans to add a pediatric cohort.

For more information on the trial, visit clinicaltrials.gov (NCT05616793).

About OPGx-LCA5

OPGx-LCA5 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 dystrophy. 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-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. Preclinical data, including animal and human iPSC models, have demonstrated preservation of retinal structure and visual function when OPGx-LCA5 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:
Ben Yerxa
[email protected]

Detectable photoreceptors and signaling activity in patients with RDH12-associated Leber congenital amaurosis support potential of functional restoration by targeted gene therapy

Strong promoter or codon optimization enhanced MERTK gene expression and encourage further evaluation of gene therapy for MERTK-associated retinitis pigmentosa

Data presented at Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2023

Research Triangle Park, N.C. – April 26, 2023 – Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases (IRDs), today announced new data to inform AAV-based gene therapy programs to address two forms of rare inherited retinal diseases (IRDs): Leber congenital amaurosis (LCA) due to mutations in the retinal dehydrogenase (RDH12) gene, and retinitis pigmentosa due to mutations in the proto-oncogene tyrosine-protein kinase MER (MERTK) gene.

In preparation for IND-enabling trials of Opus’ RDH12 gene therapy, a study was conducted to determine the structural-functional relationships in early-onset and severe inherited retinal degeneration (RDH12-EOSRD). Nineteen patients ages 6-21 years with homozygous or compound heterozygous mutations in RDH12 had a comprehensive ophthalmic evaluation with retinal imaging. The data demonstrated that despite severe retinal degeneration and vision impairment, patients displayed retinal regions with clearly detectable photoreceptors and signals distal to the photoreceptor inner segment, which suggest the potential for functional restoration of these areas. The structural-functional relationships in relatively preserved regions suggest vision loss does not result directly from the RDH12 insufficiency, but from resulting outer segment abnormalities and ultimately photoreceptor and retinal pigment epithelium degeneration and loss. The data support the therapeutic potential of gene augmentation to address RDH12-EOSRD.

In a preliminary study completed to support Opus’ internal MERTK development program, four MERTK plasmid constructs were evaluated for their efficiency to induce MERTK expression. The data demonstrated the MERTK plasmid design can be optimized to upregulate MERTK protein expression and augment MERTK-dependent phagocytosis. The data provide guidance for future preclinical studies and encourage further evaluation of the efficacy of a MERTK gene therapy.

“Patients with inherited retinal diseases due to mutations of the RDH12 or MERTK genes experience progressive vision loss and eventual blindness, and are currently without treatment options,” said Ash Jayagopal, Ph.D., Chief Scientific Officer of Opus. “Patients with RDH12 gene mutations have severe deterioration of the central retina; however, identification of structural-functional relationships in areas of preserved photoreceptors suggest a therapeutic window when functional restoration is possible. In addition, the ability to upregulate MERTK expression in patients with MERTK-associated retinitis pigmentosa suggests the possibility of delivering a functional MERTK gene to retinal cells to produce the normal protein. There is significant need for novel treatment options and these data further support the potential for targeted gene therapy to transform the lives of people living with inherited retinal diseases.”

The data were presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2023 in New Orleans.

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
[email protected]

Oral presentation on BEST1 clinical translation to be delivered during the 8th Annual Retinal Cell and Gene Therapy Innovation Summit

Two posters related to RDH12 and MERTK programs to be presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2023

Research Triangle Park, N.C. – April 18, 2023 – Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced leadership and collaborators will present data supporting multiple programs in its pipeline of gene therapies for inherited retinal diseases at upcoming scientific meetings.

Ash Jayagopal, Ph.D., Chief Scientific Officer of Opus, will deliver an oral presentation, titled, “Development of Endpoints for Clinical Translation of BEST1 Gene Therapy,” at the 8^th Annual Retinal Cell and Gene Therapy Innovation Summit sponsored by the Foundation Fighting Blindness and the Oregon Health and Science University (OHSU) Casey Eye Institute, on Friday, April 21, 2023, at 11 a.m. CDT. 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-2023-509.

Preclinical research and advances related to retinal degenerations caused by mutations in the RDH12 and MERTK genes will be presented in poster sessions at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2023, being held April 23-27, 2023, in New Orleans. The ARVO Annual Meeting is the premiere gathering for eye and vision scientists to share the latest research findings and collaborate on innovative solutions.

Details of the ARVO presentations are as follows:

Title: Structural and Functional Relationships in RDH12-Associated Early-Onset Severe Inherited Retinal Degeneration
Session: Inherited Retinal Degeneration
Poster Number: B0197
Location: Exhibit Hall, New Orleans Ernest N. Morial Convention Center
Date / time: Monday, April 24, 2023, 11:30 a.m.—1:15 p.m. CDT
Presenter: Erin O’Neil, M.D., Children’s Hospital of Philadelphia

Title: Evaluation of MERTK Gene Therapy Constructs in Cell Culture Models
Session: Retinal Pigment Epithelium
Poster Number: C0099
Location: Exhibit Hall, New Orleans Ernest N. Morial Convention Center
Date / time: Tuesday, April 25, 2023, 11:45 a.m.—1:30 p.m. CDT
Presenter: Elisebeth Torretti, presenting author

For more information on the ARVO Annual Meeting, visit https://www.arvo.org/annual-meeting/.

Dr. Jayagopal has also been selected by the ARVO Annual Meeting Program Committee to organize and moderate a Special Interest Group (SIG) session, titled “Development of Endpoints for Successful Translation of Inherited Retinal Disease Therapies,” on Thursday, Sept. 28, 2023, at 6 p.m. EDT. The session will feature a global panel of experts in retinal diseases and will be held virtually. For more information, visit https://www.arvo.org/annual-meeting/program/special-interest-group-meetings/#endpoints.

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
[email protected]

Research Triangle Park, N.C. – Jan. 3, 2023 – Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced that Ben Yerxa, Ph.D., Chief Executive Officer of Opus, will present a company overview and provide an update on Opus’ gene therapy programs for inherited retinal diseases at the Biotech Showcase™ 2023 in San Francisco.

Details of the presentation are as follows:

Date:              Monday, January 9, 2023
Time:             10:15 a.m. PST
Location:      Hilton San Francisco Union Square, 333 O’Farrell Street, San Francisco, CA 94102
Track:            Franciscan A (Ballroom Level)

Biotech Showcase, produced by Demy-Colton and EBD Group, is an investor conference focused on driving advances in therapeutic development by providing a sophisticated networking platform for executives and investors that fosters investment and partnership opportunities. The conference takes place each year during the course of one of the industry’s largest gatherings and busiest weeks. For more information or to register, visit www.biotechshowcase.com.

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

[email protected]

Opus to advance preclinical development programs for BEST1– and RHO-related retinal diseases

Deal expands Opus’ addressable patient population for its novel treatments for rare inherited retinal diseases

Research Triangle Park, N.C. – December 28, 2022 – Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced it has acquired the rights to two preclinical-stage AAV-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio. Opus will develop the novel gene therapy candidates to address bestrophin-1 (BEST1)-related inherited retinal diseases and rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), respectively.

BEST1-related IRDs are estimated to affect approximately one in 69,000 people, or nearly 5,000 people, in the United States. The BEST1 gene therapy is designed to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells to produce bestrophin-1 protein and normalize homeostasis between the photoreceptors and retinal pigment epithelial cells. RHO-adRP is one of the most common IRDs, estimated to affect approximately one in 51,000 people, or more than 6,000 people, in the United States alone.

“The addition of these innovative BEST1 and RHO-adRP programs significantly increases the patient population that could benefit from Opus therapies and complements our existing pipeline of gene therapies for inherited retinal diseases,” said Ben Yerxa, PhD, Chief Executive Officer of Opus. “Opus is building an engine for addressing multiple IRDs, and deals like this one help us achieve our desire for sustainable growth of our infrastructure, operations, scientific expertise and ultimately our patient impact, with the goal of at least one IND per year.”

Opus anticipates completing additional IND-enabling studies and filing an IND for BEST1 in the second half of 2023.

As part of the deal, Opus will assume responsibility for the global research, development and commercialization of BEST1 and RHO-adRP programs. In exchange, Iveric received an upfront payment of $500,000 and high single-digit percentage ownership of Opus. Iveric is also eligible to receive development and regulatory milestone payments, sales milestone payments, and a low single-digit earnout on net sales of the products. Iveric retains certain rights with respect to the potential future commercialization of gene therapy products for BEST1 and/or RHO-adRP under certain circumstances.

“Affecting over 28,000 people across the U.S., EU and UK, BEST1 and RHO-adRP represent a significant portion of all inherited retinal diseases and an urgent unmet need for effective treatment. IRDs are ideal targets for genetic therapies to stop the retinal degeneration and improve the lives of patients living with severe vision loss or blindness,” said Bart P Leroy, MD, PhD, head of the Department of Ophthalmology, professor of Ophthalmology and member of the Center for Medical Genetics at Ghent University and Ghent University Hospital in Belgium; and attending physician in the Division of Ophthalmology and The Raymond G. Perelman Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia.

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

[email protected]