Press Releases

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

gmangiaracina@6degreesPR.com

 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
byerxa@opusgtx.com

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
byerxa@opusgtx.com

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
handerson@6degreespr.com

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
handerson@6degreespr.com

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

handerson@6degreespr.com

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

handerson@6degreespr.com

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

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

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