OCUP Ocuphire Pharma Inc

Opus Genetics Featured on Good Morning America Spotlighting Breakthrough Gene Therapy Restoring Sight in Patients with Inherited Blindness

Opus Genetics Featured on Good Morning America Spotlighting Breakthrough Gene Therapy Restoring Sight in Patients with Inherited Blindness

RESEARCH TRIANGLE PARK, N.C., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Opus Genetics (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs), announced today that its pioneering work was featured on (ABC News) in honor of World Blindness Awareness Month.

The national feature highlighted the story of Lindsey Rambo, the second participant in Opus Genetics’ ongoing Phase 1/2 trial of gene therapy OPGx-LCA5, aimed at restoring vision for people living with a rare genetic form of blindness caused by mutations in the LCA5 gene, which encodes lebercilin, a protein essential for photoreceptor structure.

The GMA segment, titled ,” explores how Opus Genetics’ investigational gene therapy is helping individuals born blind regain partial sight, an unprecedented step forward for the IRD community. The story brings national attention to the life-changing potential of gene therapy and the scientists and patients driving progress in the fight against inherited blindness.

“We are deeply honored to have Opus Genetics and Lindsey’s story featured on Good Morning America,” said George Magrath, M.D., Chief Executive Officer of Opus Genetics. “For the millions affected by inherited retinal diseases, these advancements represent hope. Our mission has always been to strive to bring the most promising gene therapies from the lab to patients as quickly and safely as possible.”

“Taking part in this study has given me hope not just for myself, but for people living with inherited blindness,” said Lindsey Rambo, the second participant in the Opus Genetics LCA5 clinical trial. “Being featured on Good Morning America and sharing my experience means helping others understand that research like this can potentially change lives.”

By delivering a functional copy of the defective gene directly to retinal cells, Opus Genetics’ approach aims to restore the function of light-sensing photoreceptors and, ultimately, vision itself.

The gene therapy featured on Good Morning America was made possible by Opus Genetics, with support from the Retinal Degeneration (RD) Fund and the Foundation Fighting Blindness.

The GMA feature coincides with Opus Genetics’ growing clinical and community momentum, including multiple ongoing trials for LCA5 and BEST1 gene therapies, a newly activated site at the Retina Foundation of the Southwest.

Watch the full Good Morning America feature here:

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, expectations regarding us, our business prospects and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, our Quarterly Reports on Form 10-Q for the quarters ended March 31, 2025 and June 30, 2025, and our other filings with the U.S. Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. These forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “strive”, “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.

About Opus Genetics

Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders. Opus Genetics’ pipeline features AAV-based gene therapies targeting inherited retinal diseases including Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa. Its lead gene therapy candidates are OPGx-LCA5, which is in an ongoing Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1, a gene therapy targeting BEST1-related retinal degeneration. Opus Genetics is also advancing Phentolamine Ophthalmic Solution 0.75%, a partnered therapy currently approved in one indication and being studied in two Phase 3 programs for presbyopia and reduced low light vision and nighttime visual disturbances. Opus Genetics is based in Research Triangle Park, NC. For more information, please visit .

Contacts:

Investors

Jenny Kobin

Remy Bernarda

IR Advisory Solutions

Media

Kimberly Ha

KKH Advisors

917-291-5744

Source: Opus Genetics, Inc.



EN
03/11/2025

Underlying

To request access to management, click here to engage with our
partner Phoenix-IR's CorporateAccessNetwork.com

Reports on Ocuphire Pharma Inc

 PRESS RELEASE

Opus Genetics to Present at Upcoming Investor Conferences in March 202...

Opus Genetics to Present at Upcoming Investor Conferences in March 2026 RESEARCH TRIANGLE PARK, N.C., March 03, 2026 (GLOBE NEWSWIRE) -- . (Nasdaq: IRD) (“Opus Genetics” or the “Company”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), announced today that management will present at the following investor conferences. Leerink Global Healthcare Conference Date: Tuesday, March 10, 2026Time: 3:00 p.m. ETLocation: Miami, FL Citizens Life Sciences Conference Date: Wednesday, Ma...

Wedbush Research
  • Wedbush Research
W WAYFAIR INC.
UBI UBISOFT ENTERTAINMENT SA
XNCR XENCOR INC.
XENE XENON PHARMACEUTICALS INC
WDC WESTERN DIGITAL CORPORATION
VYGR VOYAGER THERAPEUTICS INC.
TTWO TAKE-TWO INTERACTIVE SOFTWARE INC.
TRIP TRIPADVISOR INC.
SRPT SAREPTA THERAPEUTICS INC.
SIMON SILICON MOTION TECHNOLOGY CORPORATION SPONSORED ADR
RARE ULTRAGENYX PHARMACEUTICAL INC.
PSTG PURE STORAGE INC. CLASS A
NVDA NVIDIA CORPORATION
NVCR NOVOCURE LTD.
NTAP NETAPP INC.
NBIX NEUROCRINE BIOSCIENCES INC.
MU MICRON TECHNOLOGY INC.
MELI MERCADOLIBRE INC.
KURA KURA ONCOLOGY INC.
INTC INTEL CORPORATION
GOOGL ALPHABET INC. CLASS A
GERN GERON CORP.
FB FACEBOOK INC. CLASS A
FATE FATE THERAPEUTICS INC
EXPE EXPEDIA GROUP INC.
ETSY ETSY INC.
EBAY EBAY INC.
CTMX CYTOMX THERAPEUTICS INC.
BMRN BIOMARIN PHARMACEUTICAL INC.
BIIB BIOGEN INC.
BCRX BIOCRYST PHARMACEUTICALS INC.
AXTI AXT INC.
ASND ASCENDIS PHARMA A/S SPONSORED ADR
ARDX ARDELYX INC
CDR CD PROJEKT S.A.
FRX_CN FENNEC PHARMACEUTICALS
7974 NINTENDO CO. LTD.
2303 UNITED MICROELECTRONICS CORP.
EA ELECTRONIC ARTS INC.
PTGX PROTAGONIST THERAPEUTICS
ANAB ANAPTYSBIO INC.
ARGX ARGENX ADS
OVID OVID THERAPEUTICS
ISR ISORAY
SE SEA ADS
AMZN AMAZON.COM INC.
AMD ADVANCED MICRO DEVICES INC.
AAPL APPLE INC.
MSFT MICROSOFT CORPORATION
APLS APELLIS PHARMACEUTICALS
BKNG BOOKING HOLDINGS INC.
DNLI DENALI THERAPEUTICS INC
RCUS ARCUS BIOSCIENCES
SRRK SCHOLAR ROCK HOLDING CORPORATION
KNSA KINIKSA PHARMACEUTICALS
REPL REPLIMUNE GROUP
ARVN ARVINAS HOLDING
STRO SUTRO BIOPHARMA
LYFT LYFT
PINS PINTEREST INC. CLASS A
UBER UBER TECHNOLOGIES INC.
CHWY CHEWY INC. CLASS A
STOK STOKE THERAPEUTICS
SMCI SUPER MICRO COMPUTER
IDYA INC.
PASG IDEAYA BIOSCIENCES
BDTX PASSAGE BIO
ACET BLACK DIAMOND THERAPEUTICS
KROS ADICET BIO INC
ZNTL KEROS THERAPEUTICS
BEAM ZENTALIS PHARMACEUTICALS
LRMR BEAM THERAPEUTICS
OCUP LARIMAR THERAPEUTICS INC
PRAX OCUPHIRE PHARMA INC
COGT PRAXIS PRECISION MEDICINES
U COGENT BIOSCIENCES INC
TVTX UNITY SOFTWARE
DASH TRAVERE THERAPEUTICS INC
ABNB DOORDASH
VRDN AIRBNB INC. CLASS A
PLTK VIRIDIAN THERAPEUTICS INC
NUVB PLAYTIKA HOLDING
RBLX NUVATION BIO INC (A)
DAWN ROBLOX
TNGX DAY ONE BIOPHARMACEUTICALS INC
STX TANGO THERAPEUTICS
HOWL INC
APP SEAGATE TECHNOLOGY HLDGS PLC
TTD WEREWOLF THERAPEUTICS INC
QUBT APPLOVIN CORP
GFS THE TRADE DESK
GMTX INC.
TYRA QUANTUM COMPUTING INC
EWTX GLOBALFOUNDRIES INC
SLDB GEMINI THERAPEUTICS INC
DDI TYRA BIOSCIENCES INC
APGE EDGEWISE THERAPEUTICS INC
EMBRAC B SOLID BIOSCIENCES INC
DNTH DOUBLEDOWN INTERACTIVE CO LTD
CART APOGEE THERAPEUTICS INC
NATL EMBRACER GROUP AB
CGON DIANTHUS THERAPEUTICS INC
ORKA MAPLEBEAR INC.
BCAX NCR ATLEOS CORPORATION
ZBIO CG ONCOLOGY INC
TLX ORUKA THERAPEUTICS INC
MAZE BICARA THERAPEUTICS INC.
JBIO ZENAS BIOPHARMA INC.
TELIX PHARMACEUTICALS LIMITED
MAZE THERAPEUTICS INC
JADE BIOSCIENCES INC
 PRESS RELEASE

Opus Genetics Announces Initial Clinical Data from Phase 1/2 OPGx-BEST...

Opus Genetics Announces Initial Clinical Data from Phase 1/2 OPGx-BEST1 Gene Therapy Study at the Macula Society Annual Meeting Sentinel participant showed OPGx-BEST1 was well tolerated with no ocular inflammation, treatment-related adverse events, or dose-limiting toxicities at three months Early signals of functional and structural improvement observed at one month and three months12-letter BCVA gain and 23% CST reduction observed in the treated eye at three monthsFull cohort data expected in mid-year 2026 RESEARCH TRIANGLE PARK, N.C., Feb. 27, 2026 (GLOBE NEWSWIRE) -- . (Nasdaq: IRD) ...

Yun Zhong
  • Yun Zhong

Initial BEST1 Data Compelling; Fast Pivotal Entry At Dose 1 A Big Poss...

Figure 1 - BCVA Improvement Reached 12 Letters At Month 3 after OPGx-BEST1 TreatmentSource: Company presentationFigure 2 - OPGx-BEST1 Treatment Leads to Consistent Improvement in CST as wellSource: Company presentationFigure 3 - Adaptive Design of the Phase 1/2 Study of OPGx-BEST1Source: company pr

ResearchPool Subscriptions

Get the most out of your insights

Get in touch