OIS RETINA PRESENTING COMPANIES
Daniel Chao, MD, PhD, Associate Vice President, Head of Ophthalmology - ADARx Pharmaceuticals
We are a private clinical stage biotech company based in San Diego developing next generation RNA medicines for a number of therapeutic areas. We have developed a best in class small interfering RNAs (siRNAs) to target the liver, allowing us to reliably downregulate liver proteins in the serum over 90% reduction from baseline, and lasting over 3 months with a single subcutaneous injection. We have 3 molecules currently in the clinic. Our lead program is a subcutaneously delivered siRNA against Complement factor B, which we are planning a Phase 2b study in GA starting later this year, as well as trials in Paroxysmal nocturnal hemoglobinuria, and IgA Nephropathy to start this year. In addition, we are developing technologies to allow siRNAs to access extrahepatic tissues such as the CNS, muscle, fat, and the eye. Ophthalmology is one of 4 strategic focus areas of the company, with a focus on high prevalence retinal diseases (e.g. GA, retinal vascular diseases), utilizing both systemic subcutaneous as well as local IVT ROA. We have raised over $350 million from top tier investors.
Laurent Fischer, President & CEO - Adverum
Adverum Biotechnologies is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing functional cures to restore vision and prevent blindness. Leveraging the research capabilities of its proprietary intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians’ offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. By overcoming the challenges associated with current treatment paradigms for these debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe.
Seemi Khan, MD, MPH, MBA, Chief Medical Officer - Alkeus Pharmaceuticals
Alkeus is a clinical-stage biopharma company located in Cambridge, Massachusetts, dedicated to preserving the sight of individuals impacted by retinal diseases. Our initial focus is Stargardt disease and our investigational therapy, gildeuretinol, is a once daily oral therapy designed to reduce the dimerization of vitamin A without modulating the visual cycle.
Anders Haegerstrand, MD, PhD, CEO - Annexin Pharmaceuticals
Annexin Pharmaceuticals is a biotechnology company in clinical stage and a global leader in the Annexin A5 area. The company is listed at First North, Stockholm, and is headquartered in Stockholm, Sweden.
Carl Erickson, CEO - C5 Therapeutics
C5 Therapeutics is developing a pipeline of next generation intraocular complement inhibitors for the treatment of GA. By analogy to anti-VEGF for wet AMD, the company strategy is to leapfrog from the first generation approved therapeutics ("IZERVAY / SYFOVRE" = "Macugen"... modest benefit/risk profile, frequent dosing) directly to 3rd generation therapeutics ("C5Tx-001" = "VABYSMO" ... significantly superior benefit / risk, q16w dosing). Lead candidate C5Tx-001 is ready to begin IND enabling studies and the biomarker technology has clinical proof of concept across multiple data sets.
Jay Duker, MD, President & CEO - EyePoint Pharmaceuticals
EyePoint (Nasdaq: EYPT) is a clinical-stage biopharmaceutical company committed to developing and commercializing innovative therapeutics to help improve the lives of patients with serious retinal diseases. The Company's pipeline leverages its proprietary bioerodible Durasert E™ technology for sustained intraocular drug delivery. The Company’s lead product candidate, DURAVYU™ is an investigational sustained delivery treatment for VEGF-mediated retinal diseases combining vorolanib, a selective and patent-protected tyrosine kinase inhibitor with bioerodible Durasert E™. Supported by robust safety and efficacy data to date, DURAVYU is presently in Phase 3 global, pivotal clinical trials for wet age-related macular degeneration (wet AMD), the leading cause of vision loss among people 50 years of age and older in the United States and recently completed a Phase 2 clinical trial in diabetic macular edema (DME).
Max Cotler, President - GelMEDIX
GelMEDIX is a preclinical ophthalmology company, developing a retinal pigment epithelial (RPE) cell therapy to treat Geographic Atrophy, the leading cause of blindness in American adults. The first and recently approved treatments have marginal clinical benefit, and current retinal cell therapies in clinical development have shown encouraging early benefits, but suffer from fundamental procedural limitations potentially limiting their utility. GelMEDIX's proprietary, in situ forming hydrogel technology provides an injectable scaffold that protects cells during injection and provides a framework for tissue regeneration while localizing them to the target site, overcoming the limitations of current approaches. GelMEDIX has demonstrated compelling pre-clinical evidence of overcoming the delivery challenges with potential to regenerate the retina in both porcine and rabbit models, including improved OCT and ERG compared to current approaches in the clinic. GMP process development will be complete in the second half of 2025 with IND-enabling studies commencing thereafter.
Thomas Ciulla, MD, MBA, President and CEO - Ikarovec
Ikarovec is a leader in ophthalmology innovation, with a sophisticated field-changing technology for the long-term relief of prevalent eye diseases. The company’s flexible proprietary bicistronic gene therapy generates multiple synergistic therapeutic proteins where they are needed in the eye, bringing a differentiated approach to treating a range of large and growing ophthalmic disorders.
Its lead product, IKAR-001, (previously known as IKC159V) will proceed to a geographic atrophy clinical trial supported by studies showing preservation of cells critical for vision. GA leads to significant vision loss, affecting 5 million people worldwide. Current treatment options (only approved in the US) require regular injections directly into the eye. Ikarovec’s novel bicistronic gene therapy leads to the expression of two therapeutically relevant proteins, targeting multiple causes of the disease with a single injection which is expected to be disease modifying. Follow-on programmes are targeting wet age-related macular degeneration and other chronic poorly managed ophthalmic indications.
Ikarovec is directed by an international Board and management team with industry-leading ophthalmology and gene therapy expertise. This experience comes via Astellas, EyeBio, OXB (Oxford Biomedica), Spark Therapeutics and others. Ikarovec, which is based at Norwich Research Park, is backed by LifeArc Ventures, Parkwalk Advisors and UKI2S. More information at www.ikarovec.com and on LinkedIn.
Brian Levy, OD, CEO - InflammX Therapeutics
InflammX Therapeutics is a clinical-stage company developing a small molecule , orally dosed drug (Xiflam) to treat intermediate AMD (iAMD) and Diabetic Retinopathy (DR) /Chronic Kidney Disease (CKD). iAMD is the early dry form of AMD, at which stage patients still have good vision. iAMD represents +/- 80% of patients with AMD for which there is no approved pharmacological treatment. iAMD progresses to geographic atrophy (GA) and significant vision loss.
Xiflam is a tablet taken orally QD to treat and inhibit the progression of iAMD to GA and the associated loss of vision.by inhibiting the disease related inflammation as a result of an activated, pathologically perpetuated innate immune system, the NLRP3 Inflammasome.
By combining our oral drug Xiflam, with a cutting-edge and novel, OCT imaging strategy to identify high risk patients at the early disease stage, the FDA accepted our protocol to proceed with our Phase 2B clinical study. The drug target is the pathological Connexin43 (Cx43) hemichannel protein. InflammX technology has been developed to target the perpetuated source of inflammation (NLRP3 inflammasome) for patients with iAMD and diabetic eye disease. This provides the patient with the advantage of a well-tolerated oral tablet, with a good safety profile and once a day dosing for bilateral treatment of a bilateral disease. This presents a disruptive, differentiated, future state, to treat patients with an early diagnosis of the dry form of AMD without the need for invasive injections. The treatment has the potential of preventing patients with a diagnosis of iAMD and good vision, from progressing to GA and lost vision.
The advantage of an orally dosed drug for iAMD, and the lack of need for intravitreal injections (administered primarily by retinal specialists), provides the opportunity for expansion of the provider to include general ophthalmologists and optometrists, expanding the therapeutic potential to a significantly larger patient base.
Clark E. Tedford, PhD, President & CEO - LumiThera
LumiThera, Inc. is a medical device company commercializing a multi-wavelength instrument for the ophthalmologist’s office that improves vision and slows the progression of vision loss in patients with dry age-related macular degeneration (AMD). The Company’s focus to date has been on the treatment of patients with early/intermediate dry AMD. The Valeda Light Delivery System is the first FDA approved treatment that improves vision in dry AMD patients. The Company has significant expertise in the use of Photobiomodulation (PBM), which is the use of light therapy to stimulate cells using light emitting diodes (LEDs) of selected wavelengths.
Sam Barone, MD, Chief Medical Officer - Nanoscope Therapeutics
Nanoscope Therapeutics is developing mutation-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases for which no treatment currently exists. Following positive end-of-study results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), the FDA has granted Nanoscope a rolling BLA submission for MCO-010 to treat RP in 2025. The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and plans to initiate a Phase 3 registrational trial, also in 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical programs include an IND-ready non-viral laser-delivered MCO-020 asset for GA, as well as an AAV asset for Leber Congenital Amaurosis, in IND-enabling studies.
Amir Yazdanyar, MD, PhD - NomoCan Pharmaceutical
NomoCan Pharmaceuticals is a privately held biotechnology company based in New York, specializing in the development of first-in-class monoclonal antibody (mAB) for targeting spliced variants of MDM2. Our therapeutic agents have broad implications in oncology (ocular and non-ocular) and other retinal diseases such as proliferative vitreoretinopathy. NomoCan is in the process of humanizing its mouse-derived monoclonal antibody, NMC-001, in preparation for preclinical data submission to the FDA. Upon approval of the IND application, the company plans to conduct Phase I-III human clinical trials in collaboration with larger oncology pharmaceutical companies.
Sanjay Nayak, MBBS, PhD, Chief Strategy Officer - Ocular Therapeutix
Ocular Therapeutix, Inc. is a fully-integrated biopharmaceutical company committed to redefining the retina experience. AXPAXLI™ (also known as OTX-TKI), Ocular’s investigational product candidate for retinal disease, is an axitinib intravitreal hydrogel based on its ELUTYX™ proprietary bioresorbable hydrogel-based formulation technology. AXPAXLI is currently in Phase 3 clinical trials for wet age-related macular degeneration (wet AMD).
Ocular’s pipeline also leverages the ELUTYX technology in its commercial product DEXTENZA®, an FDA-approved corticosteroid for the treatment of ocular inflammation and pain following ophthalmic surgery in adults and pediatric patients and ocular itching associated with allergic conjunctivitis in adults and pediatric patients aged two years or older, and in its investigational product candidate PAXTRAVA™ (also known as OTX-TIC), which is a travoprost intracameral hydrogel that is currently in a Phase 2 clinical trial for the treatment of open-angle glaucoma or ocular hypertension.
Riad Sherif, MD, CEO - Oculis
Oculis is a global biopharmaceutical company (Nasdaq: OCS; XICE: OCS) purposefully driven to save sight and improve eye care. Oculis’ highly differentiated pipeline of multiple innovative product candidates in development includes: OCS-01, a topical eye drop candidate for diabetic macular edema (DME); Privosegtor (OCS-05), a neuroprotective candidate for acute optic neuritis with potentially broad clinical applications in other neuro-ophthalmic diseases and Licaminlimab (OCS-02), a topical biologic anti-TNFα eye drop candidate for dry eye disease (DED). Headquartered in Switzerland with operations in the U.S. and Iceland, Oculis is led by an experienced management team with a successful track record and supported by leading international healthcare investors.
Houman Hemmati, MD, PhD, Co-Founder & CMO - Optigo
Optigo Biotherapeutics is a ophthalmology biotech company developing a proprietary platform designed to extend the half-life of intravitreal biologics by anchoring them to specific proteins in the eye. This approach enables sustained therapeutic presence and improved durability of effect, significantly reducing the frequency of intravitreal injections. Built on this platform, Optigo’s lead asset is a next-generation anti-VEGF therapy targeting the underlying drivers of blinding retinal diseases. By enhancing treatment longevity and addressing limitations of current standard-of-care therapies, Optigo aims to ease the burden on patients and caregivers while delivering meaningful, long-term improvements in vision outcomes. The platform’s modular design also offers potential applicability to other intravitreal biologics requiring extended half-life. To date the company has raised over $10 M in financing and is now raising $30 to move its lead asset into IND and Phase 1-2a Human clinical trials.
Sally Tucker, PhD, SVP, Head of Clinical Development - Opus Genetics
The company is a clinical-stage ophthalmic biotechnology company developing therapies to treat patients with inherited retinal diseases (IRDs) and other ophthalmic disorders. The pipeline includes adeno-associated virus (AAV)-based gene therapies that address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. The company’s most advanced gene therapy program is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein and is currently being evaluated in a Phase 1/2 open-label trial, with encouraging early data. The pipeline also includes Phentolamine Ophthalmic Solution 0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist to reduce pupil size, and APX3330, a novel small-molecule inhibitor of Ref-1 to slow the progression of non-proliferative diabetic retinopathy (DR). Phentolamine Ophthalmic Solution 0.75% is currently being evaluated in Phase 3 trials for presbyopia and reduced dim (mesopic) light vision following keratorefractive surgery. We have reached agreement with the FDA on a SPA for a Phase 3 trial to evaluate oral APX3330 for the treatment of DR.
TBD - Outlook Therapeutics
Outlook Therapeutics is a biopharmaceutical company working to develop and launch ONS-5010/ LYTENAVA™ (bevacizumab-vikg) as the first FDA-approved ophthalmic formulation of bevacizumab for use in retinal indications, including wet AMD, DME and BRVO. If ONS-5010 ophthalmic bevacizumab is approved, Outlook Therapeutics expects to commercialize it as the first and only FDA-approved ophthalmic formulation of bevacizumab for use in treating retinal diseases in the United States, United Kingdom, Europe, Japan and other markets. Outlook Therapeutics expects to submit ONS-5010 ophthalmic bevacizumab to the U.S. FDA as a BLA under the PHSA 351(a) regulatory pathway.
James A. Stefater MD, PhD, President & Co-Founder - Pykus Therapeutics
Pykus Therapeutics, Inc., based in Cambridge, Massachusetts, is a clinical-stage medical technology company committed to advancing care for retinal surgical diseases. Using a proprietary, patented hydrogel platform originally developed at Mass Eye and Ear/Harvard Medical School, Pykus is focused on delivering transformative surgical solutions that improve outcomes and enhance the patient experience.
Nearly two million vitrectomies are performed each year globally, with retinal detachment being one of the most common indications. Current standard-of-care treatments rely on intraocular gas or silicone oil to tamponade the retina, requiring patients to maintain face-down positioning postoperatively and endure blurred vision for weeks. These methods often fall short, with high failure rates and the potential for permanent vision loss.
Pykus’ lead product candidate, PYK-2101, is a patented, first-in-class biodegradable retinal sealant designed to directly seal retinal breaks without obscuring vision. The platform eliminates the need for uncomfortable face-down positioning and enables faster visual recovery while potentially reducing complications such as cataract progression, elevated intraocular pressure, and proliferative vitreoretinopathy.
Pykus is currently conducting a prospective, multicenter, open-label pilot study (PYK-2101-RD001) in Australia to evaluate the safety and efficacy of PYK-2101 in 11 participants undergoing pars plana vitrectomy for retinal detachment. Key outcome measures include anatomical attachment rate, speed of visual acuity recovery, intraocular pressure changes, and adverse events. The study is being conducted at leading vitreoretinal centers, with top-line results expected in Q3 2025.
Peter Francis, MD, PhD, Chief Medical and Scientific Officer - Ray Therapeutics
Ray Therapeutics (RayTx) is a clinical-stage biotechnology company pioneering optogenetic gene therapies for blinding retinal diseases. RayTx aims to provide best-in-class and first-in-class, mutation-agnostic, vision restoration to patients without available treatment options. Founded in 2021 and headquartered in Berkeley, CA, the company raised $100 million in a Series A financing led by Novo Holdings in May 2023.
RayTx's lead asset, RTx-015, delivers a highly bioengineered light-sensitive protein, is currently in phase 1 / 2 clinical trials, initially for retinitis pigmentosa (RP) and choroideremia. RTx-021, the second asset, is expected to enter clinical trials in 2025 for Stargardt macular dystrophy (STGD) and geographic atrophy age related macular degeneration (GA AMD).
Ram Palanki, PharmD, CCO - REGENXBIO
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people.
Michael O'Rourke, CEO - Re-Vana Therapeutics
Re-Vana Therapeutics: Advancing Sustained Release Biologics for Retinal Disease.
Re-Vana aims to be the world's first company to deliver an In Clinic Sustained Release (SR) Biologic in humans treating retina diseases.
Re-Vana is redefining sustained ocular drug delivery with a disruptive platform designed to overcome the most pressing challenge in retinal —reducing the high treatment burden of frequent biologic injections. Current anti-VEGF biologics remain the standard of care for conditions like wet AMD, DME, and RVO, but require repeated injections that impact adherence and outcomes.
Re-Vana’s mission is to extend therapeutic duration beyond six months, directly addressing this critical unmet need.
Our proprietary photo-crosslinked biodegradable hydrogel platform represents a breakthrough in sustained delivery. Unlike PLGA-based systems, which compromise protein stability, our technology protects and preserves biologic activity while enabling tunable, customizable release profiles. These implants are biodegradable, well tolerated, and can be administered without surgical intervention—paving the way for broader clinical use.
Re-Vana’s progress is supported by strong strategic momentum. Our pipeline includes sustained-release formulations of aflibercept and next-generation bi-specifics, building on experience with nine biologics and five small molecules. We’ve established collaborations with top-tier pharmaceutical partners and validated our platform with robust preclinical data, including favorable tolerability and efficacy profiles across multiple models.
Our team includes global leaders in retinal R&D, clinical development, and commercialization, guided by internationally recognized retina advisors. Backed by $18M in funding from leading ophthalmic VCs and corporate pharma, Re-Vana is now advancing a $40M Series B to propel our clinical programs forward.
Re-Vana stands apart with a clinically viable, customizable, scalable platform that unlocks the full potential of sustained-release biologics in retinal disease. Our strategic vision is to build
a full Continuum of SR biologic products in Retina to become the leader in long-acting biologic drug delivery.
Ramanath Bhandari, MD, CEO - RevOpsis Therapeutics
RevOpsis Therapeutics is a next-generation biopharmaceutical company that is trailblazing a new era in multispecific antibody discovery and development to unlock transformative treatments for complex disease.
Our proprietary Rev-Mod Platform uses a modular plug-and-play system to streamline drug discovery and development. The Rev-Mod Platform features a library of fully human and fully compatible antibody components that are ready to be assembled into functional Multi-Mod biologics. Using this platform, we can select and integrate multiple antibody components into a single, multispecific therapeutic molecule that binds targets implicated in known, clinically validated disease pathways.
Our lead asset from this platform, RO-104, is a first-in-class trispecific biologic. RO-104 targets 3 clinically validated pathways that are implicated in retinal vascular diseases, such as neovascular age-related macular degeneration (nAMD). Whereas current anti-VEGF therapies used to treat nAMD predominantly target only the VEGF-A pathway, RO-104 builds on a body of literature that suggests that pan-VEGF and Ang-2 inhibition can improve efficacy and extend treatment durability. RO-104 has the potential to offer these benefits with a single therapeutic molecule, as preclinical studies indicate high affinity and superior efficacy to Vabysmo and Eylea in a choroidal neovascularization model, and ideal drug-like properties.
Chiho Fukiage, Head of SLOS (Senju Laboratory of Ocular Science) - Senju Pharmaceutical Co
Senju is a privately held, research-based Japanese pharmaceutical company that develops, manufactures, and commercializes a variety of innovative products, focusing on the field of ophthalmology to improve the health and quality of vision for people worldwide through the company’s products.
The characters that comprise Senju’s company name in Japanese language are “Sen” and “Ju“, or “thousand” and “bringing happiness”. Senju’s mission is to improve the joy of living of people associated with the company through the development of original pharmaceutical products.
Emmett Cunningham, MD, PhD, Advisor - Uneedle
Uneedle, based in the Netherlands, specializes in designing and manufacturing advanced silicon microneedles for intradermal and suprachoroidal injections. Their proprietary technology, CE marked and FDA approved, enables precise and easy-to-use solutions for medical applications such as cancer vaccines and ophthalmic therapies. Uneedle's device platform facilitates efficient drug delivery with improved patient compliance. The company operates a GMP-compliant manufacturing site and is ISO13485 certified, partnering with pharmaceutical companies to enhance drug administration methods.
Wesley M. Jackson, President & CSO - Valitor
Valitor is engineering a new generation of long-acting ophthalmic medicines using its multivalent polymer (MVP) platform. The MVP platform is based on proprietary conjugation chemistry for covalent attachment of multiple copies of drug moieties to hyaluronic acid (HA). This technology enables independent control of multiple drug attributes, and conjugation to HA is a favorable strategy for driving ophthalmic drug durability due to its biocompatibility and demonstrated safety in ocular use. In research studies, Valitor’s novel compounds have shown 10-fold increases in potency and up to 5-fold longer target tissue localization. These pharmacological advantages contribute synergistically to unprecedented retention of MVP conjugates in ocular tissues and a strategy for highly durable retinal medicines with an excellent preclinical safety profile.
Using the MVP technology, Valitor is developing a pipeline of best-in-class ophthalmic medicines with increased potency and extended therapeutic duration based on validated mechanisms of action. The MVP platform is designed for plug-and-play versatility and can be used to efficiently develop long-acting therapies using a variety of payloads, including compounds from industry collaborators as part of ongoing co-development activities.
Leanne Labriola, MD, CEO - Visionaire Products
Visionaire Products is a med tech company that has developed the first device to be FDA approved for anterior chamber paracentesis. This device has been developed to address the unmet needs for this procedure, including better handling of ocular fluid and increase control during collection. Features of hte device have been engineered with careful user feedback and iterations on design to achieve a major advancement over the current standard of care. We have an approved presubmission package from the FDA and will be completing verification and validation testing this summer with planned submission in November. These devices will be available for purchase in 2026.