CFH

Dry macular degeneration is a common eye disorder among people aged above 50 years. It causes blurred or reduced central vision due to thinning of the macula. The macula is the part of the retina responsible for clear vision in a direct line of sight. The pharmaceutical industry is increasingly focused on developing pathway-based therapies for dry AMD, with promising treatments like ALK-001 and LBS-008 in trials, though a combination approach may be needed to fully inhibit disease progression. LAS VEGAS, Jan. 23, 2025 /PRNewswire/ -- DelveInsight's ' Dry Age-Related Macular Degeneration Pipeline Insight 2025 ' report provides comprehensive global coverage of pipeline dry AMD therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the dry AMD pipeline domain. Key Takeaways from the Dry Age-Related Macular Degeneration Pipeline Report DelveInsight's dry AMD pipeline report depicts a robust space with 70+ active players working to develop 80+ pipeline therapies for dry AMD treatment. Key dry AMD companies such as Alkeus Pharmaceuticals, Dobecure, Belite Bio, Cognition Therapeutics, Aviceda Therapeutics, Stealth BioTherapeutics, Allergo Ophthalmics, Annexon, Inc., Johnson & Johnson, InflammX Therapeutics, Lineage Cell Therapeutics, Ionis Pharmaceuticals, Evergreen Therapeutics, Inc., Alexion, Luxa Biotechnology, Astellas Pharma, OliX Pharmaceuticals, Hoffmann-La Roche, Boehringer Ingelheim, Eyevensys and others are evaluating new dry AMD drugs to improve the treatment landscape. Promising dry AMD pipeline therapies such as ALK-001, Etamsylate, Tinlarebant (LBS-008), CT1812, AVD-104, Elamipretide, Risuteganib, ANX 007, JNJ 81201887, Xiflam, OpRegen, IONIS-FB-LRx, EG-301, ALXN2040, RPESC-RPE-4W, MA09-hRPE, OLX301A, RO7303359, BI 771716, EYS 611 and others are under different phases of dry AMD clinical trials. In December 2024, Galimedix Therapeutics announced the initiation of its Phase II eDREAM study to assess the safety and efficacy of GAL-101 eye drops in patients with dry age-related macular degeneration. In December 2024, Ocugen, Inc. announced that the Data and Safety Monitoring Board (DSMB) for the OCU410 ArMaDa clinical trial recently convened and approved continuation of the second phase of the Phase 1/2 study. OCU410 (AAV5-hRORA) is a novel modifier gene therapy candidate being developed for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). In November 2024, Ocugen, Inc. announced positive preliminary efficacy and safety data from the Phase I dose-escalation portion of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD). Key findings include: no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and most importantly there was a positive effect on the functional visual measure of low luminance visual acuity (LLVA). In October 2024, Eyestem Research, focused on creating scalable cell therapies for incurable diseases, had announced a positive outcome of the Drug Safety Monitoring Board (DSMB) review for the first cohort of its Phase one trial to treat Geographic Atrophy secondary to Dry Age-related Macular Degeneration, marking a milestone for Indian science and medical research. In September 2024, Stealth BioTherapeutics Inc. announced the presentation of new bevemipretide (SBT-272) preclinical data demonstrating topical ocular delivery to the retina with protective effects observed in models of AMD. In June 2024, Stealth BioTherapeutics Inc. announced that it has enrolled and dosed its first patient in the ReNEW trial as part of its Phase III clinical program for elamipretide in patients with dry age-related macular degeneration (dry AMD). The program consists of the two Phase III trials, ReNEW and ReGAIN. Both trials will evaluate the efficacy, safety, and pharmacokinetics of daily subcutaneous injections of elamipretide in participants with dry AMD. In April 2024, Eyestem received approval from India's Central Drugs Standards Control Organisation (CDSCO) to commence human trials for Eyecyte RPE, a potential treatment for geographic atrophy arising from dry age-related macular degeneration (AMD). Request a sample and discover the recent advances in dry AMD treatment drugs @ Dry Age-Related Macular Degeneration Pipeline Report The dry AMD pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage dry AMD drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the dry AMD clinical trial landscape. Dry Age-Related Macular Degeneration Overview Age-related macular degeneration (AMD) is one of the top four causes of blindness in older adults and the leading cause of irreversible blindness in developed countries. Around 170 million people globally are affected by AMD, making it the third most common cause of vision loss worldwide. Dry AMD typically progresses slowly, but wet AMD can cause severe visual impairment within days or weeks of onset. In the early stages of AMD, there are no noticeable symptoms, though an ophthalmologist can detect drusen before symptoms arise. Some individuals experience slight vision changes during the intermediate stage, but many remain symptom-free. Others may notice dark or grey spots in their central vision or have difficulty transitioning from bright to dim lighting. Clinicians currently use various imaging techniques to monitor changes in the retina, retinal pigment epithelium (RPE), and choroid, including fundus exams, color fundus photography, fundus autofluorescence (FAF), optical coherence tomography (OCT), and infrared reflectance imaging. Over the past two decades, OCT has significantly advanced the early detection, monitoring, and treatment assessment of dry AMD. Modern spectral-domain OCT (SD-OCT) systems provide detailed cross-sectional and volumetric views of the retina, enabling clinicians to visualize, measure, and monitor retinal layers, RPE, hyperreflective foci, geographic atrophy (GA), and drusen. While SD-OCT remains the standard of care, emerging technologies like polarization-sensitive OCT (PS-OCT), which offers tissue-selective imaging, may revolutionize AMD research and clinical practices. Currently, no approved treatments exist for dry AMD. Management involves regular monitoring, timely detection of visual deterioration, appropriate rehabilitation, and early identification of choroidal neovascularization (CNV). Research is underway to explore therapeutic strategies to prevent AMD, slow its progression, or restore vision. Potential treatments include lifestyle and behavioral changes, age-related eye disease study (AREDS) supplements, visual cycle modulators, anti-inflammatory agents, complement inhibitors, gene therapy, cell-based therapies, and approaches to reduce toxic by-products. Find out more about dry AMD treatment drugs @ Drugs for Dry Age-Related Macular Degeneration Treatment A snapshot of the Dry Age-Related Macular Degeneration Pipeline Drugs mentioned in the report: Learn more about the emerging dry AMD pipeline therapies @ Dry Age-Related Macular Degeneration Clinical Trials Dry Age-Related Macular Degeneration Therapeutics Assessment The dry AMD pipeline report proffers an integral view of the dry AMD emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Dry Age-Related Macular Degeneration Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Dimerisation inhibitors, Vitamin A replacements, RBP4 protein inhibitors, Sigma-2 receptor antagonists, Complement factor H stimulants, Macrophage inhibitors, Cell replacements, RNA interference, Key Dry Age-Related Macular Degeneration Companies: Alkeus Pharmaceuticals, Dobecure, Belite Bio, Cognition Therapeutics, Aviceda Therapeutics, Stealth BioTherapeutics, Allergo Ophthalmics, Annexon, Inc., Johnson & Johnson, InflammX Therapeutics, Lineage Cell Therapeutics, Ionis Pharmaceuticals, Evergreen Therapeutics, Inc., Alexion, Luxa Biotechnology, Astellas Pharma, OliX Pharmaceuticals, Hoffmann-La Roche, Boehringer Ingelheim, Eyevensys and others Key Dry Age-Related Macular Degeneration Pipeline Therapies: ALK-001, Etamsylate, Tinlarebant (LBS-008), CT1812, AVD-104, Elamipretide, Risuteganib, ANX 007, JNJ 81201887, Xiflam, OpRegen, IONIS-FB-LRx, EG-301, ALXN2040, RPESC-RPE-4W, MA09-hRPE, OLX301A, RO7303359, BI 771716, EYS 611 and others. Dive deep into rich insights for new drugs for dry AMD treatment, visit @ Dry Age-Related Macular Degeneration Drugs Table of Contents For further information on the dry AMD pipeline therapeutics, reach out @ Dry Age-Related Macular Degeneration Treatment Drugs Related Reports Dry Age-Related Macular Degeneration Market Dry Age-Related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key dry AMD companies including Alkeus Pharmaceuticals, Belite Bio, Aviceda Therapeutics, Johnson & Johnson Innovative Medicine, Allegro Ophthalmics, Lineage Cell Therapeutics (CellCure Neurosciences), Roche, Cognition Therapeutics, Stealth BioTherapeutics, Evergreen Therapeutics, Annexon Biosciences, NGM Biopharmaceuticals, AstraZeneca, Alexion Pharmaceuticals, Ionis Pharmaceuticals, Novartis, Regenerative Patch Technologies, among others. Dry Age-related Macular Degeneration Epidemiology Forecast Dry Age-related Macular Degeneration Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted dry AMD epidemiology in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan. Age-related Macular Degeneration Market Age-related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key AMD companies including Regeneron Pharmaceuticals, Novartis, Roche, Opthea Limited, Kodiak Sciences Inc., REGENXBIO, Alkahest Inc, Graybug Vision, Ribomic USA Inc, Outlook Therapeutics, Inc., Unity Biotechnology, Inc, PanOptica, Inc., Clearside Biomedical, Alexion Pharmaceuticals, AstraZeneca, Evergreen Therapeutics, Alkeus Pharmaceuticals, Stealth BioTherapeutics, CellCure Neurosciences, Regenerative Patch Technologies, Allegro Ophthalmics, Annexon Biosciences, NGM Biopharmaceuticals, Ionis Pharmaceuticals, Apellis Pharmaceuticals, Iveric Bio, Gyroscope Therapeutics, Luxa Biotechnology, Gemini Therapeutics , among others. Wet Age-related Macular Degeneration Market Wet Age-related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Wet Age-related Macular Degeneration companies including EyePoint Pharmaceuticals, Inc., AbbVie, Caregen Co. Ltd., Exegenesis Bio, Shanghai Henlius Biotech, Skyline Therapeutics, 4D Molecular Therapeutics, Ocugenix Corporation, Adverum Biotechnologies, Inc., Ashvattha Therapeutics, Inc., AiViva BioPharma, Inc., Ocular Therapeutix, Inc., Clearside Biomedical, Inc., Hoffmann-La Roche, Kyowa Kirin, Inc., Opthea Limited, AffaMed Therapeutics Limited, EyeBiotech Ltd., Novartis , among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

CAMBRIDGE, Mass.--( BUSINESS WIRE )--Aviceda Therapeutics (“Aviceda”), a private, clinical-stage biotech company focused on developing next-generation immunomodulators with a proprietary High Affinity Ligands of Siglecs (HALOS™) nanotechnology platform to alleviate chronic, non-resolving inflammation, today announced the closing of a $207.5 million Series C financing. Proceeds will support the ongoing Phase 2b/3 and planned pivotal Phase 3 clinical trial for AVD-104 in geographic atrophy (GA), a vision-threatening condition secondary to advanced age-related macular degeneration, and the expansion into other ophthalmic indications. The Series C round was co-led by renowned healthcare funds, Omega Funds and TCGX, with participation from leading investment firms Enavate Sciences, Jeito Capital, Blue Owl Healthcare Opportunities, Longitude Capital, OrbiMed, Logos Capital, Marshall Wace, Catalio Capital Management, funds managed by abrdn Inc., and Digitalis Ventures. “ We are proud to support Aviceda and its team of retinal specialists, whose blend of ophthalmic expertise and operational excellence drives Aviceda’s advancement of AVD-104 toward critical milestones,” commented Bernard Davitian, Partner at Omega Funds. AVD-104’s dual mechanism of action targets immune modulation and complement inhibition. By repolarizing overactivated macrophages to their healing stage and stabilizing complement factor H, AVD-104 has shown positive proof-of-concept efficacy in slowing GA lesion growth and preserving/enhancing visual function in a Phase 2a trial completed in early 2024, which underscores its potential for first-in-class efficacy and safety. Aviceda’s ongoing Phase 2b/3 trial evaluating two doses of AVD-104 versus avacincaptad pegol (Izervay) in patients with GA is fully enrolled , with 12-month primary endpoint data anticipated in the second half of 2025. Chen Yu, M.D., M.B.A., Founder and Managing Partner at TCGX, added, “ Aviceda’s focus on data quality and execution is evident in the progress of the AVD-104 program, and we believe the company is poised for significant impact in the geographic atrophy field with strong potential upside as it advances into a pivotal study.” In connection with the Series C financing, Bernard Davitian of Omega Funds and Chen Yu, M.D., M.B.A., of TCGX have joined Aviceda’s Board of Directors. Evelyn Harrison, M.B.A., former Chief Operating Officer of Iveric Bio (part of Astellas Pharma Inc.) has also joined as an independent director, bringing valuable expertise in advancing novel therapies through late-stage clinical development and commercialization. In addition, Sara Nayeem, M.D. of Enavate Sciences and Rafaèle Tordjman, M.D. Ph.D. of Jeito Capital are appointed Board Observers. David Guyer, M.D., Board Chair of Aviceda, said, " We are pleased to welcome our new board members and a syndicate of top institutional investors. This raise reflects the strong conviction from investors in Aviceda’s lead program AVD-104 for geographic atrophy—a market poised for disruption and an improved standard of care, given current treatments offer limited benefits in visual improvements, lesion control, and safety. With this upsized financing, the company is well-capitalized to advance a pivotal program for AVD-104 in GA.” Mintz, Levin, Cohn, Ferris, Glovsky, and Popeo, P.C. is acting as legal counsel to Aviceda, with Cooley LLP representing Omega Funds and Foley & Lardner LLP advising TCGX. Leerink Partners served as financial advisor to Aviceda. About Aviceda Therapeutics and AVD-104 Aviceda is a private, clinical-stage biotechnology company located in Cambridge, MA, with a proprietary High Affinity Ligands of Siglecs (HALOS™) nanotechnology platform and a clinical-stage ophthalmic candidate, AVD-104, for the treatment of geographic atrophy secondary to age-related macular degeneration. AVD-104 is an intravitreal glycan-coated nanoparticle with a dual mechanism of action that modulates critical inflammatory cellular and complement pathways through 1) direct deactivation of inflammatory microglia/macrophages’ phagocytic activity and repolarization of these activated microglia/macrophages to their healing state and 2) inhibition of complement cascade amplification. Along with AVD-104, Aviceda has a broad pipeline of products in development in ophthalmology and multiple other therapeutic areas, including immunology, fibrosis, oncology, and neurology. Learn more about Aviceda Therapeutics and follow the company on LinkedIn .

4D-175 comprises the proprietary low-dose intravitreal R100 AAV vector and a codon-optimized transgene encoding a highly functional shortened form of human complement factor H (sCFH) Complement factor H (CFH) variants with reduced function are a well-validated genetic risk factor for geographic atrophy (GA), with approximately 75% of age-related macular degeneration (AMD) patients carrying a high-risk variant of CFH Over 150 patients have been treated with the R100 vector, including those with wet AMD and diabetic macular edema, further validating the modularity of our Therapeutic Vector Evolution platform Enrollment for Phase 1 GAZE clinical trial is expected to begin in H2 2024 EMERYVILLE, Calif., June 24, 2024 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced U.S. Food and Drug Administration (FDA) clearance of the Investigational New Drug Application (IND) for 4D-175, an R100 vector-based intravitreal genetic medicine, for the treatment of patients with GA. The Phase 1 GAZE clinical trial will assess 4D-175 in patients with GA secondary to AMD. The study design consists of an open-label, sequential cohort Dose Exploration stage, in which patients will receive a single intravitreal injection of 4D-175 at one of three dose levels. Clinical trial objectives include safety and tolerability, definition of the Phase 2 trial dose level(s), transgene expression and biological activity. The IND clearance enables the initiation of GAZE clinical study sites, and 4DMT expects to begin enrollment in H2 2024. “GA is a leading cause of irreversible vision loss for over 5 million people globally and while current bolus complement inhibitor treatments reduce the rate of growth in GA lesions, they require burdensome monthly or bimonthly intravitreal injections and do not demonstrate functional vision benefit,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “4D-175 has the potential for durable clinical benefit with a single intravitreal injection, greatly reducing the current treatment burden for patients, which may lead to better long-term vision outcomes. In Phase 1, we aim to explore safety and transgene expression levels to select doses for Phase 2. We look forward to beginning enrollment in the Phase 1 GAZE clinical trial in the second half of 2024.” sCFH is an engineered and optimized version of CFH that can fit into adeno associated virus (AAV) vectors with robust expression and full functionality confirmed in human cells in vitro, as well as in multiple preclinical animal models and species in vivo. The construct was co-invented by Wenchao Song, Ph.D., Professor of Pharmacology at the Perelman School of Medicine at the University of Pennsylvania. Dr. Song has extensive experience researching complement-mediated inflammatory, autoimmune and thrombotic vasculopathy disorders. Restoring CFH function through targeted delivery of a therapeutic sCFH transgene could restore normal complement regulation and reduce retinal injury that manifests as progressive GA. Preclinical proof-of-concept for this approach using 1) human sCFH delivered systemically using an AAV vector in a mouse model of atypical hemolytic uremic syndrome (aHUS) and 2) a mouse version of sCFH delivered using an AAV vector in mouse models of C3 glomerulopathy and aHUS each demonstrated recovery from complement dysregulation, reduced organ damage and improved survival. Preclinical data from 4D-175 in vitro and in vivo characterization studies were presented at the 2024 ARVO Annual Scientific Meeting in May; the presentation can be found on the 4DMT website here. About Geographic Atrophy Geographic atrophy (GA) is a highly prevalent disease with a significant unmet medical need. It is estimated that there are approximately 2.5 million individuals with GA in the United States and major European markets, and an estimated 5 million individuals globally. GA is an advanced and severe form of age-related macular degeneration (AMD) that leads to irreversible vision loss. GA is characterized by atrophic lesions in the outer retina that affect central vision and lead to irreversible vision loss. Progressive loss of central vision leads to difficulties driving, reading, and completing basic daily tasks. As a result, GA has a major impact on quality of life. Complement-mediated inflammation is recognized as a main contributor to the development and worsening of GA. The only FDA approved treatments for GA are complement inhibitors administered by intravitreal (IVT) injection once every 4-9 weeks. About 4D-175 for Geographic Atrophy 4D-175 combines the customized and evolved intravitreal vector, R100, and a codon-optimized transgene encoding a highly functional shortened form of human complement factor H (sCFH). The genetic medicine candidate is designed to provide durable transgene expression in the retina without significant inflammation following a single, low-dose intravitreal injection. The sCFH payload is designed to restore normal complement regulation, which has the potential to slow progression of disease. About 4DMT 4DMT is a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases in ophthalmology and pulmonology. 4DMT’s proprietary invention platform, Therapeutic Vector Evolution, combines the power of the Nobel Prize-winning technology, directed evolution, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors for use in our wholly owned and partnered product candidates. Our product design, development, and manufacturing engine helps us efficiently create and advance our diverse product pipeline with the goal of revolutionizing medicine with potential curative therapies for millions of patients. Currently, 4DMT is advancing six clinical-stage and one preclinical product candidate, each tailored to address rare and large market diseases in ophthalmology, pulmonology and cardiology. In addition, 4DMT is also advancing programs in CNS through a gene editing partnership. 4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT. All of our product candidates are in clinical or preclinical development and have not yet been approved for marketing by the U.S. Food and Drug Administration (FDA) or any other regulatory authority. No representation is made as to the safety or effectiveness of our product candidates for the therapeutic uses for which they are being studied. Learn more at and follow us on LinkedIn. Forward Looking Statements: This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding the therapeutic potential, and clinical benefits of 4DMT’s product candidates, as well as the plans, announcements and related timing for the clinical development of 4D-175. The words "may," “might,” "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," “expect,” "estimate," “seek,” "predict," “future,” "project," "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including risks and uncertainties that are described in greater detail in the section entitled "Risk Factors" in 4D Molecular Therapeutics’ most recent Quarterly Report on Form 10-Q as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent 4D Molecular Therapeutics' views only as of today and should not be relied upon as representing its views as of any subsequent date. 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Contacts: Media: Katherine Smith Inizio Evoke Comms Katherine.Smith@inizioevoke.com Investors: Julian Pei Head of Investor Relations and Corporate Finance Investor.Relations@4DMT.com