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2026 RETINA PIPELINE

2026 RETINA PIPELINE

Dry & Wet Age-Related Macular Degeneration

A View Into Ongoing Innovation

Content guidance and source: Peter K. Kaiser, MD

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Download icon Download the AMD Retina Pipeline PDF

This content originally ran as a poster in the May/June issue. Check out the print publication to view the full-size poster, or use the links below to download the PDFs.

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Seeing the pivotal study badge peppered on both the wet AMD side and the dry AMD side of the poster, one has to wonder if we’re on the edge of an approval explosion that could alter the treatment landscape.

— Peter K. Kaiser, MD

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Watch as Peter K. Kaiser, MD, explains the poster and follow along with the graphics below.

When Retina Today updates its related macular degeneration (AMD) pipeline poster each year, I join the editorial team to review which elements need to be adjusted. One year, we re-drew the illustration of the complement cascade to make it more accurate. Another year, we added a color-coding system to the wet AMD pipeline candidate list to reflect which molecules targeted which receptors. In yet another year, we depicted the process by which gene therapy penetrates a cell to create a biofactory of therapeutic proteins. Each and every year, we added candidates to the list of pipeline products.

These tweaks slowly changed the face of the poster in service of creating a more perfect resource.

But this year, we did it all. New look, new illustration, new complement system schematic. And we actually cut some candidates rather than merely expanding the list. Think of it like pruning a bush: by removing candidates that have stalled in limbo for years, we made space for healthier candidates to grow.

We added badges to candidates that are undergoing pivotal clinical trials at the time of publication. Given the US FDA’s new rules (which may consider sufficient a single pivotal study rather than a pair of studies), we might expect to see even more candidates with this badge in the coming years. Seeing the pivotal study badge peppered on both the wet AMD side and the dry AMD side of the poster, one has to wonder if we’re on the edge of an approval explosion that could alter the treatment landscape.

Tyrosine kinase inhibitors (TKIs) in particular are in the spotlight. If FDA approval is granted to one or both candidates currently in the pre-approval period, shifts in practice patterns seem inevitable. We recommend keeping a close eye on these two molecules—while keeping the other eye, of course, on other candidates’ trials, lest a press release announcing their results sneaks up on you.

We simplified the complement cascade schematic on the dry AMD side of this year’s poster. The previous iterations, while accurate, were cluttered. This year’s edition trims the cascade to only those areas targeted by a pipeline candidate. Don’t let its simplicity fool you (the complement cascade remains exceedingly complex), but do enjoy the ease of this year’s layout.

As always, we thrive on feedback. If you have ideas for next year’s poster or want to include a technology not listed here, email Executive Director of Special Projects, Cara Deming, at cdeming@bmctoday.com.

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Dry AMD

Click an area on the diagram below to see how certain therapies play a role in treating dry AMD.

MOBILE USERS:

We recommend that you access this portion of the Pipeline on a desktop computer.

While a larger screen will make it easier to navigate and visualize the Suppress Inflammation diagram below, the mobile experience contains the same content.

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A. Suppress Inflammation

Hover icon

Hover over the numbers in the diagram or in the list below to see where each therapy suppresses inflammation along the pathway.

See the diagram below showing where each therapy suppresses inflammation along the pathway.

    Targeting the Complement Cascade:
  • 1

    vonaprument (Annexon)

    C1q Inhibition

    Pivotal Study

  • 2

    pegcetacoplan (Apellis/Biogen)

    C3 Inhibition

  • 3

    CB-2782-PEG (Catalyst Biosciences)

    C3 Protease

  • 4

    AMY106 (Amyndas Pharmaceuticals)

    C3 Inhibition

  • 5

    SLN501 (Silence Therapeutics/Mallinckrodt Pharmaceuticals)

    siRNA C3 mRNA Inhibition

  • 6

    ABBV-6628 (AbbVie)

    C5 Inhibition

  • 7

    cemdisiran (Alnylam Pharmaceuticals)

    siRNA C5 mRNA Inhibition

  • 8

    KNP-302 (Kanaph Therapeutics)

    C3b and CD59 Inhibition

  • 9

    avacincaptad pegol (Astellas)

    C5 Inhibition

    FDA Approved

  • 10

    ALXN1720 (Alexion)

    C5 Inhibition

  • 11

    pozelimab/cemdisiran (Regeneron)

    C5 Inhibition

    Pivotal Study

  • 12

    PASylated Nomacopan (Akari Therapeutics)

    C5/LTB4 Inhibition

  • 13

    Gene Therapy icon JNJ-1887 (Janssen)

    sCD59 Gene Therapy

  • 14

    Danicopan (Alexion Pharmaceuticals/AstraZeneca)

    Complement Factor D Inhibition

  • 15

    BCX9930 (BioCryst Pharmaceuticals)

    Complement Factor D Inhibition

  • 16

    Gene Therapy icon sFH GTx (4DMT)

    Complement Factor H Gene Therapy

  • 17

    Gene Therapy icon CFH GTx (Syncona)

    Complement Factor H Gene Therapy

  • 18

    Gene Therapy icon AAV.sFH (Aevitas Therapeutics)

    Short-Form Complement Factor H Gene Therapy

  • 19

    Gene Therapy icon VOY-101 (Perceive Biotherapeutics)

    Complement Factor H Gene Therapy

  • 20

    CB-4332 (Catalyst Biosciences)

    Recombinant Complement Factor I

  • 21

    OMS721 (Omeros)

    MASP-2 Inhibition

  • 22

    KNP-301 (Kanaph Therapeutics)

    C3b/VEGF Inhibition

  • 23

    Efdamrofusp alfa (Innovent Biologics)

    sCR1/VEGF Inhibition

  • 24

    NM5072 (NovelMed)

    Properdin Inhibition

  • 25

    Gene Therapy icon KRIYA-825 (Kriya Therapeutics)

    CR1/CR2 Gene Therapy

  • 26

    Gene Therapy icon CTx001 (Complement Therapeutics)

    CR1 Gene Therapy

  • 27

    iptacopan (Novartis)

    Complement Factor B Inhibition

  • 28

    ADX-038 (ADARx)

    Complement Factor B siRNA

  • 29

    SAR446597 (Sanofi)

    Complement C1s and Bb Inhibition

  • 31

    STL303 (Sitala Bio)

    Oral Complement Factor B Inhibition

  • Macrophage Repolarization
  • 30

    AVD-104 (Aviceda Therapeutics)

    Complement Factor H Activation

  • TMi-018 (Translatum Medicus)

    Blocks M1 Transcriptome Expression

  • Inflammasome Inhibition
  • K8 (Inflammasome Therapeutics)

  • Matrix Modulation
  • doxycycline (Galderma Labs)

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B. Lipid Metabolism

  • OSA-1-101 (Osanni Bio) HMG-CoA Reductase Inhibition
  • Gene Therapy icon VGX-0111 (VisgenX) ELOVL2 Gene Therapy
  • BRX011 (Biojiva) Deuterium-Stabilized DHA
  • BI 1584862 (Boehringer Ingelheim) Phospholipid Modulator
  • CTX203 (CharacterBio) Apo-mimetic (L4F) Peptide

C. Reduce Toxic By-Product Accumulation

    Prevents Amyloid Aβ Oligomer Assembly
  • GAL-101 (Galimedix Therapeutics/Thea)
  • valiltramiprosate (Alzheon)

D. Neuroprotection

Repair Mitochondrial Dysfunction/Oxidative Stress:
  • elamipretide (Stealth Biotherapeutics)

    Pivotal Study

  • risuteganib (Allegro/Affamed/Hanmi)
  • Gene Therapy icon OCLX-001 (Oculogenex) Gene Therapy to Reduce Oxidative Stress

E. Visual Cycle Modulation

  • gildeuretinol (Alkeus Pharmaceuticals)

    Pivotal Study

  • tinlarebant (Belite Bio)

    Pivotal Study

F. Other Approaches

  • Gene Therapy icon OCU410 (Ocugen) RORA (RAR-Related Orphan Receptor A) protein
  • CT1812 (Cognition Therapeutics) Selective σ-2 Inhibition
  • Xiflam (Inflammx/Bausch + Lomb) Connexin 43 Inhibition
  • Valeda Light Delivery System (LumiThera/Alcon) Photobiomodulation

    FDA Cleared

  • Xelafaslatide (ONL Therapeutics) FAS Inhibition
  • PST-611 (PulseSight Therapeutics) DNA Plasmid Expressing Transferrin
  • vutrisiran (Alnylam Pharmaceuticals) siRNA TTR mRNA Inhibition
  • FRX-001 (FerRx Bio) Ferroptosis Inhibition
  • Gene Therapy icon BS01 (Bionic Sight) ChronosFP Optogenetics
  • BISC-109S (BISC Therapeutics) Bax inhibitor
  • i-Lumen (i-Lumen Scientific) Microcurrent Stimulation

    Pivotal Study

  • EG-301 (Evergreen Therapeutics)

G. Stem Cells

    Human Embryonic Stem Cells (hESCs)
  • OpRegen (Lineage Cell Therapeutics, Genentech/Roche)
  • CPCB-RPE1 Implant (Regenerative Patch Technologies)
  • ASP7317 (Astellas)
  • Eyecyte-RPE (Eyestem Research Ltd)
  • RPESC-RPE-4W (Luxa Biotechnology)
  • hESC RPE Sheets (Pfizer)
  • HLS001 Cell Sheets (Sumitomo Dainippon Pharma)

  • Umbilical Stem Cells (hUTCs)
  • Human Umbilical Stem Cells (jCyte)

Wet AMD

Down icon

Continue to scroll to see which pathway and receptors are targeted with each therapy.

Gene Therapy

  • Green Square RGX-314 (Regenxbio, AbbVie)

    Pivotal Study

  • Green Square AL-001 (Beijing Anlong Biopharmaceutical Co)
  • Green Square HG202 (HuidaGene Therapeutics Co)
  • Triangle Circle Green Square ixoberogene soroparvovec (Adverum Biotechnologies)

    Pivotal Study

  • Triangle Circle Green Square NG101 (Neuracle Genetics)
  • Triangle Circle Green Square LX102 (InnoStellar Biotherapeutics)
  • Triangle Circle Green Square E301 (Samsung)
  • Triangle Circle Green Square FT-003 (Frontera)
  • Triangle Circle Green Square ABI-110 (Avirmax Biopharma)
  • Green Circle Triangle Circle Green Square 4D-150 (4D Molecular Therapeutics)

    Pivotal Study

  • Diamond Triangle Circle Green Square EXG-102-031 (Exogenesis Bio)
  • Triangle Circle Green Square KH631 (Kanghong Biotech)
  • Triangle Circle Green Square KH658 (Vanotech)
  • Green Square HG202 (HuidaGene Therapeutics)
    CRISPR/Cas13 RNA Editing
  • Green Square BD311 (Shanghai BDgene Therapeutics Co)
  • Triangle Circle Green Square SKG0106 (Skyline Therapeutics)
  • Green Square RRG001 (Refreshgene Therapeutics)
  • Green Square IVB103 (Innovec Biotherapeutics)
  • Green Square PST-809 (Pulsesight Therapeutics)
    Non-Viral Anti-VEGF with Decorin Plasmid
  • Triangle Circle Green Square SAR402663 (Sanofi)
  • Green Square AL-001 (Beijing Anlong Biopharmaceutical Co)
  • Diamond Green Square CoTx-101 (Coave Therapeutics)

Extracellular VEGF Pathways

  • Green Square pegaptanib (Bausch + Lomb)

    FDA approved

  • Green Square Port Delivery System (Genentech/Roche)

    FDA approved

  • Green Square ranibizumab (Genentech/Roche)

    FDA approved

  • biosimilars to ranibizumab:
  • Green Square ranibizumab-nuna (Samsung/Biogen)

    FDA approved

  • Green Square ranibizumab-eqrn (Sandoz)

    FDA approved

  • Green Square ranibizumab-Ximluci (Xbrane Biopharma)

    EMA approved

  • Green Square ranibizumab-Ranivisio (Teva)

    EMA Approved

  • Green Square ranibizumab-RanizuRel (Reliance Life Sciences)

    India FDA approved

  • Green Square ranibizumab-Razumab (Intas Pharmaceuticals)

    India FDA approved

  • Green Square ranibizumab-leyk (Formycon/Bioeq)
  • Green Square LUBT010 (Lupin Ltd/Amman)
  • Green Square SCT520FF (SinoCellTech)
  • Green Square SJP1-0133 (Senju Pharmaceutical)
  • Green Square BCD100 (BIOCND/Qilu)
  • Green Square CKD-701 (Chong Kun Dang)
  • Green Square bevacizumab
  • biosimilars to bevacizumab:
  • Green Square bevacizumab-vikg (Outlook Therapeutics)

    EMA/UK approved

  • Green Square TAB014 (Zhaoke Ophthalmology)
  • Green Square CT-P16 (Celltrion)
  • Green Square alymsys (Amneal Pharmaceuticals/mAbxience)
  • Green Square EB12-20145P (EssexBio)
  • Green Square AK-3008 (Anhui Anke Biotechnology)
  • Green Square MIL-60 (Beijing Mabworks Biotech)
  • Green Square brolucizumab (Novartis)

    FDA approved

  • Green Square abicipar pegol (Molecular Partners)
  • Green Square KNP-301 (Kanaph Therapeutics)
  • Green Square tarcocimab tedromer (Kodiak Sciences)

    Pivotal Study

  • Green Square VLTR-559 (Valitor)
    Multivalent Polymer Conjugation
  • Triangle Circle Green Square high-dose aflibercept (Regeneron)

    FDA approved

  • Triangle Circle Green Square aflibercept (Regeneron)

    FDA approved

  • biosimilars to aflibercept:
  • Triangle Circle Green Square aflibercept-ayyh (Amgen)

    FDA Approved

  • Triangle Circle Green Square aflibercept-jbvf (Biocon)

    FDA Approved

  • Triangle Circle Green Square aflibercept-yszy (Samsung/Biogen)

    FDA Approved

  • Triangle Circle Green Square aflibercept-abzv (Sandoz)

    FDA Approved

  • Triangle Circle Green Square aflibercept-mrbb (Formycon)

    FDA Approved

  • Triangle Circle Green Square aflibercept-boav (Celltrion)

    FDA Approved

  • Triangle Circle Green Square ALT-L9 (Altos Biologics)
  • Triangle Circle Green Square MYL-1701 (Mylan/Momenta)
  • Triangle Circle Green Square RBS-001 (Rophibio)
  • Triangle Circle Green Square SCD411 (Sam Chun Dang Pharmaceutical Co./Fresenius Kabi)
  • Triangle Circle Green Square OT-702 (Ocumension Therapeutics/Shandong Boan)
  • Triangle Circle Green Square AVT-06 (Alvotech)
  • Triangle Circle Green Square conbercept (Kanghong Biotech)

    CDE Approved

  • Triangle Circle Green Square IBI302 (Innovent Biologics)
    Plus CR1 Inhibition
  • Green Circle Circle Green Square IBI333 (Innovent Biologics)
  • TH103 (Kalaris Therapeutics)
    VEGFR1 domain3 + heparin-binding proteins
  • OPT-640 (OptigoBio)

TIE2 Activation Pathways

  • Diamond Green Square faricimab (Genentech/Roche)

    FDA Approved

  • Diamond Green Square BI 836880 (Boehringer Ingelheim)
  • Diamond Green Square VIS-101 (Visara)
  • Diamond Green Square ABP-201 (AbPro)
  • Diamond Green Square OLN324 (Ollin Biosciences)
  • Diamond Green Square zifibancimig (Roche)
  • Green Circle Diamond Green Square RO-104 (RevOpsis)
  • Triangle Circle Diamond Green Square EB-105 (Eluminex Biosciences)
  • Diamond Green Square AM-712 (AffaMed Therapeutics)
  • PMC-403 (PharmAbcine)
    TIE2 Activating MAb
  • tiespectus (Merck/EyeBio)
    TIE2 Activating bi-specific
  • IGT-427 (Ingenia Therapeutics/Mosaic)
    TIE2 Activating/anti-VEGF

Tyrosine Kinase inhibitor (TKi) Pathways

  • Axpaxli/axitinib (Ocular Therapeutix)

    Pivotal Study

  • AR-14034/axitinib (Alcon/Aerie)
  • Duravyu/vorolanib (EyePoint Pharmaceuticals)

    Pivotal Study

  • AIV007/lenvatinib (AiViva Biopharma)
  • D-4517.2/migaldendrinib / sunitinib analog (Ashvattha Therapeutics)
  • KPI-287 (Kala Pharmaceuticals)
  • TO-O-1002 (TheratOcular Biotek Co., Ltd.)
  • PAN 90806 (PanOptica)
  • KHK4951/tivozanib (Kyowa Kirin Group)
  • HX9428 (Fujian Haixi Pharmaceuticals)

WNT Pathways

  • Diamond Outline SYL-1801 (Sylentis)
    siRNA Nrarp Inhibitor
  • Diamond Outline MK-3000 (EyeBio/Merck)
    WNT Agonist
  • SZN-413 (Surrozen)
    Fzd4 Agonist
  • SZN-8141 (Surrozen)
    Fzd4 Agonist/Anti-VEGF

Interleukin Pathways

  • KSI-501 (Kodiak Sciences)
    Anti-VEGF/IL6 Antibody Polymer Conjugate (ABC)

    Pivotal Study

  • KSI-101 (Kodiak Sciences)
    Anti-IL6/VEGF
  • SZN-8143 (Surrozen)
    Anti-IL6/anti-VEGF/Fzd4 Agonist
  • LNR-653.1 (Lanier Biotherapeutics)
    Anti-IL17A/VEGF
  • RO7497372 (Genentech)
    Anti-IL6/VEGF

Other Pathways

  • CVX-51401 (CavtheRx)

    Caveolin Modulator

  • EXN407 (Exonate/Janssen)

    SRPK1 Inhibitor

  • aganirsen (Gene Signal)

    Inhibition of Insulin Receptor Substrate 1 (IRS-1)

  • PL9654 (Palatin Technologies)

    Melanocortin Receptor Agonist

  • foselutoclax (Unity Biotechnology)

    Bcl-xL Inhibitor

  • EOM147 (EOM Pharmaceuticals)

    Squalamine Analog

  • ISTH0036 (Isarna Therapeutics)

    TGF-Beta2 Antisense Oligonucleotide

  • ATX-9874 (Augen/Duet Therapeutics)

    Indoleamine 2,3-dioxygenase (IDO) Inhibitor

  • CU06 (Curacle/Thea)

    Block cAMP/Cortactin Pathway

  • OLX-301D (OliX)

    cp-asiRNA-CTGF/CCN2 Inhibitor

  • umedaptanib pegol (Ribomic)

    FGF2 Inhibitor

  • RC28-E (RemeGen)

    FGF2/VEGF Inhibitor

    Pivotal Study

  • CG-P5 (Caregen)

    CG-P5 Peptide

  • OGX-110 (Ocugenix)

    CXC Chemokine Receptor 3 Agonist

  • BBC1501 (BenoBio)

    Targets BET (bromodomain and extra-terminal) Proteins

  • ABF-101/103 (AptaBio)

    Nox Inhibitor

  • OCUL101 (Oculgen)

    Anti-VEGF/C5

Integrin Pathways

  • Green Square Star AG-73305 (Allgenesis Biotherapeutics)
  • Diamond Green Square Star gersizangitide (AsclepiX Therapeutics)
  • PAN:
  • risuteganib (Allegro/Affamed/Hanmi)
  • OCU200 (Ocugen)
    Tumstatin and Transferrin Fusion Protein
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Download the AMD Retina Pipeline PDF

This content originally ran as a poster in the May/June issue. Check out the print publication to view the full-size poster, or download the PDF.

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Question Icon

Did we miss a drug candidate?

If you wish to include a candidate for wet AMD, dry AMD, or GA therapy in next year's poster, email Peter K. Kaiser, MD, at pkkaiser@gmail.com and Cara Deming, Executive Director of Special Projects at Bryn Mawr Communications, at cdeming@bmctoday.com.

View previous pipelines to see how it has evolved over the years

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Peter K. Kaiser, MD Headshot

Peter K. Kaiser, MD

Medical advisor, Retina Today

Chaney Family Endowed Chair in Ophthalmology Research; Professor of Ophthalmology, Cleveland Clinic Lerner College of Medicine; staff surgeon in the vitreoretinal department at the Cole Eye Institute, Cleveland Clinic; founding director of the Digital Optical Coherence Tomography Reading Center at the Cole Eye Institute, all in Cleveland, Ohio

pkkaiser@gmail.com

Financial disclosures: Advisory Board and Consultant (AbbVie/Allergan, Aerie, Aerpio, Alcon, Allegro, Annexon Biosciences, AsclepiX, Bayer, Bausch + Lomb, Biogen Idec, Boehringer Ingelheim, Carl Zeiss Meditec, Clearside Biomedical, Eyevensys, Formycon/BioEq GmbH, Galecto Biotech, Galimedix, Glaukos, iRenix, jCyte, Kala Pharmaceuticals, Kanghong, Kodiak, NGM Biopharmaceuticals, Novartis, Ocugenix, Oculis, Omeros, Opthea, Oxurion [ThromboGenics], Regeneron, RegenexBio, Retinal Sciences, Roivant, Santen, SciFluor, Shire, Spark, Stealth Biotherapeutics, Takeda, Verana Health [Digisight])