Meiragtx Holdings PlcNASDAQ: MGTX

Corporate Deck January

· MarketScreener
Corporate Presentation

January 2026

1



Company Overview




MeiraGTx: late-stage clinical pipeline and comprehensive end-to-end capabilities s technologies in genetic medicine

Diverse Program

Pipeline

Broad pipeline across neuro, salivary gland, and ophthalmology

4 pivotal and BLA ready programs:

  • Radiation-induced xerostomia

  • Parkinson's disease

  • AIPL1 retinal dystrophy (Eli Lilly)

  • X-linked retinitis pigmentosa1

    Diverse preclinical pipeline:

  • ALS, Neuropathic pain

  • Obesity: incretin combinations, leptin, BDNF, PTH, Epo, hGH

  • Ophthalmology: pipeline

    Stargardt's, wet and Dry AMD

  • Specials license: BBS10, pain

End-to-End GMP

Manufacturing

In-house manufacturing and industry-leading process

  • 2 cGMP viral vector manufacturing facilities

  • cGMP plasmid production

  • QC facility for release and stability

  • In-house Fill C Finish, warehouse and supply chain

  • Dedicated MSAT facility

  • Commercial ready Platform

Process and QC

Commercial licenses for both viral vector production as well as QC

Next-Generation

Vector Optimization

Improved potency C safety, lower dose and lower COGS

  • >250k promoter library

  • AI enhanced promoter

    optimization

  • Proprietary intravitreal capsids

  • Capsid development: muscle, CNS

  • Human organoids

Improve potency up to 3 to 4 logs, reducing dose 3-4 logs, reducing Cost of Goods and improving safety

Transformative in vivo

production Technology

Proprietary Riboswitch platform for precise control of therapeutic proteins

in vivo production of physiological, efficacious levels of any therapeutic protein via bespoke small molecule oral dosing

  • Gene agnostic: multiple antibodies, peptides, hormones, nucleases, cell therapy validated in animal models

  • Delivery agnostic: AAV, Lentivirus and CRISPR all demonstrated equivalent tight control

  • Leptin: first into the clinic, 2026





1 Remaining interests in program sold to Johnson s Johnson Innovative Medicine in December 2023; MeiraGTx to receive up to an aggregate of $350.0 million upon achievement of milestones and will manufacture and supply commercial product for Johnson s Johnson Innovative Medicine

4

Broad Pipeline of Transformative Genetic Medicines

Advanced clinical programs across multiple therapeutic areas

Product

Indication

Preclinical

Phase 1

Phase 2

Phase 3 /

Registrational

Salivary Gland

AAV-AQP1

Radiation-induced xerostomia





RMAT, Orphan Drug

Sjögren's disease

IND ready

PSMA radioligand xerostomia

prophylaxis and treatment



Neurodegenerative Disease

AAV-GAD1

Parkinson's disease



RMAT

Phase 3 ready

AAV-UPF1, AAV-CNTFR

ALS



Ophthalmology

Botaretigene sparoparvovec2

X-linked RP (RPGR)





PRIME, Fast Track, Orpha

n Drug

AAV-AIPL1



LCA4 congenital blindness

RPDD, Orphan Drug, MHR

A Specials License

AAV-ABCA4

Stargardt's disease



AAV-VEGFR2

Wet AMD



Undisclosed

Dry AMD/GA



BBS10

Bardet-Biedl syndrome



RPDD, Orphan Drug

Developed under MHRA Spe

cials License

Riboswitch Regulated Therapies

RiboLeptin

Lipodystrophies



Undisclosed

Intractable neuropathic pain



GLP-1, GIP, incretin combinations

Obesity/MASH/Metabolic Disease



Ribo-CAR-T

Oncology, autoimmune disease



Genetic Obesity

AAV-BDNF1

MC4R/BDNF genetic obesity





1 Joint venture with Hologen AI (see press release here)

2 Remaining interests in program sold to Johnson s Johnson Innovative Medicine in December 2023; MeiraGTx to receive up to an aggregate of $350.0 million upon achievement of milestones and will manufacture and supply commercial 5

product for Johnson s Johnson Innovative Medicine

Four pivotal stage programs in prevalent and rare indications


01 02 03 04

Radiation-Induced

Xerostomia

  • Pivotal Phase 2

  • Potential BLA filing early 2027

  • Large patient population with no effective therapies available

  • 'Pipeline in a product'

    Parkinson's

    Disease

  • Phase 3 ready

  • Potential BLA filing in 2028

  • Large patient population inadequately controlled by dopamine therapy

    AIPL1-Associated Congenital Blindness

  • Developed under 'specials'

    license

  • Near term BLA and MAA filings-FDA and MHRA

  • Potential approvals 2026

  • Transformative effect - 11/11 blind to seeing children under 4 years

    X-Linked Retinitis Pigmentosa (RPGR)

  • Completed Phase 3

  • BLA and MAA ready -

    Clinical and PPQ

  • Acquired by Johnson C Johnson Innovative Medicine; MeiraGTx manufactures commercial product

Unique end-to-end, in-house GMP manufacturing infrastructure and production platform process, scalable and flexible, fit for clinical s commercial supply

End-to-end internal manufacturing infrastructure, capabilities and production process

Best in class, fully end-to end internal capabilities: Two flexible C scalable cGMP vector production facilities, London, UK and Shannon, Ireland; cGMP plasmid production facility; cGMP QC facility for release and stability; fill and finish; dedicated MSAT process development facility



Commercial licenses in Ireland and UK for

viral vector production and QC

Proprietary Commercial Ready Manufacturing Process at IND: Saves 2-3 years in AAV clinical development timeline from IND to commercial and allows faster move to pivotal with expedited time to market

Global regulatory relationships and extensive

experience from pre-IND through BLA/commercial

Commercial grade manufacturing with scalable and flexible capacity, clinical and commercial supply



7

Comprehensive vector engineering technologies

Potency C safety optimization and precise control of gene expression

In-house vector engineering platforms

Extensive in-house vectorology capabilities addressing each element of the vector genome sequence

Promoter engineering s discovery

>250k promoter library



Combine rational design, massive high throughput screening, and AI to optimize proprietary promoters

ITR

Gene sequence optimization Intron/exon configuration, poly A, translation efficiency, mRNA stability, reduced immunogenicity

ITR

Promoter/ enhancer

Intron Therapeutic gene

Poly A

Vector Engineering

Platforms

Capsid design

Proprietary capsids with high transduction efficiency, including novel intravitreal capsids targeting back or front of the eye - screened directly in NHPs; muscle and CNS capsids

Riboswitch gene regulation

Precise dose responsive control

of in vivo therapeutic protein production with bespoke oral small molecule inducer

Manufacturability

Optimal plasmid design and vector genome

sequence optimization for industry-leading high yield and full/empty ratio



8

Riboswitch platform: precise in vivo production of therapeutic proteins using oral small molecule inducers

Riboswitch technology enables unprecedented

precise in vivo delivery of biologic therapeutics with orally administered inducers

1

Riboswitch-regulated therapeutic transgene is delivered via AAV, other vector, or via gene editing

2 Oral pill induces

precise production of the peptide or protein therapeutic

+

Small molecule

Unbound Aptamer

Expression Platform

OFF

Bound Aptamer

Expression Platform

ON

Riboswitch technology can be applied

across many therapeutic areas and modalities, providing titratable control of gene expression with an oral pill

Vectorized biologics

Cell Therapy

Gene Editing

Short-lived hormones s peptides

Control CNS s PNS Therapies

Control Ocular Therapies





9

A broad range of therapeutic proteins encoded by Riboswitch-containing transgenes show tight control via oral small molecule dosing, in vivo

Therapeutic Antibodies

Cell Therapy

Therapeutic Hormones/Cytokines

/ Peptides

Gene/RNA Editing Nucleases

  • Anti-PCSK9

  • Anti-VEGFR2 (eye)

  • Anti-Amyloid

  • Anti-IL-17

  • Anti-PD1

  • Anti-HER2

  • Anti-IL4Ra

  • Anti-Myostatin

    RiboCAR:

  • Anti-CD19

  • Anti-PSMA

  • Anti-mesothelin

  • Anti-HER2

  • Cytokines

  • ProTcell (progenitor T cell)

    derived riboCAR-T

  • Epo

  • hGH

  • PTH

  • Insulin

  • GLP-1R agonists

  • Gut peptide combinations: GLP1- GIP; GLP1, GIP, PYY,

    Glucagon, Amylin,

    Oxyntomodulin

  • Myokines

  • Adipokines e.g: leptin

  • Cas9

  • CasRx

10



Attention: This is an excerpt of the original content. To continue reading it, access the original document here.

Earlier from Meiragtx

All Meiragtx news releases