January 2026
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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
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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 indications01 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
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
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Comprehensive vector engineering technologiesPotency 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
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Riboswitch platform: precise in vivo production of therapeutic proteins using oral small molecule inducersRiboswitch technology enables unprecedented
precise in vivo delivery of biologic therapeutics with orally administered inducers
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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
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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
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