AAV-hAQP1
Gene Therapy Target: AQP1, hAQP1
- Radiation-Induced Xerostomia
- Sjogren's Syndrome
MeiraGTx
Most recent annual filing: 2026-03-30
- Bota-vec BLA filing2026Bota-vec BLA filing, 2026, notable
- Beacon Therapeutics laru-zova VISTA readout2H 2026Beacon Therapeutics laru-zova VISTA readout, 2H 2026, notable
- Bota-vec FDA approval2027Bota-vec FDA approval, 2027, material
- AAV-hAQP1 BLA filing1H 2027AAV-hAQP1 BLA filing, 1H 2027, notable
- AAV-hAQP1 pivotal topline2Q 2027AAV-hAQP1 pivotal topline, 2Q 2027, thesis-defining
Bar width shows how precise the guidance is — a pin is a stated date, a wide band a six-month window. Deeper blue means more of the thesis rides on the event; it is not a view on the stock.
Pipeline (16 active programs)
AAV-GAD
Gene Therapy Target: GAD, GABA
AAV-UPF1
Gene Therapy Target: UPF1
meduretgene parvec
AAV-AIPL1 · medu-vec Gene Therapy Target: AIPL1
AAV-CNGB3
Gene Therapy Target: CNGB3
- Achromatopsia
AAV-CNGA3
Gene Therapy Target: CNGA3
- Achromatopsia
AAV-RPE65
Gene Therapy Target: RPE65
- RPE65-Associated Retinal Dystrophy
AAV-BBS10
Gene Therapy Target: BBS10
AAV-RDH12
Gene Therapy Target: RDH12
- RDH12 Mutation-Associated Retinal Dystrophy
AAV-RetGC
Gene Therapy Target: GUCY2D, RetGC
AAV-BDNF
Gene Therapy Target: BDNF
Ribo-leptin
Gene Therapy Target: leptin
- Leptin Deficiency
Ribo-Leptin
Gene Therapy Target: leptin
- Undisclosed indication
RPGR Product
Gene Therapy Target: RPGR
Technology platforms
Riboswitch Gene Regulation Platform
Proprietary synthetic riboswitch technology enabling precise, dose-responsive control of gene expression via oral small molecules; focused on in vivo delivery of metabolic peptides (GLP-1, GIP, glucagon, amylin, PYY, leptin), cell therapy for oncology and autoimmune diseases, and long-term intractable pain
ProTcell
T-cell progenitor-based allogeneic cell therapy platform acquired from Smart Immune; harnesses the patient's own thymus to re-arm the immune system; intended for off-the-shelf allogeneic CAR-T therapies incorporating MeiraGTx riboswitch technology; focus areas include cancer and autoimmune conditions
Riboswitch · Eli Lilly and Company
Proprietary gene regulation platform enabling precise, dose-responsive in vivo delivery of any biologic therapeutic (peptides, hormones, proteins) via oral small molecule inducers. Disease focus areas include metabolic disease (leptin, GLP-1, GIP, Glucagon, Amylin, PYY), cell therapy (CAR-T for liquid/solid tumors and autoimmune diseases), neuropathic/intractable pain, and ophthalmology (gene editing in the eye via Lilly collaboration).
Targets: GLP-1, GIP, Glucagon, Amylin, PYY, Leptin
Gene Regulation
Proprietary synthetic riboswitch technology enabling precise, dose-responsive expression of any transgene controlled by oral small molecules; focused on in vivo delivery of biologic therapeutics (GLP-1, GIP, glucagon, amylin, PYY, leptin), cell therapy for oncology and autoimmune diseases, and long-term intractable pain.
Riboswitch Gene Regulation Technology · Eli Lilly and Company
In vivo gene regulation platform enabling dose-responsive, oral small molecule control of gene expression; focused on regulated delivery of metabolic peptides (GLP-1, GIP, Glucagon, Amylin, PYY, Leptin), CAR-T cell therapy for liquid and solid tumors and autoimmune diseases, and PNS targets for chronic pain. Lead clinical program targets inherited and acquired leptin deficiency.
Targets: GLP-1, GIP, Glucagon, Amylin, PYY, Leptin
Intravitreal Capsids · Eli Lilly and Company
Proprietary intravitreal AAV capsid platform for ocular gene therapy delivery; licensed to Eli Lilly as part of ophthalmology collaboration.
AI Driven Promoter Platform · Eli Lilly and Company
Artificial intelligence-driven platform for generating bespoke gene promoters for ocular and other gene therapy applications; licensed to Eli Lilly as part of ophthalmology collaboration.
Riboswitch Gene Regulation Platform · Sanofi
Proprietary synthetic riboswitch technology enabling precise, dose-responsive expression of any transgene under control of oral small molecules. Applications include in vivo delivery of metabolic peptides (GLP-1, GIP, glucagon, amylin, PYY, leptin), CAR-T cell therapy for oncology and autoimmune diseases, and gene editing nuclease regulation.
Targets: GLP-1, GIP, glucagon, amylin, PYY, leptin, IL-4, IL-13
Riboswitch Gene Regulation Platform · Eli Lilly and Company
Proprietary riboswitch technology enabling in vivo delivery of biologic therapeutics using oral small molecules; allows precise, dose-responsive gene expression regulation. Focus areas include metabolic peptides (GLP-1, GIP, glucagon, amylin, PYY, leptin), CAR-T cell therapy for liquid and solid tumors and autoimmune diseases, and PNS targets for intractable pain.
Riboswitch Gene Regulation Technology
Proprietary in vivo gene regulation platform enabling dose-responsive control of gene expression via oral small molecules; focused on regulated delivery of metabolic peptides (GLP-1, GIP, Glucagon, Amylin, PYY, Leptin), CAR-T cell therapy for liquid/solid tumors and autoimmune diseases, and PNS targets for chronic pain
Riboswitch
Gene regulation technology platform enabling precise, dose-responsive in vivo control of any biologic therapeutic transgene expression via daily oral small molecule; applications include metabolic peptides (GLP-1, GIP, Glucagon, Amylin, PYY, Leptin), CAR-T (liquid/solid tumors, autoimmune), and PNS targets for neuropathic pain. Delivered via AAV, lentivirus, CRISPR, or LNPs.
Targets: GLP-1, GIP, Glucagon, Amylin, PYY, Leptin
Discontinued (1)
- botaretigene sparoparvovec — X-Linked Retinitis Pigmentosa · divested via Asset Purchase Agreement to Johnson & Johnson Innovative Medicine in December 2023
Biotech Distilled coverage (1)
- 2026-09-08 MeiraGTx Holdings (MGTX) — Scientific Deep Dive for AAV-hAQP1, Bota-vec, AAV-GAD and Pipeline Products 🔒 Paid
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