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MeiraGTx

MGTX Rare disease Lead: Phase 3 1 Biotech Distilled article ↓ 19 filings & releases ↓ Cash $143M (Jun 30, 2026) ↓ 1 verdict ↓
ImmunologyMetabolicNeurologyOphthalmology Gene Therapy

Market cap $864M 96.0M shares (10-Q, Aug 11, 2026) × $9.00 close Nov 6, 2026

Most recent annual filing: 2026-03-30

Catalyst timeline

5 trackedQuarter catalyst calendar →
  1. Bota-vec BLA filing2026Bota-vec BLA filing, 2026, notable
  2. Beacon Therapeutics laru-zova VISTA readout2H 2026Beacon Therapeutics laru-zova VISTA readout, 2H 2026, notable
  3. Bota-vec FDA approval2027Bota-vec FDA approval, 2027, material
  4. AAV-hAQP1 BLA filing1H 2027AAV-hAQP1 BLA filing, 1H 2027, notable
  5. 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)

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

Capital (as filed)

Cash
$143M cash and cash equivalents · Jun 30, 2026 (10-Q)
Long-term investments
$39.8M Jun 30, 2026
Operating cash flow
−$38.5M quarter Mar 31, 2026 → Jun 30, 2026, year-to-date difference (10-Q)
As stated in coverage
$145M cash per the deep dive of Sep 4, 2026 — the company's own words as read there, not the filed figure

Cash, investments and operating cash flow are the company's own XBRL facts as filed with the SEC, each shown with its period end and tag. As-stated figures come from the company's own words as read in Biotech Distilled coverage, dated, and routinely count securities and planned financing that a balance sheet does not. Offering rows are the SEC cover pages as filed. Not investment advice. Figures as of Nov 7, 2026.

Biotech Distilled coverage (1)

Articles open on Substack. Some are subscriber-only; those marked ★ Distilled Pro are for Distilled Pro members.

Verdict history (1 call)

Every verdict Biotech Distilled has published on MeiraGTx, oldest first — the full history, never a selection. How calls are scored: the track record.

  1. 2026-09-08 Watch MeiraGTx Holdings (MGTX) — Scientific Deep Dive for AAV-hAQP1, Bota-vec, AAV-GAD and Pipeline Products The AQP1 mechanism is elegant and human-validated, but three of four 'pivotal' programs rest on failed, per-protocol, or compassionate-use datasets, and the fourth is 24 open-label patients on a questionnaire with no validated threshold; revisit on the AQUAx2 readout in Q2 2027, a sub-$9 print, or Hologen paying the $95M.

Every verdict Biotech Distilled has published on this company, in order, with the post it came from — the full history, never a selection. Position figures are the same ones shown on /track-record, computed by the same method. Editorial opinion, not investment advice.

Filings & releases (19)

SEC filings from EDGAR and press releases captured from the company's investor-relations site, newest first. Releases captured from Sep 23, 2026; filings from March 2026. Summaries come from the BD update that read the document; "BD extracted" counts point at the pipeline table above. All companies →

4 more filings (13G holder notices, S-8, proxy material, SEC correspondence …)