Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of th
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Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Partnerships Hologen (Mar 2025 strategic collaboration via Reogen joint venture): $200M upfront + up to $230M to fund AAV-GAD development to commercialization; uses Hologen AI on trial data; company manufactures/supplies drug product
Indications
Parkinson's disease(Phase 1)
Status note Phase 1 bridging study MGT-GAD-025 (14 pts, commercial platform process) completed; data announced Oct 2024. FDA RMAT designation granted May 2025 for Parkinson's disease not adequately controlled with anti-Parkinsonian medications. Hologen collaboration funded to advance to commercialization.
AAV-BDNF
Stage not disclosed · ActiveModality: Gene therapy (local CNS delivery)
Target BDNF
Partnerships Hologen / Reogen joint venture (Clinical Programs); company manufactures/supplies
Indications
Genetic obesity disorders(Stage not disclosed)
Status note Investigational gene therapy for genetic obesity disorders, one of the CNS Clinical Programs under the Hologen/Reogen collaboration. 10-Q does not state trial phase.
AAV-AIPL1
Stage not disclosed · ActiveModality: Gene therapy (subretinal rAAV8.hRKp.AIPL1)
Target AIPL1
Partnerships Eli Lilly (Nov 2025 strategic collaboration and license): exclusive worldwide rights to research, develop and commercialize AAV-AIPL1, plus two other preclinical IRD candidates, intravitreal capsids/promoters licenses, ophthalmology right of first designation
Indications
Leber congenital amaurosis 4 (AIPL1 gene mutations)(Stage not disclosed)
Status note AAV-AIPL1 manufactured/released for compassionate use (MHRA specials license, UK; 11 children). First-in-human interventional study published in The Lancet (Feb 2025). No phase stated in the 10-Q.
AAV-CNGB3
Phase 1/2 · ActiveModality: Gene therapy
Target CNGB3
Indications
Achromatopsia (CNGB3 mutations)(Phase 1/2)
Status note One of three Phase 1/2 IRD programs the company may develop itself or with a strategic partner.
AAV-CNGA3
Phase 1/2 · ActiveModality: Gene therapy
Target CNGA3
Indications
Achromatopsia (CNGA3 mutations)(Phase 1/2)
Status note One of three Phase 1/2 IRD programs the company may develop itself or with a strategic partner.
AAV-RPE65
Phase 1/2 · ActiveModality: Gene therapy
Target RPE65
Indications
Retinal dystrophy (RPE65 deficiency)(Phase 1/2)
Status note One of three Phase 1/2 IRD programs the company may develop itself or with a strategic partner.
AAV-BBS10
Stage not disclosed · ActiveModality: Gene therapy
Target BBS10
Indications
Bardet-Biedl syndrome (BBS10 mutations)(Stage not disclosed)
Status note Manufactured and released for compassionate use (MHRA specials license, UK). No phase stated in the 10-Q.
Per-asset detail extracted from the 10-Q filed 2026-08-13. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Pipeline
Lead drug
AAV2-hAQP1 Concentration 1
Lead indication
Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of th
Sponsor-matched active trials from ClinicalTrials.gov (4 as sponsor, 0 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.
Institutional holder positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.
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