Partnerships Nippon Shinyaku: Japan Distribution Agreement (2023). U.S. Commercialization and Distribution Agreement (Jan 2022) — disputed, Capricor filed for preliminary injunction May 7, 2026; Europe term sheet expired Apr 1, 2026, not extended
Indications
Duchenne muscular dystrophy (DMD)(Phase 3)
Catalysts
— August 22, 2026
Status note BLA submitted late 2024, Priority Review, PDUFA Aug 31, 2025; CRL received Jul 2025. Type A meeting Aug 2025 aligned regulatory path on additional HOPE-3 Phase 3 data; CRL response submitted (FDA accepted, new PDUFA Aug 22, 2026). HOPE-3 published in The Lancet Jul 2026; Cellular, Tissue and Gene Therapies Advisory Committee voted 3-9 against substantial evidence of effectiveness for cardiomyopathy (Jul 29, 2026); BIMO inspection Jul 2026 (Form 483, one observation). Company plans BLA amendment with 24-month open-label extension data. Mesoblast patent infringement suit filed Jul 16, 2026.
Partnerships NIH, NIAID, Johns Hopkins University (research collaborations); Life Technologies/Thermo Fisher (cell line license, Mar 2022); CSMC (Exosomes License Agreement, May 2014)
Indications
Various diseases (therapeutic applications under exploration)(Stage not disclosed)
Status note Platform exploration for targeted RNA, protein and small molecule therapeutics; no clinical candidate stated in excerpts.
Per-asset detail extracted from the 10-Q filed 2026-08-14. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (2 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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