DYRK1A inhibition for tauopathy and repetitive head injury
preclinicalresearch program · medium · Thu Jan 01 2026 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate DYRK1A inhibition as a therapeutic approach to mitigate tau pathology, chronic neuroinflammation, and functional deficits after repetitive head injury.
Preclinical study in human Tau transgenic mice subjected to repetitive head injury and treated with SM07883, described as a potent brain-penetrant DYRK1A inhibitor; behavioral and neuropathological assessments were conducted.
A 2026 Neurotherapeutics publication reported preclinical results for SM07883 in repetitive head injury and tauopathy models.
SM07883 restored locomotor performance in injured animals, ameliorated age-related motor decline in sham-treated mice, reduced injury-induced phosphorylated Tau accumulation in midbrain and brainstem, and decreased astroglial and microglial activation in multiple brain regions.
FAM53C/DYRK1A cell-cycle regulation research
exploratoryresearch program · medium · Thu Jan 01 2026 00:00:00 GMT+0000 (Coordinated Universal Time)
Study the FAM53C/DYRK1A axis as a regulator of G1/S cell-cycle transition with potential relevance to cancer, developmental disorders, and other diseases involving DYRK1A dysregulation.
Research using Cancer Dependency Map data, mass spectrometry, biochemical and cellular assays, human cortical organoids, and Fam53C knockout mice to validate FAM53C interaction with and inhibition of DYRK1A.
A 2026 eLife publication identified FAM53C as a regulator of G1/S transition acting upstream of Cyclin D-CDK4/6-RB and p53, and interacting with DYRK1A.
FAM53C was reported as critical for G1/S transition; FAM53C knockout cortical organoids showed increased cell-cycle arrest and growth defects, while Fam53C knockout mice showed minor behavioral phenotypes.
Lorecivivint knee osteoarthritis program
phase 3drug program · high · Wed Jan 01 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Develop an intra-articular treatment for moderately to severely symptomatic knee osteoarthritis, with evaluation of pain, function, radiographic outcomes, long-term safety, and compatibility with corticosteroid use.
Single-dose intra-articular lorecivivint injection; lorecivivint is described as a CLK/DYRK inhibitor, including CLK2/DYRK1A inhibition, and Wnt pathway modulator. Evaluated through randomized placebo-controlled phase 2 and phase 3 trials, open-label and observational extension studies, and drug-drug interaction testing with triamcinolone acetonide.
A 2025 publication reported a multicenter observational extension study of earlier phase 2 trials, and public records list phase 3 STRIDES and related knee osteoarthritis trials.
The 2025 extension publication reported lorecivivint was generally safe and well tolerated, with no treatment-related serious adverse events, similar adverse event incidence between groups, and post hoc durable WOMAC pain and function improvements for at least 12 months in a completer subgroup; no medial joint-space-width differences were observed out to 18 months.
RNA splicing modulation with BCL2 inhibition in leukemia
exploratoryresearch program · medium · Sun Jan 01 2023 00:00:00 GMT+0000 (Coordinated Universal Time)
Investigate whether modulation of RNA splicing can enhance response to BCL2 inhibition in leukemia.
Research publication evaluating RNA splicing modulation in combination with BCL2 inhibition in leukemia.
A 2023 Cell publication titled "Modulation of RNA splicing enhances response to BCL2 inhibition in leukemia" is listed among Biosplice-related publications.
The publication title indicates enhanced response to BCL2 inhibition in leukemia through RNA splicing modulation, but detailed results are not provided in the supplied material.
Cirtuvivint advanced solid tumors program
phase 1drug program · high · Wed Nov 03 2021 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate orally administered SM08502 in combination with hormonal therapy or chemotherapy for adults with advanced solid tumors, including safety, tolerability, pharmacokinetics, and preliminary anti-tumor efficacy.
Open-label, multicenter dose-escalation and dose-expansion clinical trial of oral once-daily SM08502 on a 5-days-on/2-days-off schedule in combination with chemotherapy or hormonal therapy, with recommended Part 2 dose and schedule to be evaluated in expansion cohorts.
ClinicalTrials.gov record published for an open-label multicenter dose-escalation and expansion study in advanced solid tumors.
Alternative splicing therapeutic platform
undisclosedplatform · medium · Thu Apr 15 2021 00:00:00 GMT+0000 (Coordinated Universal Time)
Discover and develop first-in-class therapies that harness alternative splicing by modulating disease-selective RNA splice-site selection and related pathways.
Small-molecule targeting of CLK/DYRK family kinases to modulate alternative splicing; company pipeline includes pan-CLK/DYRK, DYRK1A, CLK2, CLK3, and SM15685 programs.
Biosplice reported a $120 million equity financing in 2021 to advance its alternative splicing platform, and the site analysis lists an active clinical pipeline in osteoarthritis and oncology with earlier neurology, diabetes, and other programs.
