Boundless Bio: SERP-01 AAT exceeds protective level
The interim dataset covers four participants, and Serapha intends to initiate a U.S. Phase 2/3 trial in late 2026 or early 2027.
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Rhea-AI Filing Summary
Boundless Bio, Inc. furnished Serapha Bio’s interim clinical update on SERP-01 in connection with their proposed merger. The update reports data from an ongoing investigator-initiated trial of four PiZZ participants receiving one dose of the investigational base-editing therapy.
As of October 15, 2026, total serum AAT exceeded the 11 µM protective threshold in all four participants. By week 3, M-AAT was 98.7% at 35 mg and 93.4% at 45 mg; in the 45 mg cohort, total AAT was at or near the 20 µM lower limit of normal through up to 10 months of follow-up. A biopsy at 10 weeks from the 35 mg participant showed 54% target-site correction, with no bystander editing detected at neighboring bases. No serious adverse events or treatment-related adverse events of grade 3 or higher were observed as of October 15, 2026. Serapha intends to initiate a U.S. Phase 2/3 trial in late 2026 or early 2027. The proposed merger remains subject to satisfaction or waiver of Merger Agreement conditions, and the related S-4 has not yet been declared effective.
Filing Explained
The update adds functional-activity evidence from four participants, alongside FDA clearance of the planned trial’s IND.
Serapha reports that the FDA cleared the IND for SERP-01’s planned Phase 2/3 trial; the company still describes trial initiation as a plan for late 2026 or early 2027.
SER P-01 also has orphan drug and Regenerative Medicine Advanced Therapy designations; the filing says these do not ensure or accelerate approval.
In an elastase-inhibition assay one and two months after infusion, serum from three of four participants approached healthy-donor activity, and all four exceeded the PiZZ control.
The results are from an ongoing, third-party investigator-initiated trial of four participants that is not designed to support regulatory approval; the filing says longer follow-up and more participants are needed.
Key Figures
Key Terms
investigator-initiated trial (IIT) medical
liquid chromatography/mass spectrometry assay technical
elastase inhibition assay medical
Regenerative Medicine Advanced Therapy (RMAT) designation regulatory
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