PKU

Participant 1 Achieved Clinically Meaningful Reduction in Plasma Phe of Up to 99% Change from Baseline and Below the U.S. PKU Treatment Guideline Threshold (<360 μmol/L)*; At 31 Weeks Post-Dose, Phe Level 319 μmol/L With a 59% Reduction…

CAMBRIDGE, Mass., July 11, 2023 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage biotechnology company advancing novel, oral, non-systemically absorbed biotherapeutics to transform the care of serious diseases, today…

Registrational study designed to support BLA submission of first-in-class biotherapeutic Company further streamlining organization to focus resources on execution of late clinical-stage programs in rare metabolic diseases CAMBRIDGE,…

JNT-517 demonstrated proof of mechanism and was safe and well tolerated in healthy volunteers Results support advancement to Phase 1b study in individuals with PKU; study now open for enrollment BOSTON, May 31, 2023 (GLOBE NEWSWIRE) --…

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CAMBRIDGE, Mass., Jan. 19, 2023 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), the leading company advancing therapeutics based on synthetic biology, today announced that SYNB1934 was granted Rare Pediatric Disease Designation (RPDD)…

- Oral Presentation on In Vivo Gene Therapy Product Candidate and Data on Gene Editing Candidate Paved Way for Initiation of juMPStart and pheEDIT Trials - BEDFORD, Mass., Oct. 20, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc.…

- IND Clearance of pheEDIT Study to Evaluate a One-Time Dose of Investigational HMI-103 Incorporating a Novel Nuclease-Free Gene Editing Approach - - Company Provides Update on Enrollment in Ongoing pheNIX Trial of HMI-102 Gene Therapy…