ImmuneSensor Therapeutics Receives Orphan Drug and Rare Pediatric Disease Designations from the U.S. FDA for cGAS Inhibitor Drug Candidate, IMSB301, for the Treatment of Aicardi Goutières Syndrome (AGS) Company is evaluating IMSB301 in a Phase 1 clinical trial in healthy volunteers and plans to move rapidly into Phase 1b/2 clinical studies in patients with AGS, lupus and other cGAS-driven diseases ...

ConSynance Therapeutics Announces U.S. FDA Grants Rare Pediatric Disease Designation to CSTI-500, a Potential First-in-Class Therapy for Prader-Willi Syndrome FDA awards ConSynance's CSTI-500 Rare Pediatric Disease Designation for innovative Prader-Willi Syndrome treatment.

Synlogic Receives Rare Pediatric Disease Designation from FDA for SYNB1934 for Phenylketonuria (PKU) 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...

Synlogic Provides Corporate Update and Outlook for 2023 Pivotal Phase 3 for SYNB1934 in phenylketonuria (PKU) to initiate in H1 2023 Rare Pediatric Disease Designation received for SYNB1353 for homocystinuria (HCU) Cash runway expected into H2 2024 ...