July 21, 2025 -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, announced today that it has been granted orphan drug designation by the European Medicines Agency (EMA) Committee, who adopted a positive opinion on KRRO-110, Korro’s investigational medicine for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). “The EMA orphan drug desi

July 18, 2025 -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, announced that three posters on MIPLYFFA® (MY-PLY-FAH) (arimoclomol) and one on OLPRUVA® (sodium phenylbutyrate) are being presented at the 42nd Annual Meeting of the Southeastern Regional Genetics Group (SERGG), taking place July 17-19, 2025, in Asheville, North Carolina. MIPLYFFA is approved in the U.S. for the treat

July 17, 2025 -- BioAtla, Inc. (Nasdaq: BCAB) (the “Company” or “BioAtla”), a global clinical-stage biotechnology company focused on the development of Conditionally Active Biologic (CAB) antibody therapeutics for the treatment of solid tumors, today announced an oral presentation at the upcoming 2025 European Society for Medical Oncology (ESMO) TAT Asia Meeting to be held in Hong Kong Sar, China from July 18–20, 2025. Title: First-in-human phase I study of a dual-Conditionally Active Biologi

- Zervimesine-treated participants tested 86% better on behavioral outcomes (NPI 12), 52% on activities of daily living, 91% on cognitive fluctuations, and 62% on motor symptoms as compared to placebo - - Additional presentations highlight positive clinical and biomarker effects of zervimesine in the low p-tau217 population in Phase 2 Alzheimer’s disease study - July 16, 2025 -- Cognition Therapeutics, Inc., (the Company or Cognition) (NASDAQ: CGTX), a clinical stage company developing drugs

July 17, 2025 -- Keros Therapeutics, Inc. (“Keros”) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta (“TGF-ß”) family of proteins, today announced that the first patient was dosed in the Phase 3 RENEW clinical trial of elritercept in adults with transfusion-dependent anemia with very low, low, or

July 18, 2025 -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced that data from a study of patients describing the impact of diabetic peripheral neuropathic pain (DPNP) despite treatment with standard of care will be presented at the 7th Annual Meeting of the American Society of Pain and Neuroscience (ASPN 2025). The conference is being held July 17 - 20, 2025 at the Fontainebleau Miami Beach Hotel in Miami Beach, Florida. DPNP is a highly prevalent but often overlooked debilitati

July 18, 2025. Sanofi today announces the completion of its acquisition of Blueprint Medicines Corporation (Blueprint), adding to its portfolio a commercialized medicine, a promising pipeline, and the expertise of a company specializing in systemic mastocytosis (SM), a rare immunological disease, and other KIT-driven diseases. In addition, the acquisition of Blueprint brings Sanofi an established presence among allergists, dermatologists, and immunologists which is expected to enhance Sanofi’s

July 17, 2025 -- OKYO Pharma Limited (NASDAQ: OKYO), an ophthalmology-focused bio-pharmaceutical company which is developing urcosimod to treat neuropathic corneal pain (NCP), an ocular condition associated with chronic and often severe nerve-related pain but without an FDA-approved therapy, today announced it has received $1.9 million in non-dilutive funding to support its ongoing research and development programs. The funding will specifically accelerate the clinical development of urcosimod,

- Announces Completion of Phase 1 Study of Elraglusib in Pediatric Patients - Prolonged and Durable Complete Responses (CRs) Observed in Two of Ten Refractory Ewing Sarcoma Patients - Two Additional Patients Had Durable Stable Disease in Notoriously Difficult-to-Treat Cancer - Confirmed Partial Response (PR) in a patient with a desmoplastic small-round-cell tumor (DSRCT) - Company Initiating Planning of Phase 2 Trial of Elraglusib in Children, Adolescents, and Adults with Refractory/Resistant Ew

Clinical data showing unprecedented remission rates in newly diagnosed AML patients support advancing ICT01 into pivotal trials July 18, 2025, 11:00 am CET – ImCheck Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead program, ICT01, a humanized anti-butyrophilin 3A (BTN3A) monoclonal antibody designed to selectively activate γ9δ2 T cells, for the treatment of acute myeloid leukemia (AML). AML remains a significant