AUSTIN, TX – IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients...
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Hello everyone! My name is Claire Bevec and I’m so excited to announce my monthly column that I will be writing for Harmony 4 Hope. I am so thankful for this opportunity to share some of the most beautiful stories about life with a rare disease. Each month I will...
Daix (France) and New York, NY – Inventiva (Euronext Paris and Nasdaq: IVA) (“Inventiva” or the “Company”), a clinical-stage biopharmaceutical company focused on the development of oral therapies for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), today announces the publication in Journal of Hepatology Reports, a peer-reviewed, scientific journal, of results...
COPENHAGEN, Denmark -Genmab A/S(Nasdaq:GMAB) announced today updated data from cohort B1 of the Phase 1/2 RAINFOL-01 study of rinatabart sesutecan (Rina-S®), an investigational folate receptor-alpha (FRα)-targeted, TOPO1 antibody-drug conjugate (ADC) that showed Rina-S 120 mg/m2 every 3 weeks (Q3W) resulted in a confirmed objective response rate (ORR) of 55.6% (95% CI: 30.8-78.5)...
ZUG, Switzerland — Micreos is pleased to share the positive results of an investigational study: Endolysin inhibits skin colonization by patient-derived Staphylococcus aureus and malignant T cell activation in cutaneous T cell lymphoma, published in the Journal of Infectious Disease, 2023 (https://doi.org/10.1016/j.jid.2023.01.039). The study, led by Prof Niels Ødum, University...
The Cullen Education and Research Fund (CERF) announced the recipients of the first CERF Medical Engineering Prize for ALS Research: Dr. Leigh Hochberg, Dr. Conor Walsh, and Dr. Sabrina Paganoni. Their “BrainGate+SoftRobotics” team from Brown University, Harvard University and Massachusetts General Hospital (MGH) is creating a series of technologies intended...
SAN FRANCISCO & HONG KONG – With more than 100,000 patients losing sight to Bietti’s Crystalline Dystrophy (BCD) worldwide, Invincible Vision and ReflectionBio® have joined forces to preserve vision of those impacted. Today, ahead of World Rare Disease Day on February 28, the patient-led nonprofit and biotech company are launching...
SAN FRANCISCO, Calif. — Invitae (NYSE: NVTA), a leading medical genetics company, today announced a partnership with BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, designed to advance genetics-based drug discovery for rare diseases. The goal of the collaboration is to generate new insights...
NEW YORK, NY — IO Biotech (Nasdaq: IOBT), a clinical-stage biopharmaceutical company developing novel, immune-modulatory, off-the-shelf therapeutic cancer vaccines, today announced the publication in Nature Communications of the long-term clinical and immunological outcomes from MM1636, the Phase 1/2 trial evaluating the investigational peptide vaccine IO102-IO103 targeting both tumor cells and immune-suppressive...
GENEVA, Switzerland and AMSTERDAM, The Netherlands — iOnctura, a clinical-stage biotech developing selective cancer therapies against targets that play critical roles in multiple tumor survival pathways, today announces that the US Food and Drug Administration (FDA) has granted permission to proceed with clinical investigations of roginolisib in the United States...