NEW YORK and CLEVELAND – Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced its sponsorship of the second annual Venture Into Cures, a virtual event supporting EB Research Partnership’s mission to find a cure for epidermolysis bullosa (EB). The event will take place virtually...
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NEW YORK and CLEVELAND – Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced the appointment of Dr. Leila Alland and Mr. Donald Wuchterl as new independent members to its Board of Directors. “Leila and Donald bring a collective wealth of diverse biologics experience...
BOSTON – AbFero Pharmaceuticals, Inc., a privately-held clinical stage pharmaceutical company dedicated to treating diseases of iron overload, today announced the initiation of a Phase 1 study of its lead iron chelator, SP-420, in myelodysplastic (MDS) and myelofibrosis patients (MF) with transfusional iron overload (TIO). “The opening of this trial...
TOULOUSE, France – ABIONYX Pharma (FR0012616852 – ABNX – PEA PME eligible), a new generation biotech company dedicated to innovative therapies for patients, today announces positive clinical results from CER-001 in the LCAT (lecithin-cholesterol acyltransferase) deficiency disease published in the Annals of Internal Medicine. The patient with inherited mutations in the lecithin-cholesterol...
PARIS, FRANCE – Abivax SA (Euronext Paris:ABVX))(NASDAQ:ABVX), a clinical-stage biotechnology company focused on developing therapeutics that harness the body’s natural regulatory mechanisms to stabilize the immune response in patients with chronic inflammatory diseases, today announced that four scientific abstracts on its lead drug candidate, obefazimod for the treatment of moderately to...
LUND, Sweden — Abliva AB (Nasdaq Stockholm: ABLI), a clinical-stage company developing drugs for the treatment of rare and severe primary mitochondrial disease, today announced a positive outcome of the interim analysis for FALCON, the potentially registrational study evaluating KL1333 in patients with primary mitochondrial disease. The study evaluates fatigue...
Patient registries collect information about the health status of people with a certain disease. They are particularly important for rare diseases such as Lambert-Eaton myasthenic syndrome (LEMS), bringing together a small patient population and gathering information about how the disease affects them. Researchers in academia and pharmaceutical companies can then use this...
– Trofinetide met co-primary efficacy endpoints demonstrating statistically significant improvement over placebo in the Rett Syndrome Behaviour Questionnaire (RSBQ) (p=0.0175) and the Clinical Global Impression of Improvement (CGI-I) (p=0.0030) – Trofinetide met key secondary endpoint demonstrating statistically significant improvement over placebo in CSBS-DP-IT–Social (p=0.0064), caregiver scale of ability to communicate...
SAN DIEGO, Calif. — Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has accepted its New Drug Submission (NDS) for trofinetide for the treatment of Rett syndrome, a rare neurodevelopmental disorder. Health Canada has granted Priority Review for Acadia’s submission. “Rett syndrome is a profoundly debilitating and complex...
According to the results of a phase 1/2 study, the Bruton tyrosine kinase (BTK) inhibitor acalabrutinib was safe and efficacious, with durable remissions, in patients with previously untreated chronic lymphocytic leukemia (CLL). The findings were reported in Blood. The single-arm study, ACE-CL-001 (ClinicalTrials.gov Identifier: NCT02029443), evaluated efficacy and safety of acalabrutinib monotherapy...