CAMBRIDGE, Mass. – AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases, today announced new additions to its leadership team with the appointments of Paul Herzich as Chief Technology Officer and Jared Simons as Vice President of Manufacturing. Both Messrs. Herzich and Simons...
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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 activation of a second clinical trial site in its pivotal Phase 3 VIITAL™ study of its investigational EB-101 treatment for recessive dystrophic epidermolysis bullosa (RDEB) at UMass Memorial Medical Center...
NEW YORK and CLEVELAND – Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, announced the appointment of two with significant biotechnology and life sciences experience: Faith L. Charles, Partner, Corporate Transactions and Securities at Thompson Hine LLP and Mark Alvino, President at life sciences strategic...
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...
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...
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...
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...