CAMBRIDGE, Mass. – AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today provided an update on its regulatory plans for AVR-RD-01, the first investigational lentiviral gene therapy for Fabry disease. This update follows a recent U.S....
treatment News
CAMBRIDGE, Mass. – AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-04, the company’s investigational gene therapy for the treatment of cystinosis. AVR-RD-04...
NEW YORK, NY — Axsome Therapeutics, Inc., a biopharmaceutical company developing and delivering novel therapies for the management of central nervous system (CNS) disorders, today announced topline results from the CRESCENDO (Characterizing Patient Perspectives on Unmet Needs in Narcolepsy) survey of patients with narcolepsy type 1 (NT1, i.e., narcolepsy with...
REHOVOT, Israel and MONMOUTH JUNCTION, N.J. — Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, today announced that patient enrollment has been completed in the Phase 3 RINGSIDE study evaluating AL102 in desmoid tumors. A total of 156 patients were enrolled. ”Completion of enrollment in RINGSIDE represents a significant...
LONDON, UK — Ayrmid Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., a cell therapy pioneer working to turn cells into powerful therapeutics, today announced that the U.S. Food and Drug Administration (“FDA”) has accepted the Company’s priority review application for omidubicel for the treatment of...
It has only been a month since AstraZeneca’s $39 billion takeover of Alexion was completed, but the merger has already had a setback. Alexion has dropped development of its Ultomiris drug in amyotrophic lateral sclerosis (ALS), chalking up the first clinical failure for the drug since AZ took control. The...
Leiden, Netherlands– Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW study. The randomized, double-blind, placebo-controlled study, conducted in Brazil, involved 13 patients from the age of 12 years who are affected by GM2 gangliosidosis or Niemann-Pick disease type C (NPC). The aim of the...
LEIDEN, Netherlands — Azafaros, a company focused on developing treatments for the unmet needs of patients with rare lysosomal storage disorders, today announced that the first patient has been dosed in the company’s pivotal, multicenter Phase 3 clinical program to evaluate the safety and efficacy of the company’s lead asset,...
Patients with idiopathic pulmonary fibrosis (IPF) being treated with low dose azithromycin for cough did not see significant improvements compared with placebo, according to the results of a recent study published in the Annals of the American Thoracic Society. Researchers conducted a double-blind, randomized placebo-controlled cross-over clinical trial (ClinicalTrials.gov Identifier: NCT02173145) treating...
AzurRx BioPharma Announces Completion of Enrollment in First Cohort of Phase 2b OPTION 2 Extension Study of MS1819 DELRAY BEACH, Fla., Jan. 28, 2021 – AzurRx BioPharma, Inc. (“AzurRx” or the “Company”) (NASDAQ: AZRX), a company specializing in the development of targeted non-systemic, recombinant therapies for gastrointestinal (GI) diseases, today...