CAMBRIDGE, Mass. – AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a vision to free people from a lifetime of genetic disease, hosted a panel of investigators managing the patient experience in the Phase 2 FAB-GT clinical trial of AVR-RD-01, an investigational one-time gene therapy for Fabry...
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CAMBRIDGE, Mass. – AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a vision to free people from a lifetime of genetic disease, today hosted a panel of investigators managing the patient experience in the Phase 2 FAB-GT clinical trial of AVR-RD-01, an investigational one-time gene therapy for Fabry...
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....
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...
Shenzhen, China – Thalassemia, a hereditary hemoglobinopathy, occurs in 4.4 out of every 10,000 live births and is prevalent in Mediterranean coastal areas, Africa, the Middle East, Southeast Asia, and southern China. Screening and antenatal diagnosis reduced the frequency of β-thalassemia in many Mediterranean countries. Focusing on other regions with...
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...