RegenxBio Bets Its Gene Therapy Technology Provides Competitive Edge

The U.S. Food and Drug Administration recently…

Rare Leader: Beth McGinn, Co-founder and Executive Director, A Cure for Ellie
The Basics Name:  Beth McGinn Title: Co-founder and executive director Organization: A Cure for Ellie Disease focus: Leukoencephalopathy… Continue Reading
Orchard Therapeutics Reports Clinical Proof-of-Concept Data for X-CGD Gene Therapy at ASH
Rare Daily Staff Orchard Therapeutics said it presented the first demonstration that its experimental gene therapy OTL-102 for the treatment… Continue Reading
Mereo BioPharma to Merge with OncoMed Pharmaceuticals
Rare Daily Staff Rare disease drug developer Mereo BioPharma Group and cancer therapeutics developer OncoMed Pharmaceuticals, two… Continue Reading
The 100,000 Genomes Project Reaches Its Goal
Rare Daily Staff The 100,000 Genomes Project, led by Genomics England in partnership with NHS England, has reached its goal of sequencing… Continue Reading
Abeona Expands Pipeline with Addition of Cystic Fibrosis Gene Therapy
Rare Daily Staff Abeona Therapeutics said it expanded its pipeline with the addition of ABO-401, an experimental gene therapy for cystic… Continue Reading
GAO Finds FDA Granted Orphan Drug Designation to Some Applications Missing Required Information
Rare Daily Staff The Government Accountability Office in a new report said that the U.S. Food and Drug Administration granted orphan drug… Continue Reading
U.S. Food and Drug Administration Orphan Drug Designations in November 2018
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