by Peter Ciszewski | Jul 18, 2018
The U.S. Food and Drug Administration (FDA) and European Medicines Agency’s (EMA) Committee for Orphan Medical Products (COMP) granted an orphan drug designation to Inozyme Pharma for their product INZ-701 for the treatment of ENPP1 deficiency, a serious,...
by Peter Ciszewski | Jul 18, 2018
Daniel de Boer, Founder and CEO of ProQR, discusses his company’s clinical program focusing on Usher syndrome is a rare genetic disease that is the leading cause of combined deafness and blindness. Patients with Usher syndrome type 2 (USH2), the most...
by Peter Ciszewski | Jul 17, 2018
Editor’s Note: This interview was conducted at #Bio2018. Please pardon the background noise. Dr. Joe Trebley, Co-founder and CEO of Scioto Biosciences, discusses his company’s focus on developing a novel probiotic delivery platform that provides enhanced...
by Peter Ciszewski | Jul 16, 2018
The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to dilanubicel (Nohla Therapeutics) for the reduction of morbidity and mortality associated with hematopoietic stem cell transplant (HSCT). Dilanubicel is a universal donor, off-the-shelf-, ex...
by Peter Ciszewski | Jul 14, 2018
Statement from FDA Commissioner Scott Gottlieb, MD on agency’s efforts to advance development of gene therapies Once just a theory, gene therapies are now a therapeutic reality for some patients. These platforms may have the potential to treat and cure some of our...
